                         SEQUENCE LISTING

<110>  Fred Hutchinson Cancer Research Center
       Bender, Michael A.  M.D., Ph.D.
       Groudine, Mark  T. M.D., Ph.D.
       Stoddard, Barry L.  Ph.D.
       Takeuchi, Ryo   Ph.D.
 
<120>  COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

<130>  54428.0006WOU1

<140>  TBD
<141>  2013-02-22

<150>  US 61/603,231
<151>  2012-02-24

<160>  69    

<170>  PatentIn version 3.5

<210>  1
<211>  3603
<212>  DNA
<213>  Homo sapiens

<400>  1
ccagtgagca ggttggttta agataagcag ggtttcatta gtttgtgaga atgaaaaatg       60

aaccttcatt ccactattcc cttaacttgc cctgagattg gctgttctgt catgtgtgtc      120

ttgactcaga aaccctgttc tcctctacat atctccccac cgcatctctt tcagcagttg      180

tttctaaaaa tatcctccta gtttcatttt tgcagaagtg ttttaggcta atatagtgga      240

atgtatctta gagtttaact tatttgtttc tgtcacttta tactaagaaa acttatctaa      300

aagcagatgt tttaacaagt tgactcaata taaagttctt ctttgcctct agagattttt      360

gtctccaagg gaattttgag aggttggaat ggacaaatct attgctgcag tttaaacttg      420

cttgcttcct ccttcttttg gtaaattctt cctataataa aactctaatt ttttattata      480

ttgaaataaa tatccattaa aagaatattt aaaaaatgaa tagtgtttat ttaccagtta      540

ttgaaatagg ttctggaaac atgaatttta aggttaacat tttaatgaca gataaaatca      600

aatattatat acaaatattt tgaatgttta aaattatggt atgactaaag aaagaatgca      660

aagtgaaaag tagatttacc atattcagcc agattaaatt taacgaagtt cctgggaata      720

tgctagtaca gaacattttt acagatgtgt tcttaaaaaa aaatgtggaa ttagacccag      780

gaatgaagat cccagtagtt tttcactctt ttctgaattc aaataatgcc acaatggcag      840

acaaatacac acccatgagc atatccaaaa ggaaggattg aaggaaagag gaggaagaaa      900

tggagaaagg aaggaaggaa gaggggaaga gagaggatgg aagggatgga ggagaagaag      960

gaaaaataaa taatggagag gagaggagaa aaaaggaggg gagaggagag gagaagggat     1020

agggaagaga aagagaaagg gaagggaaga gaggaaagaa gagaagagga gagaaaagaa     1080

acgaagagag gggaagggaa ggaaaaaaaa gaggaaaaaa gagacaagag aagagataag     1140

actgacagtt caaattttgg tggtgatatg gatcaataga aactcaaact ctgttggtga     1200

cactgtacaa tagtataacc cctttggaaa acctttaata gtatccacaa atgctggatg     1260

cttgataagt ctattaccta gcaattacat ttttagatat tcagaaacac atgcatgtgt     1320

gtatccaaag acatgtatag aaatgcttat gacagcaata atcataaaaa cctcaaaccg     1380

gtagccactt aaatgcttac caacagtaga attgataaat tacggtatag tcaaagaata     1440

gaatattaca cagaaatgaa aagaatcaac tactgcttaa cacgtagcga tacaaatgca     1500

ttttacagca tttggttgat taaaagtaac cagaggtgag ttcaaactat atgactttat     1560

ttgtatatag aaagatggat gatgtgcctg agattctgat cacaagggga aatgttataa     1620

aatagggtag agaggagcca tgaatgacct ttaaactttg ttacaagtta tttttctgta     1680

acctggaagc caacgaaaga tattgaataa ttcaagaaag gtggtggcat ggtttgattt     1740

gtgtctttaa aagattattc tcacttagtg aagaaatgta ttttagaagt agagaaaatg     1800

ggagacaaat agctgggctt ctgttgcagt agggaagaaa gtgacaatgc catttctatt     1860

atcagacttg gaccatgacg gtgatgtcag tcgtgaacac aagaataggg ccacatttgt     1920

gagtttagtg gtacgataaa atcagaaata cagtcttgga tacattgtat tgtatgcact     1980

cttgtaaaat gcaaaaagat gtacttagat atgtggatct ggagctcaga aagaatacaa     2040

ccaggtcaag aatacagaat ggaacagaac atacaagaac agatcataat gtgctgtgtg     2100

aatcactacc actacctgtt aaaaatgaca gatgatgtac ttcatcaata tctccttaaa     2160

atcttagaat gtgtttgtga gggaggaatt atgtttccaa ttcatatata agaaaattga     2220

ttctaaaaaa aatgttaggt aaattcttaa ggccatgagg actgttattt gatctttgtc     2280

tgttaattcc aaagacttgg cttttcactt taattctgtt ctacctgaaa tgattttaca     2340

cattgggaga tctggttaca tgtttattct atatggattg cattgagagg atttgtataa     2400

cagaataagg tctttttttc ttttctcttc tgagatggag tttcatccct attgcccaag     2460

ctagagtgca atggtgcaat ctaggctcac cgcaacctct gcctcctggg ttcaagcaat     2520

tctcctgcct cagccacctg aatagctggg actgcaggca tgcaccacac gcccggctga     2580

ttttgtattt ttagtagaga tggggtttca ccatgttggt caggctggtc ttgaactcct     2640

gacctcaagt gatctgcctg ccttggcctc ccaaagtgct gggtttacaa gcctgagcca     2700

ccgcatccag ccaggataag gtctaaaagt ggaaagaata gcatctactc ttgttcagga     2760

aacaatgagg acctgactgg gcagtaagag tggtgattaa tagataggga caaattgaag     2820

cagaatcgaa ctgttgatta gaggtaggga aatgatttta atctgtgacc ttggtgaatg     2880

ggcaagtagc tatctaatga ctaaaatgga aaacactgga agagaaacag ttttagtata     2940

acaagtgaaa tacccatgct gagtctgagg tgcctatagg acatctatat aaataagccc     3000

agtacattgt ttgatatatg ggtttggcac tgaggttgga ggtcagaggt tagaaatcag     3060

agttgggaat tgggattata caggctgtat ttaagagttt agatataact gtgaatccaa     3120

gagtgtgatg aatacaaagt taaatgaagg acctttaatg aacaccaaca tttaatgtga     3180

aatctcaagg aagtatgaag taagacatag tccccaaaat ccccgatgat tttagaactc     3240

agtatcgatt ttaattagtg taatgccaat gtgggttaga atggaagtca acttgctgtt     3300

ggtttcagag caggtaggag ataaggttct agattttgac acagtgaaaa gctgaaacaa     3360

aaaggaaaag gtagggtgaa agatgggaaa tgtatgtaag gaggatgagc cacatggtat     3420

gggaggtata ctaaggactc tagggtcaga gaaatatggg ttatatcctt ctacaaaatt     3480

cacattcttg gctgggtgtg gtggctcacg cctgtgatcc cagcactttc agaggccgag     3540

gagggtggat cacctgatgt taggagttcg agatcagcct gaccaacatg gtgaaacccc     3600

cta                                                                   3603


<210>  2
<211>  350
<212>  DNA
<213>  Homo sapiens

<400>  2
aaagatggat gatgtgcctg agattctgat cacaagggga aatgttataa aatagggtag       60

agaggagcca tgaatgacct ttaaactttg ttacaagtta tttttctgta acctggaagc      120

caacgaaaga tattgaataa ttcaagaaag gtggtggcat ggtttgattt gtgtctttaa      180

aagattattc tcacttagtg aagaaatgta ttttagaagt agagaaaatg ggagacaaat      240

agctgggctt ctgttgcagt agggaagaaa gtgacaatgc catttctatt atcagacttg      300

gaccatgacg gtgatgtcag tcgtgaacac aagaataggg ccacatttgt                 350


<210>  3
<211>  21
<212>  DNA
<213>  Homo sapiens

<400>  3
gtgcacctca ctccagagga g                                                 21


<210>  4
<211>  21
<212>  DNA
<213>  Homo sapiens

<400>  4
gtgcacctca ctccagtgga g                                                 21


<210>  5
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  5
tttccactta ttcaaccttt ta                                                22


<210>  6
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  6
tgtggcccta ttcttgtgtt ca                                                22


<210>  7
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  7
cattgtcact ttcttcccta ct                                                22


<210>  8
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  8
taaaatacat ttcttcacta ag                                                22


<210>  9
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  9
actaagtgag aataatcttt ta                                                22


<210>  10
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  10
gccaccacct ttcttgaatt at                                                22


<210>  11
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  11
tcttgaatta ttcaatatct tt                                                22


<210>  12
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  12
ttaaaggtca ttcatggctc ct                                                22


<210>  13
<211>  2606
<212>  DNA
<213>  Homo sapiens

<400>  13
gcaatgaaaa taaatgtttt ttattaggca gaatccagat gctcaaggcc cttcataata       60

tcccccagtt tagtagttgg acttagggaa caaaggaacc tttaatagaa attggacagc      120

aagaaagcga gcttagtgat acttgtgggc cagggcatta gccacaccag ccaccacttt      180

ctgataggca gcctgcactg gtggggtgaa ttctttgcca aagtgatggg ccagcacaca      240

gaccagcacg ttgcccagga gctgtgggag gaagataaga ggtatgaaca tgattagcaa      300

aagggcctag cttggactca gaataatcca gccttatccc aaccataaaa taaaagcaga      360

atggtagctg gattgtagct gctattagca atatgaaacc tcttacatca gttacaattt      420

atatgcagaa atatttatat gcagagatat tgctattgcc ttaacccaga aattatcact      480

gttattcttt agaatggtgc aaagaggcat gatacattgt atcattattg ccctgaaaga      540

aagagattag ggaaagtatt agaaataaga taaacaaaaa agtatattaa aagaagaaag      600

cattttttaa aattacaaat gcaaaattac cctgatttgg tcaatatgtg tacacatatt      660

aaaacattac actttaaccc ataaatatgt ataatgatta tgtatcaatt aaaaataaaa      720

gaaaataaag tagggagatt atgaatatgc aaataagcac acatatattc caaatagtaa      780

tgtactaggc agactgtgta aagttttttt ttaagttact taatgtatct cagagatatt      840

tccttttgtt atacacaatg ttaaggcatt aagtataata gtaaaaattg cggagaagaa      900

aaaaaaagaa agcaagaatt aaacaaaaga aaacaattgt tatgaacagc aaataaaaga      960

aactaaaacg atcctgagac ttccacactg atgcaatcat tcgtctgttt cccattctaa     1020

actgtaccct gttacttatc cccttcctat gacatgaact taaccataga aaagaagggg     1080

aaagaaaaca tcaagcgtcc catagactca ccctgaagtt ctcaggatcc acgtgcagct     1140

tgtcacagtg cagctcactc agtgtggcaa aggtgccctt gaggttgtcc aggtgagcca     1200

ggccatcact aaaggcaccg agcactttct tgccatgagc cttcacctta gggttgccca     1260

taacagcatc aggagtggac agatccccaa aggactcaaa gaacctctgg gtccaagggt     1320

agaccaccag cagcctaagg gtgggaaaat agaccaatag gcagagagag tcagtgccta     1380

tcagaaaccc aagagtcttc tctgtctcca catgcccagt ttctattggt ctccttaaac     1440

ctgtcttgta accttgatac caacctgccc agggcctcac caccaacttc atccacgttc     1500

accttgcccc acagggcagt aacggcagac ttctcctcag gagtcagatg caccatggtg     1560

tctgtttgag gttgctagtg aacacagttg tgtcagaagc aaatgtaagc aatagatggc     1620

tctgccctga cttttatgcc cagccctggc tcctgccctc cctgctcctg ggagtagatt     1680

ggccaaccct agggtgtggc tccacagggt gaggtctaag tgatgacagc cgtacctgtc     1740

cttggctctt ctggcactgg cttaggagtt ggacttcaaa ccctcagccc tccctctaag     1800

atatatctct tggccccata ccatcagtac aaattgctac taaaaacatc ctcctttgca     1860

agtgtattta cgtaatattt ggaatcacag cttggtaagc atattgaaga tcgttttccc     1920

aattttctta ttacacaaat aagaagttga tgcactaaaa gtggaagagt tttgtctacc     1980

ataattcagc tttgggatat gtagatggat ctcttcctgc gtctccagaa tatgcaaaat     2040

acttacagga cagaatggat gaaaactcta cctcggttct aagcatatct tctccttatt     2100

tggattaaaa ccttctggta agaaaagaaa aaatatatat atatatgtgt gtatatatac     2160

acacatacat atacatatat atgcattcat ttgttgttgt ttttcttaat ttgctcatgc     2220

atgctaataa attatgtcta aaaatagaat aaatacaaat caatgtgctc tgtgcattag     2280

ttacttatta ggttttggga aacaagagat aaaaaactag agacctctta atgcagtcaa     2340

aaatacaaat aaataaaaag tcacttacaa cccaaagtgt gactatcaat ggggtaatca     2400

gtggtgtcaa ataggaggtt aactggggac atctaactgt ttctgcctgg actaatctgc     2460

aagagtgtct gggggaacaa aaagcctctg tgacttagaa agtaggggta ggaggggaaa     2520

aggtcttcta cttggctcag attatttttt tcctctagtc cactaagaat actgcgtttt     2580

aaaatcattt ccttgattca agttcc                                          2606


<210>  14
<211>  606
<212>  DNA
<213>  Homo sapiens

<400>  14
caccctgaag ttctcaggat ccacgtgcag cttgtcacag tgcagctcac tcagtgtggc       60

aaaggtgccc ttgaggttgt ccaggtgagc caggccatca ctaaaggcac cgagcacttt      120

cttgccatga gccttcacct tagggttgcc cataacagca tcaggagtgg acagatcccc      180

aaaggactca aagaacctct gggtccaagg gtagaccacc agcagcctaa gggtgggaaa      240

atagaccaat aggcagagag agtcagtgcc tatcagaaac ccaagagtct tctctgtctc      300

cacatgccca gtttctattg gtctccttaa acctgtcttg taaccttgat accaacctgc      360

ccagggcctc accaccaact tcatccacgt tcaccttgcc ccacagggca gtaacggcag      420

acttctcctc aggagtcaga tgcaccatgg tgtctgtttg aggttgctag tgaacacagt      480

tgtgtcagaa gcaaatgtaa gcaatagatg gctctgccct gacttttatg cccagccctg      540

gctcctgccc tccctgctcc tgggagtaga ttggccaacc ctagggtgtg gctccacagg      600

gtgagg                                                                 606


<210>  15
<211>  303
<212>  PRT
<213>  Ophiostoma novo-ulmi

<400>  15

Ser Ala Tyr Met Ser Arg Arg Glu Ser Ile Asn Pro Trp Ile Leu Thr 
1               5                   10                  15      


Gly Phe Ala Asp Ala Glu Gly Ser Phe Leu Leu Arg Ile Arg Asn Asn 
            20                  25                  30          


Asn Lys Ser Ser Val Gly Tyr Ser Thr Glu Leu Gly Phe Gln Ile Thr 
        35                  40                  45              


Leu His Asn Lys Asp Lys Ser Ile Leu Glu Asn Ile Gln Ser Thr Trp 
    50                  55                  60                  


Lys Val Gly Val Ile Ala Asn Ser Gly Asp Asn Ala Val Ser Leu Lys 
65                  70                  75                  80  


Val Thr Arg Phe Glu Asp Leu Lys Val Ile Ile Asp His Phe Glu Lys 
                85                  90                  95      


Tyr Pro Leu Ile Thr Gln Lys Leu Gly Asp Tyr Met Leu Phe Lys Gln 
            100                 105                 110         


Ala Phe Cys Val Met Glu Asn Lys Glu His Leu Lys Ile Asn Gly Ile 
        115                 120                 125             


Lys Glu Leu Val Arg Ile Lys Ala Lys Leu Asn Trp Gly Leu Thr Asp 
    130                 135                 140                 


Glu Leu Lys Lys Ala Phe Pro Glu Ile Ile Ser Lys Glu Arg Ser Leu 
145                 150                 155                 160 


Ile Asn Lys Asn Ile Pro Asn Phe Lys Trp Leu Ala Gly Phe Thr Ser 
                165                 170                 175     


Gly Glu Gly Cys Phe Phe Val Asn Leu Ile Lys Ser Lys Ser Lys Leu 
            180                 185                 190         


Gly Val Gln Val Gln Leu Val Phe Ser Ile Thr Gln His Ile Lys Asp 
        195                 200                 205             


Lys Asn Leu Met Asn Ser Leu Ile Thr Tyr Leu Gly Cys Gly Tyr Ile 
    210                 215                 220                 


Lys Glu Lys Asn Lys Ser Glu Phe Ser Trp Leu Asp Phe Val Val Thr 
225                 230                 235                 240 


Lys Phe Ser Asp Ile Asn Asp Lys Ile Ile Pro Val Phe Gln Glu Asn 
                245                 250                 255     


Thr Leu Ile Gly Val Lys Leu Glu Asp Phe Glu Asp Trp Cys Lys Val 
            260                 265                 270         


Ala Lys Leu Ile Glu Glu Lys Lys His Leu Thr Glu Ser Gly Leu Asp 
        275                 280                 285             


Glu Ile Lys Lys Ile Lys Leu Asn Met Asn Lys Gly Arg Val Phe 
    290                 295                 300             


<210>  16
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  16
tgggggcccc ttccccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  17
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  17
tgggggcgcc ttccccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  18
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  18
tgggggcccc ttccccacac tatctcaatg caaacatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  19
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  19
tgggggcccc ttccccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgactaata gccttgacaa                 110


<210>  20
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  20
tgggggcccc ttctccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  21
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  21
tgggggcccc ttccccacac tatctcaatg caaacatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  22
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  22
tgggggcccc ttccccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttaaccaata gccttgacaa                 110


<210>  23
<211>  110
<212>  DNA
<213>  Homo sapiens

<400>  23
tgggggcccc ttccccacac tatctcaatg caaatatctg tctgaaacgg tccctggcta       60

aactccaccc atgggttggc cagccttgcc ttgaccaata gccttgacaa                 110


<210>  24
<211>  2508
<212>  DNA
<213>  Homo sapiens

<400>  24
atgtctcgcc gcaagcaagg caaaccccag cacttaagca aacgggaatt ctcgcccgag       60

cctcttgaag ccattcttac agatgatgaa ccagaccacg gcccgttggg agctccagaa      120

ggggatcatg acctcctcac ctgtgggcag tgccagatga acttcccatt gggggacatt      180

cttattttta tcgagcacaa acggaaacaa tgcaatggca gcctctgctt agaaaaagct      240

gtggataagc caccttcccc ttcaccaatc gagatgaaaa aagcatccaa tcccgtggag      300

gttggcatcc aggtcacgcc agaggatgac gattgtttat caacgtcatc tagaggaatt      360

tgccccaaac aggaacacat agcagataaa cttctgcact ggaggggcct ctcctcccct      420

cgttctgcac atggagctct aatccccacg cctgggatga gtgcagaata tgccccgcag      480

ggtatttgta aagatgagcc cagcagctac acatgtacaa cttgcaaaca gccattcacc      540

agtgcatggt ttctcttgca acacgcacag aacactcatg gattaagaat ctacttagaa      600

agcgaacacg gaagtcccct gaccccgcgg gttggtatcc cttcaggact aggtgcagaa      660

tgtccttccc agccacctct ccatgggatt catattgcag acaataaccc ctttaacctg      720

ctaagaatac caggatcagt atcgagagag gcttccggcc tggcagaagg gcgctttcca      780

cccactcccc ccctgtttag tccaccaccg agacatcact tggaccccca ccgcatagag      840

cgcctggggg cggaagagat ggccctggcc acccatcacc cgagtgcctt tgacagggtg      900

ctgcggttga atccaatggc tatggagcct cccgccatgg atttctctag gagacttaga      960

gagctggcag ggaacacgtc tagcccaccg ctgtccccag gccggcccag ccctatgcaa     1020

aggttactgc aaccattcca gccaggtagc aagccgccct tcctggcgac gccccccctc     1080

cctcctctgc aatccgcccc tcctccctcc cagcccccgg tcaagtccaa gtcatgcgag     1140

ttctgcggca agacgttcaa atttcagagc aacctggtgg tgcaccggcg cagccacacg     1200

ggcgagaagc cctacaagtg caacctgtgc gaccacgcgt gcacccaggc cagcaagctg     1260

aagcgccaca tgaagacgca catgcacaaa tcgtccccca tgacggtcaa gtccgacgac     1320

ggtctctcca ccgccagctc cccggaaccc ggcaccagcg acttggtggg cagcgccagc     1380

agcgcgctca agtccgtggt ggccaagttc aagagcgaga acgaccccaa cctgatcccg     1440

gagaacgggg acgaggagga agaggaggac gacgaggaag aggaagaaga ggaggaagag     1500

gaggaggagg agctgacgga gagcgagagg gtggactacg gcttcgggct gagcctggag     1560

gcggcgcgcc accacgagaa cagctcgcgg ggcgcggtcg tgggcgtggg cgacgagagc     1620

cgcgccctgc ccgacgtcat gcagggcatg gtgctcagct ccatgcagca cttcagcgag     1680

gccttccacc aggtcctggg cgagaagcat aagcgcggcc acctggccga ggccgagggc     1740

cacagggaca cttgcgacga agactcggtg gccggcgagt cggaccgcat agacgatggc     1800

actgttaatg gccgcggctg ctccccgggc gagtcggcct cggggggcct gtccaaaaag     1860

ctgctgctgg gcagccccag ctcgctgagc cccttctcta agcgcatcaa gctcgagaag     1920

gagttcgacc tgcccccggc cgcgatgccc aacacggaga acgtgtactc gcagtggctc     1980

gccggctacg cggcctccag gcagctcaaa gatcccttcc ttagcttcgg agactccaga     2040

caatcgcctt ttgcctcctc gtcggagcac tcctcggaga acgggagttt gcgcttctcc     2100

acaccgcccg gggagctgga cggagggatc tcggggcgca gcggcacggg aagtggaggg     2160

agcacgcccc atattagtgg tccgggcccg ggcaggccca gctcaaaaga gggcagacgc     2220

agcgacactt gtgagtactg tgggaaagtc ttcaagaact gtagcaatct cactgtccac     2280

aggagaagcc acacgggcga aaggccttat aaatgcgagc tgtgcaacta tgcctgtgcc     2340

cagagtagca agctcaccag gcacatgaaa acgcatggcc aggtggggaa ggacgtttac     2400

aaatgtgaaa tttgtaagat gccttttagc gtgtacagta ccctggagaa acacatgaaa     2460

aaatggcaca gtgatcgagt gttgaataat gatataaaaa ctgaatag                  2508


<210>  25
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  25
atgggattca tattgcagac aa                                                22


<210>  26
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  26
agccattgga ttcaaccgca gc                                                22


<210>  27
<211>  22
<212>  DNA
<213>  Homo sapiens

<400>  27
caaacagcca ttcaccagtg ca                                                22


<210>  28
<211>  771
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of I-HjeMI, Codon Optimized for Expression in
       E. coli

<400>  28
atgggatccc acatggacct gacctacgct tacctggttg gtctgttcga aggtgacggt       60

tacttctcta tcaccaaaaa gggtaaatac ctgacctacg aactgggtat cgaactgtct      120

atcaaagacg ttcagctgat ctacaaaatc aaagacatcc tgggtgttgg taaagtttct      180

ttccgtaaac gtaacgaaat cgaaatggtt tctctgcgta tccgtgacaa gaatcacctg      240

aaaaacttca tcctgccgat cttcgacaaa tacccgatgc tgtctaacaa gcagtacgac      300

tacctgcgtt tcaaagacgc tctcctgtct aacattatct actctgacga tctgccggaa      360

tacgctcgtt ctaacgaatc tatcaactct gttgactcta ttatcaacac ctcttacttc      420

tctgcttggc tggttggttt catcgaagct gaaggttgct tctctaccta caaactgaac      480

aaagatgacg attacctgat cgcttctttc gacatcgctc agaaagacgg tgacatcctg      540

atctctgcta tccacaaata cctgtctttc accacgaaaa tctacctgga caaaaccaac      600

tgctctcgtc tgaaagtgac cggtgtacgt tctgttaaaa acgtggttaa attcatccag      660

ggtgctccgg ttaaactgct cggtaacaag aaactgcagt acaaactgtg gatcaaacag      720

ctgcgtaaaa tctctcgtta ctctgaaaaa atccagctgc cgtctaacta c               771


<210>  29
<211>  771
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of I-HjeMI, Codon Optimized for Expression in
       Mammals

<400>  29
atgggcagcc acatggacct gacctacgcc tatctggtcg gcctgttcga gggcgacggc       60

tattttagca taaccaagaa gggcaagtat ctgacgtatg aactgggcat cgagctctcc      120

atcaaggacg tgcagctcat ctacaagatc aaggacatcc tcggcgtggg caaagtgtct      180

tttaggaaga ggaacgagat cgagatggtc agcctgcgaa tcagggacaa aaaccacctg      240

aagaacttca tcctgcccat cttcgacaag taccccatgc tgagcaacaa gcagtacgac      300

tatctccgat tcaaggatgc cctcctgtcc aacatcatct atagcgacga cctgcccgag      360

tacgccagga gcaacgagtc aatcaatagc gtggacagca tcatcaacac ctcatacttc      420

agcgcctggc tggttggctt catcgaggcc gagggctgct tcagcaccta caagctcaac      480

aaggacgacg attatttgat cgcgagcttc gatatagccc agaaggacgg cgacattctc      540

atctccgcga tccacaaata cctgagcttc acgaccaaaa tctacctgga caagaccaac      600

tgtagcaggc tcaaggtcac cggcgtgagg agcgtcaaga acgtggttaa gttcatccag      660

ggtgcgccgg tcaagttgct gggtaacaag aagctgcagt acaaactttg gataaagcag      720

ctgcgcaaga tctcccgata cagcgagaaa atccagctgc ccagtaacta c               771


<210>  30
<211>  254
<212>  PRT
<213>  Trichoderma reesei

<400>  30

Met Gly Asp Leu Thr Tyr Ala Tyr Leu Val Gly Leu Phe Glu Gly Asp 
1               5                   10                  15      


Gly Tyr Phe Ser Ile Thr Lys Lys Gly Lys Tyr Leu Thr Tyr Glu Leu 
            20                  25                  30          


Gly Ile Glu Leu Ser Ile Lys Asp Val Gln Leu Ile Tyr Lys Ile Lys 
        35                  40                  45              


Asp Ile Leu Gly Val Gly Lys Val Ser Phe Arg Lys Arg Asn Glu Ile 
    50                  55                  60                  


Glu Met Val Ser Leu Arg Ile Arg Asp Lys Asn His Leu Lys Asn Phe 
65                  70                  75                  80  


Ile Leu Pro Ile Phe Asp Lys Tyr Pro Met Leu Ser Asn Lys Gln Tyr 
                85                  90                  95      


Asp Tyr Leu Arg Phe Lys Asp Ala Leu Leu Ser Asn Ile Ile Tyr Ser 
            100                 105                 110         


Asp Asp Leu Pro Glu Tyr Ala Arg Ser Asn Glu Ser Ile Asn Ser Val 
        115                 120                 125             


Asp Ser Ile Ile Asn Thr Ser Tyr Phe Ser Ala Trp Leu Val Gly Phe 
    130                 135                 140                 


Ile Glu Ala Glu Gly Cys Phe Ser Thr Tyr Lys Leu Asn Lys Asp Asp 
145                 150                 155                 160 


Asp Tyr Leu Ile Ala Ser Phe Asp Ile Ala Gln Lys Asp Gly Asp Ile 
                165                 170                 175     


Leu Ile Ser Ala Ile His Lys Tyr Leu Ser Phe Thr Thr Lys Ile Tyr 
            180                 185                 190         


Leu Asp Lys Thr Asn Cys Ser Arg Leu Lys Val Thr Gly Val Arg Ser 
        195                 200                 205             


Val Lys Asn Val Val Lys Phe Ile Gln Gly Ala Pro Val Lys Leu Leu 
    210                 215                 220                 


Gly Asn Lys Lys Leu Gln Tyr Lys Leu Trp Ile Lys Gln Leu Arg Lys 
225                 230                 235                 240 


Ile Ser Arg Tyr Ser Glu Lys Ile Gln Leu Pro Ser Asn Tyr 
                245                 250                 


<210>  31
<211>  768
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence for a BCL11A Gene Targeting Nuclease Based on
       the Homing Endonuclease I-HjeMI; Codon Optimized for Expression 
       in E. coli and Obtained through Directed Evolution in IVC and in 
       Bacteria

<400>  31
atgggatccc acatggacct gacctacgct tacctggttg gtctgttcga aggtgacggt       60

tacttcacta tcgctaaagc gggtaagtac ctgaattacg agctgggtat cacgctgtct      120

atcaaagacg ctcagctgat ctacaaaatc aaagacatcc tgggtgttgg taatgtttat      180

ttccggaaat ataggcagca tgaaatggtt tctctgcgga tccaggacaa gaatcacctg      240

aaaaacttca tcctgccgat cttcgacaaa tacccgatgc tgtctaacaa gcagtacgac      300

tacctgcgtt tcaaagacgc tctcctgtct aacattatct actctgacga tctgccggaa      360

tacgctcgtt ctaacgaatc tatcaactct gttgactcta ttatcaacac ctcttacttc      420

tctgcttggc tggttggttt catcgaagct gaaggttgct tcacgaccta caaagcgagt      480

aaagataagt acctgacggc tgggttcagt atcgctcaga aagacggtga catcctgatc      540

tctgctatcc acaaatacct gtctttcacc acgaaaccgt acaaggacaa aaccaactgc      600

tctcatctga aggtgaccgg tgtacgttct gttaacaacg tggttaaatt catccagggt      660

gctccggtta aactgctcgg taacaagaaa ctgcagtaca aactgtggat caaacagctg      720

cgtaaaatct ctcgttactc tgaaaaaatc cagctgccgt ctaactac                   768


<210>  32
<211>  768
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of a BCL11A Gene Targeting Nuclease Based on 
       the Homing Endonuclease I-HjeMI; Codon Optimized for Expression 
       in Mammals and Obtained through Directed Evolution in IVC and in 
       Bacteria

<400>  32
atgggcagcc acatggacct gacctacgcc tatctggtcg gcctgttcga gggcgacggc       60

tattttacaa tagctaaggc cggcaagtat ctgaactacg agctgggcat cacactctcc      120

atcaaggacg ctcagctcat ctacaagatc aaggacatcc tcggcgtggg caacgtgtac      180

tttaggaagt acaggcaaca tgagatggtc agcctgcgaa tccaggacaa aaaccacctg      240

aagaacttca tcctgcccat cttcgacaag taccccatgc tgagcaacaa gcagtacgac      300

tacctgcgat tcaaggatgc cctcctgtcc aacatcatct atagcgacga cctgcccgag      360

tacgccagga gcaacgagtc aatcaatagc gtggacagca tcatcaacac ctcatacttc      420

agcgcctggc tggttggctt catcgaggcc gagggctgct tcaccaccta caaggcatcc      480

aaggacaagt acctgacagc gggcttctcc atagcccaga aggacggcga cattctcatc      540

tccgcgatcc acaaatacct gagcttcacg accaaaccct acaaagacaa gaccaactgt      600

agccacctca aggtcaccgg cgtgaggagc gtcaataacg tggttaagtt catccagggt      660

gcgccggtca agctgctggg taacaagaag ctgcagtaca aactttggat aaagcagctg      720

cgcaagatct cccgatacag cgagaaaatc cagctgccca gtaactac                   768


<210>  33
<211>  256
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Amino Acid Sequence of a BCL11A Gene Targeting Nuclease Based on 
       the Homing Endonuclease I-HjeMI

<400>  33

Met Gly Ser His Met Asp Leu Thr Tyr Ala Tyr Leu Val Gly Leu Phe 
1               5                   10                  15      


Glu Gly Asp Gly Tyr Phe Thr Ile Ala Lys Ala Gly Lys Tyr Leu Asn 
            20                  25                  30          


Tyr Glu Leu Gly Ile Thr Leu Ser Ile Lys Asp Ala Gln Leu Ile Tyr 
        35                  40                  45              


Lys Ile Lys Asp Ile Leu Gly Val Gly Asn Val Tyr Phe Arg Lys Tyr 
    50                  55                  60                  


Arg Gln His Glu Met Val Ser Leu Arg Ile Gln Asp Lys Asn His Leu 
65                  70                  75                  80  


Lys Asn Phe Ile Leu Pro Ile Phe Asp Lys Tyr Pro Met Leu Ser Asn 
                85                  90                  95      


Lys Gln Tyr Asp Tyr Leu Arg Phe Lys Asp Ala Leu Leu Ser Asn Ile 
            100                 105                 110         


Ile Tyr Ser Asp Asp Leu Pro Glu Tyr Ala Arg Ser Asn Glu Ser Ile 
        115                 120                 125             


Asn Ser Val Asp Ser Ile Ile Asn Thr Ser Tyr Phe Ser Ala Trp Leu 
    130                 135                 140                 


Val Gly Phe Ile Glu Ala Glu Gly Cys Phe Thr Thr Tyr Lys Ala Ser 
145                 150                 155                 160 


Lys Asp Lys Tyr Leu Thr Ala Gly Phe Ser Ile Ala Gln Lys Asp Gly 
                165                 170                 175     


Asp Ile Leu Ile Ser Ala Ile His Lys Tyr Leu Ser Phe Thr Thr Lys 
            180                 185                 190         


Pro Tyr Lys Asp Lys Thr Asn Cys Ser His Leu Lys Val Thr Gly Val 
        195                 200                 205             


Arg Ser Val Asn Asn Val Val Lys Phe Ile Gln Gly Ala Pro Val Lys 
    210                 215                 220                 


Leu Leu Gly Asn Lys Lys Leu Gln Tyr Lys Leu Trp Ile Lys Gln Leu 
225                 230                 235                 240 


Arg Lys Ile Ser Arg Tyr Ser Glu Lys Ile Gln Leu Pro Ser Asn Tyr 
                245                 250                 255     


<210>  34
<211>  912
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of I-OnuI, Codon Optimized for Expression in 
       E. coli

<400>  34
atgtccgcct acatgtcccg tcgcgagtcc attaacccgt ggattctcac cggtttcgcc       60

gacgcggaag gctccttttt gctgcgcatc cgcaacaaca acaagtccag cgtcggctac      120

tccactgagc tcggcttcca aattacactt cataacaagg acaagagcat tcttgagaac      180

atccagtcaa catggaaggt gggcgtgatc gccaacagcg gtgacaacgc cgtgtcgctg      240

aaggtcacgc gttttgagga cctgaaggtc attatcgacc attttgaaaa atacccactg      300

attacgcaga agctcggtga ctacatgctg tttaagcagg cgttttgcgt catggagaac      360

aaggagcatt tgaagattaa tggtatcaag gagctggtgc gcattaaggc aaagctcaat      420

tggggtctga cggatgagct gaagaaggcc tttccggaga tcatctcgaa ggagcgctcc      480

ctcatcaaca agaacatccc taatttcaag tggctggcgg gttttacctc gggcgagggt      540

tgcttctttg ttaacctgat caagtcaaag tcgaagctag gtgtccaggt gcagctggtg      600

ttcagcatta cccaacacat caaggataag aacctcatga actctctgat tacctacttg      660

ggctgcggct acattaagga gaaaaacaag agtgagttct cctggcttga cttcgtcgtc      720

acgaaattct ccgacatcaa cgacaagatc attccggtct ttcaggaaaa cacgctcatc      780

ggcgtgaagc tcgaggactt cgaggattgg tgtaaggtcg ctaagctgat cgaggagaaa      840

aagcacctga cagaaagtgg cctggacgag atcaagaaga ttaagctgaa catgaacaag      900

ggcagagtat tc                                                          912


<210>  35
<211>  1998
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of MegaTAL:5.5 RVD + Y2 I-AniI

<400>  35
gtggatctac gcacgctcgg ctacagtcag cagcagcaag agaagatcaa accgaaggtg       60

cgttcgacag tggcgcagca ccacgaggca ctggtgggcc atgggtttac acacgcgcac      120

atcgttgcgc tcagccaaca cccggcagcg ttagggaccg tcgctgtcac gtatcagcac      180

ataatcacgg cgttgccaga ggcgacacac gaagacatcg ttggcgtcgg caaacagtgg      240

tccggcgcac gcgccctgga ggccttgctc acggatgcgg gggagttgag aggtccgccg      300

ttacagttgg acacaggcca acttgtgaag attgcaaaac gtggcggcgt gaccgcaatg      360

gaggcagtgc atgcatcgcg caatgcactg acgggtgccc ccctgaacct gaccccggac      420

caagtggtgg ctatcgccag caacaatggc ggcaagcaag cgctcgaaac ggtgcagcgg      480

ctgttgccgg tgctgtgcca ggaccatggc ctgactccgg accaagtggt ggctatcgcc      540

agccacgatg gcggcaagca agcgctcgaa acggtgcagc ggctgttgcc ggtgctgtgc      600

caggaccatg gcctgacccc ggaccaagtg gtggctatcg ccagcaacat tggcggcaag      660

caagcgctcg aaacggtgca gcggctgttg ccggtgctgt gccaggacca tggcctgacc      720

ccggaccaag tggtggctat cgccagcaac aatggcggca agcaagcgct cgaaacggtg      780

cagcggctgt tgccggtgct gtgccaggac catggcctga ctccggacca agtggtggct      840

atcgccagcc acgatggcgg caagcaagcg ctcgaaacgg tgcagcggct gttgccggtg      900

ctgtgccagg accatggcct gaccccggac caagtggtgg ctatcgccag caacggtggc      960

ggcaagcaag cgctcgaaag cattgtggcc cagctgagcc ggcctgatcc ggcgttggcc     1020

gcgttgacca acgaccacct cgtcgccttg gcctgcctcg gcggacgtcc tgccatggat     1080

gcagtgaaaa agggattgcc gcacgcgccg gaattgatca gaagagtcaa tcgccgtatt     1140

ggcgaacgca cgtcccatcg cgttgcgata tctagagtgg gaggaagcga tcttacgtac     1200

gcgtatttag ttggtctcta cgaaggggat ggatacttta gtatcaccaa gaaaggcaag     1260

tacttgactt atgaattagg tattgagctg agcatcaaag acgtccaatt gatttacaag     1320

atcaagaaaa tcctaggtat tggcatcgta agcttcagga agagaaacga gattgaaatg     1380

gttgcattga ggatccgtga taagaatcat ctaaaatcta agatattgcc tatatttgag     1440

aagtatccaa tgttttccaa caaacagtac gactatttaa gattcaggaa tgcattgtta     1500

tctggcatta tatacctaga agacttgcct gattacacta gaagtgacga accattgaat     1560

tctatagaat ccattatcaa cacatcatac ttctccgcct ggctagttgg atttatagaa     1620

gctgagggct gtttcagtgt gtacaagctg aacaaagacg atgactactt gattgcttca     1680

ttcgacattg cccaaagaga tggtgatatc ttgatttcag caattaggaa gtacttaagt     1740

ttcactacta aggtttacct agacaagact aattgtagca aattgaaggt cactagtgtt     1800

agatccgtcg agaacatcat taagtttctg cagaatgctc ctgtcaaatt gttaggcaac     1860

aagaaactgc aatacaagtt gtggttgaaa caactaagga agatttctag gtattccgag     1920

aagatcaaga ttccatcaaa ctacgtcgac cgagcatctt accgccattt atacccatat     1980

ttgttctgtt tttcttga                                                   1998


<210>  36
<211>  665
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Amino Acid Sequence of MegaTAL:5.5 RVD + Y2 I-AniI

<400>  36

Val Asp Leu Arg Thr Leu Gly Tyr Ser Gln Gln Gln Gln Glu Lys Ile 
1               5                   10                  15      


Lys Pro Lys Val Arg Ser Thr Val Ala Gln His His Glu Ala Leu Val 
            20                  25                  30          


Gly His Gly Phe Thr His Ala His Ile Val Ala Leu Ser Gln His Pro 
        35                  40                  45              


Ala Ala Leu Gly Thr Val Ala Val Thr Tyr Gln His Ile Ile Thr Ala 
    50                  55                  60                  


Leu Pro Glu Ala Thr His Glu Asp Ile Val Gly Val Gly Lys Gln Trp 
65                  70                  75                  80  


Ser Gly Ala Arg Ala Leu Glu Ala Leu Leu Thr Asp Ala Gly Glu Leu 
                85                  90                  95      


Arg Gly Pro Pro Leu Gln Leu Asp Thr Gly Gln Leu Val Lys Ile Ala 
            100                 105                 110         


Lys Arg Gly Gly Val Thr Ala Met Glu Ala Val His Ala Ser Arg Asn 
        115                 120                 125             


Ala Leu Thr Gly Ala Pro Leu Asn Leu Thr Pro Asp Gln Val Val Ala 
    130                 135                 140                 


Ile Ala Ser Asn Asn Gly Gly Lys Gln Ala Leu Glu Thr Val Gln Arg 
145                 150                 155                 160 


Leu Leu Pro Val Leu Cys Gln Asp His Gly Leu Thr Pro Asp Gln Val 
                165                 170                 175     


Val Ala Ile Ala Ser His Asp Gly Gly Lys Gln Ala Leu Glu Thr Val 
            180                 185                 190         


Gln Arg Leu Leu Pro Val Leu Cys Gln Asp His Gly Leu Thr Pro Asp 
        195                 200                 205             


Gln Val Val Ala Ile Ala Ser Asn Ile Gly Gly Lys Gln Ala Leu Glu 
    210                 215                 220                 


Thr Val Gln Arg Leu Leu Pro Val Leu Cys Gln Asp His Gly Leu Thr 
225                 230                 235                 240 


Pro Asp Gln Val Val Ala Ile Ala Ser Asn Asn Gly Gly Lys Gln Ala 
                245                 250                 255     


Leu Glu Thr Val Gln Arg Leu Leu Pro Val Leu Cys Gln Asp His Gly 
            260                 265                 270         


Leu Thr Pro Asp Gln Val Val Ala Ile Ala Ser His Asp Gly Gly Lys 
        275                 280                 285             


Gln Ala Leu Glu Thr Val Gln Arg Leu Leu Pro Val Leu Cys Gln Asp 
    290                 295                 300                 


His Gly Leu Thr Pro Asp Gln Val Val Ala Ile Ala Ser Asn Gly Gly 
305                 310                 315                 320 


Gly Lys Gln Ala Leu Glu Ser Ile Val Ala Gln Leu Ser Arg Pro Asp 
                325                 330                 335     


Pro Ala Leu Ala Ala Leu Thr Asn Asp His Leu Val Ala Leu Ala Cys 
            340                 345                 350         


Leu Gly Gly Arg Pro Ala Met Asp Ala Val Lys Lys Gly Leu Pro His 
        355                 360                 365             


Ala Pro Glu Leu Ile Arg Arg Val Asn Arg Arg Ile Gly Glu Arg Thr 
    370                 375                 380                 


Ser His Arg Val Ala Ile Ser Arg Val Gly Gly Ser Asp Leu Thr Tyr 
385                 390                 395                 400 


Ala Tyr Leu Val Gly Leu Tyr Glu Gly Asp Gly Tyr Phe Ser Ile Thr 
                405                 410                 415     


Lys Lys Gly Lys Tyr Leu Thr Tyr Glu Leu Gly Ile Glu Leu Ser Ile 
            420                 425                 430         


Lys Asp Val Gln Leu Ile Tyr Lys Ile Lys Lys Ile Leu Gly Ile Gly 
        435                 440                 445             


Ile Val Ser Phe Arg Lys Arg Asn Glu Ile Glu Met Val Ala Leu Arg 
    450                 455                 460                 


Ile Arg Asp Lys Asn His Leu Lys Ser Lys Ile Leu Pro Ile Phe Glu 
465                 470                 475                 480 


Lys Tyr Pro Met Phe Ser Asn Lys Gln Tyr Asp Tyr Leu Arg Phe Arg 
                485                 490                 495     


Asn Ala Leu Leu Ser Gly Ile Ile Tyr Leu Glu Asp Leu Pro Asp Tyr 
            500                 505                 510         


Thr Arg Ser Asp Glu Pro Leu Asn Ser Ile Glu Ser Ile Ile Asn Thr 
        515                 520                 525             


Ser Tyr Phe Ser Ala Trp Leu Val Gly Phe Ile Glu Ala Glu Gly Cys 
    530                 535                 540                 


Phe Ser Val Tyr Lys Leu Asn Lys Asp Asp Asp Tyr Leu Ile Ala Ser 
545                 550                 555                 560 


Phe Asp Ile Ala Gln Arg Asp Gly Asp Ile Leu Ile Ser Ala Ile Arg 
                565                 570                 575     


Lys Tyr Leu Ser Phe Thr Thr Lys Val Tyr Leu Asp Lys Thr Asn Cys 
            580                 585                 590         


Ser Lys Leu Lys Val Thr Ser Val Arg Ser Val Glu Asn Ile Ile Lys 
        595                 600                 605             


Phe Leu Gln Asn Ala Pro Val Lys Leu Leu Gly Asn Lys Lys Leu Gln 
    610                 615                 620                 


Tyr Lys Leu Trp Leu Lys Gln Leu Arg Lys Ile Ser Arg Tyr Ser Glu 
625                 630                 635                 640 


Lys Ile Lys Ile Pro Ser Asn Tyr Val Asp Arg Ala Ser Tyr Arg His 
                645                 650                 655     


Leu Tyr Pro Tyr Leu Phe Cys Phe Ser 
            660                 665 


<210>  37
<211>  4146
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered type II CRISPR system for human cells; expression 
       format and full sequence of cas9 gene insert of Mali et al.

<400>  37
gccaccatgg acaagaagta ctccattggg ctcgatatcg gcacaaacag cgtcggctgg       60

gccgtcatta cggacgagta caaggtgccg agcaaaaaat tcaaagttct gggcaatacc      120

gatcgccaca gcataaagaa gaacctcatt ggcgccctcc tgttcgactc cggggagacg      180

gccgaagcca cgcggctcaa aagaacagca cggcgcagat atacccgcag aaagaatcgg      240

atctgctacc tgcaggagat ctttagtaat gagatggcta aggtggatga ctctttcttc      300

cataggctgg aggagtcctt tttggtggag gaggataaaa agcacgagcg ccacccaatc      360

tttggcaata tcgtggacga ggtggcgtac catgaaaagt acccaaccat atatcatctg      420

aggaagaagc ttgtagacag tactgataag gctgacttgc ggttgatcta tctcgcgctg      480

gcgcatatga tcaaatttcg gggacacttc ctcatcgagg gggacctgaa cccagacaac      540

agcgatgtcg acaaactctt tatccaactg gttcagactt acaatcagct tttcgaagag      600

aacccgatca acgcatccgg agttgacgcc aaagcaatcc tgagcgctag gctgtccaaa      660

tcccggcggc tcgaaaacct catcgcacag ctccctgggg agaagaagaa cggcctgttt      720

ggtaatctta tcgccctgtc actcgggctg acccccaact ttaaatctaa cttcgacctg      780

gccgaagatg ccaagcttca actgagcaaa gacacctacg atgatgatct cgacaatctg      840

ctggcccaga tcggcgacca gtacgcagac ctttttttgg cggcaaagaa cctgtcagac      900

gccattctgc tgagtgatat tctgcgagtg aacacggaga tcaccaaagc tccgctgagc      960

gctagtatga tcaagcgcta tgatgagcac caccaagact tgactttgct gaaggccctt     1020

gtcagacagc aactgcctga gaagtacaag gaaattttct tcgatcagtc taaaaatggc     1080

tacgccggat acattgacgg cggagcaagc caggaggaat tttacaaatt tattaagccc     1140

atcttggaaa aaatggacgg caccgaggag ctgctggtaa agcttaacag agaagatctg     1200

ttgcgcaaac agcgcacttt cgacaatgga agcatccccc accagattca cctgggcgaa     1260

ctgcacgcta tcctcaggcg gcaagaggat ttctacccct ttttgaaaga taacagggaa     1320

aagattgaga aaatcctcac atttcggata ccctactatg taggccccct cgcccgggga     1380

aattccagat tcgcgtggat gactcgcaaa tcagaagaga ccatcactcc ctggaacttc     1440

gaggaagtcg tggataaggg ggcctctgcc cagtccttca tcgaaaggat gactaacttt     1500

gataaaaatc tgcctaacga aaaggtgctt cctaaacact ctctgctgta cgagtacttc     1560

acagtttata acgagctcac caaggtcaaa tacgtcacag aagggatgag aaagccagca     1620

ttcctgtctg gagagcagaa gaaagctatc gtggacctcc tcttcaagac gaaccggaaa     1680

gttaccgtga aacagctcaa agaagactat ttcaaaaaga ttgaatgttt cgactctgtt     1740

gaaatcagcg gagtggagga tcgcttcaac gcatccctgg gaacgtatca cgatctcctg     1800

aaaatcatta aagacaagga cttcctggac aatgaggaga acgaggacat tcttgaggac     1860

attgtcctca cccttacgtt gtttgaagat agggagatga ttgaagaacg cttgaaaact     1920

tacgctcatc tcttcgacga caaagtcatg aaacagctca agaggcgccg atatacagga     1980

tgggggcggc tgtcaagaaa actgatcaat gggatccgag acaagcagag tggaaagaca     2040

atcctggatt ttcttaagtc cgatggattt gccaaccgga acttcatgca gttgatccat     2100

gatgactctc tcacctttaa ggaggacatc cagaaagcac aagtttctgg ccagggggac     2160

agtcttcacg agcacatcgc taatcttgca ggtagcccag ctatcaaaaa gggaatactg     2220

cagaccgtta aggtcgtgga tgaactcgtc aaagtaatgg gaaggcataa gcccgagaat     2280

atcgttatcg agatggcccg agagaaccaa actacccaga agggacagaa gaacagtagg     2340

gaaaggatga agaggattga agagggtata aaagaactgg ggtcccaaat ccttaaggaa     2400

cacccagttg aaaacaccca gcttcagaat gagaagctct acctgtacta cctgcagaac     2460

ggcagggaca tgtacgtgga tcaggaactg gacatcaatc ggctctccga ctacgacgtg     2520

gatcatatcg tgccccagtc ttttctcaaa gatgattcta ttgataataa agtgttgaca     2580

agatccgata aaaatagagg gaagagtgat aacgtcccct cagaagaagt tgtcaagaaa     2640

atgaaaaatt attggcggca gctgctgaac gccaaactga tcacacaacg gaagttcgat     2700

aatctgacta aggctgaacg aggtggcctg tctgagttgg ataaagccgg cttcatcaaa     2760

aggcagcttg ttgagacacg ccagatcacc aagcacgtgg cccaaattct cgattcacgc     2820

atgaacacca agtacgatga aaatgacaaa ctgattcgag aggtgaaagt tattactctg     2880

aagtctaagc tggtctcaga tttcagaaag gactttcagt tttataaggt gagagagatc     2940

aacaattacc accatgcgca tgatgcctac ctgaatgcag tggtaggcac tgcacttatc     3000

aaaaaatatc ccaagcttga atctgaattt gtttacggag actataaagt gtacgatgtt     3060

aggaaaatga tcgcaaagtc tgagcaggaa ataggcaagg ccaccgctaa gtacttcttt     3120

tacagcaata ttatgaattt tttcaagacc gagattacac tggccaatgg agagattcgg     3180

aagcgaccac ttatcgaaac aaacggagaa acaggagaaa tcgtgtggga caagggtagg     3240

gatttcgcga cagtccggaa ggtcctgtcc atgccgcagg tgaacatcgt taaaaagacc     3300

gaagtacaga ccggaggctt ctccaaggaa agtatcctcc cgaaaaggaa cagcgacaag     3360

ctgatcgcac gcaaaaaaga ttgggacccc aagaaatacg gcggattcga ttctcctaca     3420

gtcgcttaca gtgtactggt tgtggccaaa gtggagaaag ggaagtctaa aaaactcaaa     3480

agcgtcaagg aactgctggg catcacaatc atggagcgat caagcttcga aaaaaacccc     3540

atcgactttc tcgaggcgaa aggatataaa gaggtcaaaa aagacctcat cattaagctt     3600

cccaagtact ctctctttga gcttgaaaac ggccggaaac gaatgctcgc tagtgcgggc     3660

gagctgcaga aaggtaacga gctggcactg ccctctaaat acgttaattt cttgtatctg     3720

gccagccact atgaaaagct caaagggtct cccgaagata atgagcagaa gcagctgttc     3780

gtggaacaac acaaacacta ccttgatgag atcatcgagc aaataagcga attctccaaa     3840

agagtgatcc tcgccgacgc taacctcgat aaggtgcttt ctgcttacaa taagcacagg     3900

gataagccca tcagggagca ggcagaaaac attatccact tgtttactct gaccaacttg     3960

ggcgcgcctg cagccttcaa gtacttcgac accaccatag acagaaagcg gtacacctct     4020

acaaaggagg tcctggacgc cacactgatt catcagtcaa ttacggggct ctatgaaaca     4080

agaatcgacc tctctcagct cggtggagac agcagggctg accccaagaa gaagaggaag     4140

gtgtga                                                                4146


<210>  38
<211>  455
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of RNA Guide Strand for use with Cas9 
       Endonuclease


<220>
<221>  misc_feature
<222>  (320)..(338)
<223>  n is a, c, g, or t

<400>  38
tgtacaaaaa agcaggcttt aaaggaacca attcagtcga ctggatccgg taccaaggtc       60

gggcaggaag agggcctatt tcccatgatt ccttcatatt tgcatatacg atacaaggct      120

gttagagaga taattagaat taatttgact gtaaacacaa agatattagt acaaaatacg      180

tgacgtagaa agtaataatt tcttgggtag tttgcagttt taaaattatg ttttaaaatg      240

gactatcata tgcttaccgt aacttgaaag tatttcgatt tcttggcttt atatatcttg      300

tggaaaggac gaaacaccgn nnnnnnnnnn nnnnnnnngt tttagagcta gaaatagcaa      360

gttaaaataa ggctagtccg ttatcaactt gaaaaagtgg caccgagtcg gtgctttttt      420

tctagaccca gctttcttgt acaaagttgg catta                                 455


<210>  39
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  BCL11A gene target site

<400>  39
tccaagtgat gtctcggtgg tg                                                22


<210>  40
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Targeted Homing Endonuclease Target Site

<400>  40
tttccaatta ttcaaccttt ta                                                22


<210>  41
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Targeted Homing Endonuclease Target Site

<400>  41
tcttgaatta ttcaaccttt ta                                                22


<210>  42
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Targeted Homing Endonuclease Target Site

<400>  42
tttccattta ttcaatattt ta                                                22


<210>  43
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Targeted Homing Endonuclease Target Site

<400>  43
tttccattta ttcaatatct tt                                                22


<210>  44
<211>  12
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  U6 Promoter Sequence of a Generic Cas9 Guide RNA

<400>  44
ggacgaaaca cc                                                           12


<210>  45
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Generic Target-specific Sequence of a Generic Cas9 Guide RNA


<220>
<221>  misc_feature
<222>  (2)..(20)
<223>  n is a, c, g, or t

<400>  45
gnnnnnnnnn nnnnnnnnnn                                                   20


<210>  46
<211>  77
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Guide RNA Scaffold Sequence of a Generic Cas9 Guide RNA

<400>  46
gttttagagc tagaaatagc aagttaaaat aaggctagtc cgttatcaac ttgaaaaagt       60

ggcaccgagt cggtgct                                                      77


<210>  47
<211>  10
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Poly T Tail of a Generic Cas9 Guide RNA


<220>
<221>  misc_feature
<222>  (1)..(4)
<223>  n is a, c, g, or t

<400>  47
nnnntttttt                                                              10


<210>  48
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-B

<400>  48
gccatttcta ttatcagact tgg                                               23


<210>  49
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-C

<400>  49
gctgggcttc tgttgcagta ggg                                               23


<210>  50
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-D

<400>  50
gaaaatggga gacaaatagc tgg                                               23


<210>  51
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-E

<400>  51
gaataattca agaaaggtgg tgg                                               23


<210>  52
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-F

<400>  52
gatattgaat aattcaagaa agg                                               23


<210>  53
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-G

<400>  53
gcctgagatt ctgatcacaa ggg                                               23


<210>  54
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Target-specific Sequence for Cas9 Guide RNA GGN20GG-H

<400>  54
ggtaaattct taaggccatg agg                                               23


<210>  55
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  55
ttgaggagat gtctctgtta at                                                22


<210>  56
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  56
ttgaggtgat gtctctgtta at                                                22


<210>  57
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  57
ttgaagtgat gtctctgtta at                                                22


<210>  58
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  58
tccaagtgat gtctctgtta at                                                22


<210>  59
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  59
tccaagtgat gtctctgtta at                                                22


<210>  60
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  60
ttgaggaggt ttctgtgtta at                                                22


<210>  61
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Engineered target site

<400>  61
ttgaggaggt ttctcggtgg tg                                                22


<210>  62
<211>  900
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide Sequence of a I-CpaMI homing endonuclease; ORF, codon 
       optimized for mammalian expression

<400>  62
atgaacacca gctctagctt caatccctgg ttcctgaccg gctttagcga tgcagagtgc       60

tctttcagca tcctgataca ggccaacagc aagtactcca ccggttggag gatcaagccc      120

gtgttcgcca tcggcttgca caagaaggac ctggagcttc tgaagagaat ccagagctat      180

ctgggcgtgg gcaagataca cattcacggc aaagacagca ttcagttcag gattgacagc      240

cccaaggagc tggaggtgat catcaaccac tttgagaact accccctggt aaccgccaag      300

tgggccgact acaccctctt taagaaggcc ctggacgtaa ttctgttgaa ggagcacctg      360

agccagaagg gcctgcttaa actggtaggc attaaggcga gcctgaatct cgggttgaac      420

ggcagcctca aggaggcgtt cccgaactgg gaagaactgc agatcgacag gccgagctac      480

gtgttcaagg gcatccccga ccccaactgg atcagcggct tcgcgtcagg cgatagcagc      540

tttaatgtga aaatcagcaa ctcccccacg tcactgctca ataaaagggt gcagctgagg      600

ttcggcatcg gactgaacat cagagagaaa gcccttatcc aatacctggt ggcctacttt      660

gacctgtcag acaacctgaa gaacatctac ttcgacctga acagcgcacg gttcgaggtg      720

gtgaagttca gcgacatcac cgacaagatc atccccttct tcgacaagta cagcatacaa      780

ggcaagaaga gcctggacta catcaacttc aaggaagtgg ccgacattat caagagcaag      840

aaccatctta ctagcgaggg cttccaggaa atcttggaca tcaaagccag tatgaacaag      900


<210>  63
<211>  300
<212>  PRT
<213>  Cryphonectria parasitica

<400>  63

Met Asn Thr Ser Ser Ser Phe Asn Pro Trp Phe Leu Thr Gly Phe Ser 
1               5                   10                  15      


Asp Ala Glu Cys Ser Phe Ser Ile Leu Ile Gln Ala Asn Ser Lys Tyr 
            20                  25                  30          


Ser Thr Gly Trp Arg Ile Lys Pro Val Phe Ala Ile Gly Leu His Lys 
        35                  40                  45              


Lys Asp Leu Glu Leu Leu Lys Arg Ile Gln Ser Tyr Leu Gly Val Gly 
    50                  55                  60                  


Lys Ile His Ile His Gly Lys Asp Ser Ile Gln Phe Arg Ile Asp Ser 
65                  70                  75                  80  


Pro Lys Glu Leu Glu Val Ile Ile Asn His Phe Glu Asn Tyr Pro Leu 
                85                  90                  95      


Val Thr Ala Lys Trp Ala Asp Tyr Thr Leu Phe Lys Lys Ala Leu Asp 
            100                 105                 110         


Val Ile Leu Leu Lys Glu His Leu Ser Gln Lys Gly Leu Leu Lys Leu 
        115                 120                 125             


Val Gly Ile Lys Ala Ser Leu Asn Leu Gly Leu Asn Gly Ser Leu Lys 
    130                 135                 140                 


Glu Ala Phe Pro Asn Trp Glu Glu Leu Gln Ile Asp Arg Pro Ser Tyr 
145                 150                 155                 160 


Val Phe Lys Gly Ile Pro Asp Pro Asn Trp Ile Ser Gly Phe Ala Ser 
                165                 170                 175     


Gly Asp Ser Ser Phe Asn Val Lys Ile Ser Asn Ser Pro Thr Ser Leu 
            180                 185                 190         


Leu Asn Lys Arg Val Gln Leu Arg Phe Gly Ile Gly Leu Asn Ile Arg 
        195                 200                 205             


Glu Lys Ala Leu Ile Gln Tyr Leu Val Ala Tyr Phe Asp Leu Ser Asp 
    210                 215                 220                 


Asn Leu Lys Asn Ile Tyr Phe Asp Leu Asn Ser Ala Arg Phe Glu Val 
225                 230                 235                 240 


Val Lys Phe Ser Asp Ile Thr Asp Lys Ile Ile Pro Phe Phe Asp Lys 
                245                 250                 255     


Tyr Ser Ile Gln Gly Lys Lys Ser Leu Asp Tyr Ile Asn Phe Lys Glu 
            260                 265                 270         


Val Ala Asp Ile Ile Lys Ser Lys Asn His Leu Thr Ser Glu Gly Phe 
        275                 280                 285             


Gln Glu Ile Leu Asp Ile Lys Ala Ser Met Asn Lys 
    290                 295                 300 


<210>  64
<211>  5264
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide sequence of BCL11A gene targeting nuclease-encoding 
       plasmid pExodusBCL11Ahje

<400>  64
gacggatcgg gagatctccc gatcccctat ggtgcactct cagtacaatc tgctctgatg       60

ccgcatagtt aagccagtat ctgctccctg cttgtgtgtt ggaggtcgct gagtagtgcg      120

cgagcaaaat ttaagctaca acaaggcaag gcttgaccga caattgcatg aagaatctgc      180

ttagggttag gcgttttgcg ctgcttcgcg atgtacgggc cagatatacg cgttgacatt      240

gattattgac tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata      300

tggagttccg cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc      360

cccgcccatt gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc      420

attgacgtca atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt      480

atcatatgcc aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt      540

atgcccagta catgacctta tgggactttc ctacttggca gtacatctac gtattagtca      600

tcgctattac catggtgatg cggttttggc agtacatcaa tgggcgtgga tagcggtttg      660

actcacgggg atttccaagt ctccacccca ttgacgtcaa tgggagtttg ttttggcacc      720

aaaatcaacg ggactttcca aaatgtcgta acaactccgc cccattgacg caaatgggcg      780

gtaggcgtgt acggtgggag gtctatataa gcagagctct ctggctaact agagaaccca      840

ctgcttactg gcttatcgaa attaatacga ctcactatag ggagacccaa gctggctagc      900

gtttaaactt aagcttggta ccgagctcgg atccactagt ccagtgtggt ggaattctgc      960

aggtacgttg acgccgccac catgggatat ccatacgatg tcccagatta tgcgccacct     1020

aagaagaaac gcaaagtccc cgggggcagc cacatggacc tgacctacgc ctatctggtc     1080

ggcctgttcg agggcgacgg ctattttaca atagctaagg ccggcaagta tctgaactac     1140

gagctgggca tcacactctc catcaaggac gctcagctca tctacaagat caaggacatc     1200

ctcggcgtgg gcaacgtgta ctttaggaag tacaggcaac atgagatggt cagcctgcga     1260

atccaggaca aaaaccacct gaagaacttc atcctgccca tcttcgacaa gtaccccatg     1320

ctgagcaaca agcagtacga ctacctgcga ttcaaggatg ccctcctgtc caacatcatc     1380

tatagcgacg acctgcccga gtacgccagg agcaacgagt caatcaatag cgtggacagc     1440

atcatcaaca cctcatactt cagcgcctgg ctggttggct tcatcgaggc cgagggctgc     1500

ttcaccacct acaaggcatc caaggacaag tacctgacag cgggcttctc catagcccag     1560

aaggacggcg acattctcat ctccgcgatc cacaaatacc tgagcttcac gaccaaaccc     1620

tacaaagaca agaccaactg tagccacctc aaggtcaccg gcgtgaggag cgtcaataac     1680

gtggttaagt tcatccaggg tgcgccggtc aagctgctgg gtaacaagaa gctgcagtac     1740

aaactttgga taaagcagct gcgcaagatc tcccgataca gcgagaaaat ccagctgccc     1800

agtaactact aatctagagg gcccgtttaa acccgctgat cagcctcgac tgtgccttct     1860

agttgccagc catctgttgt ttgcccctcc cccgtgcctt ccttgaccct ggaaggtgcc     1920

actcccactg tcctttccta ataaaatgag gaaattgcat cgcattgtct gagtaggtgt     1980

cattctattc tggggggtgg ggtggggcag gacagcaagg gggaggattg ggaagacaat     2040

agcaggcatg ctggggatgc ggtgggctct atggcttctg aggcggaaag aaccagctgg     2100

ggctctaggg ggtatcccca cgcgccctgt agcggcgcat taagcgcggc gggtgtggtg     2160

gttacgcgca gcgtgaccgc tacacttgcc agcgccctag cgcccgctcc tttcgctttc     2220

ttcccttcct ttctcgccac gttcgccggc tttccccgtc aagctctaaa tcgggggctc     2280

cctttagggt tccgatttag tgctttacgg cacctcgacc ccaaaaaact tgattagggt     2340

gatggttcac gtagtgggcc atcgccctga tagacggttt ttcgcccttt gacgttggag     2400

tccacgttct ttaatagtgg actcttgttc caaactggaa caacactcaa ccctatctcg     2460

gtctattctt ttgatttata agggattttg ccgatttcgg cctattggtt aaaaaatgag     2520

ctgatttaac aaaaatttaa cgcgaattaa ttctgtggaa tgtgtgtcag ttagggtgtg     2580

gaaagtcccc aggctcccca gcaggcagaa gtatgcaaag cctatcagga catagcgttg     2640

gctacccgtg atattgctga agagcttggc ggcgaatggg ctgaccgctt cctcgtgctt     2700

tacggtatcg ccgctcccga ttcgcagcgc atcgccttct atcgccttct tgacgagttc     2760

ttctgagcgg gactctgggg ttcgaaatga ccgaccaagc gacgcccaac ctgccatcac     2820

gagatttcga ttccaccgcc gccttctatg aaaggttggg cttcggaatc gttttccggg     2880

acgccggctg gatgatcctc cagcgcgggg atctcatgct ggagttcttc gcccacccca     2940

acttgtttat tgcagcttat aatggttaca aataaagcaa tagcatcaca aatttcacaa     3000

ataaagcatt tttttcactg cattctagtt gtggtttgtc caaactcatc aatgtatctt     3060

atcatgtctg tataccgtcg acctctagct agagcttggc gtaatcatgg tcatagctgt     3120

ttcctgtgtg aaattgttat ccgctcacaa ttccacacaa catacgagcc ggaagcataa     3180

agtgtaaagc ctggggtgcc taatgagtga gctaactcac attaattgcg ttgcgctcac     3240

tgcccgcttt ccagtcggga aacctgtcgt gccagctgca ttaatgaatc ggccaacgcg     3300

cggggagagg cggtttgcgt attgggcgct cttccgcttc ctcgctcact gactcgctgc     3360

gctcggtcgt tcggctgcgg cgagcggtat cagctcactc aaaggcggta atacggttat     3420

ccacagaatc aggggataac gcaggaaaga acatgtgagc aaaaggccag caaaaggcca     3480

ggaaccgtaa aaaggccgcg ttgctggcgt ttttccatag gctccgcccc cctgacgagc     3540

atcacaaaaa tcgacgctca agtcagaggt ggcgaaaccc gacaggacta taaagatacc     3600

aggcgtttcc ccctggaagc tccctcgtgc gctctcctgt tccgaccctg ccgcttaccg     3660

gatacctgtc cgcctttctc ccttcgggaa gcgtggcgct ttctcatagc tcacgctgta     3720

ggtatctcag ttcggtgtag gtcgttcgct ccaagctggg ctgtgtgcac gaaccccccg     3780

ttcagcccga ccgctgcgcc ttatccggta actatcgtct tgagtccaac ccggtaagac     3840

acgacttatc gccactggca gcagccactg gtaacaggat tagcagagcg aggtatgtag     3900

gcggtgctac agagttcttg aagtggtggc ctaactacgg ctacactaga agaacagtat     3960

ttggtatctg cgctctgctg aagccagtta ccttcggaaa aagagttggt agctcttgat     4020

ccggcaaaca aaccaccgct ggtagcggtt tttttgtttg caagcagcag attacgcgca     4080

gaaaaaaagg atctcaagaa gatcctttga tcttttctac ggggtctgac gctcagtgga     4140

acgaaaactc acgttaaggg attttggtca tgagattatc aaaaaggatc ttcacctaga     4200

tccttttaaa ttaaaaatga agttttaaat caatctaaag tatatatgag taaacttggt     4260

ctgacagtta ccaatgctta atcagtgagg cacctatctc agcgatctgt ctatttcgtt     4320

catccatagt tgcctgactc cccgtcgtgt agataactac gatacgggag ggcttaccat     4380

ctggccccag tgctgcaatg ataccgcgag acccacgctc accggctcca gatttatcag     4440

caataaacca gccagccgga agggccgagc gcagaagtgg tcctgcaact ttatccgcct     4500

ccatccagtc tattaattgt tgccgggaag ctagagtaag tagttcgcca gttaatagtt     4560

tgcgcaacgt tgttgccatt gctacaggca tcgtggtgtc acgctcgtcg tttggtatgg     4620

cttcattcag ctccggttcc caacgatcaa ggcgagttac atgatccccc atgttgtgca     4680

aaaaagcggt tagctccttc ggtcctccga tcgttgtcag aagtaagttg gccgcagtgt     4740

tatcactcat ggttatggca gcactgcata attctcttac tgtcatgcca tccgtaagat     4800

gcttttctgt gactggtgag tactcaacca agtcattctg agaatagtgt atgcggcgac     4860

cgagttgctc ttgcccggcg tcaatacggg ataataccgc gccacatagc agaactttaa     4920

aagtgctcat cattggaaaa cgttcttcgg ggcgaaaact ctcaaggatc ttaccgctgt     4980

tgagatccag ttcgatgtaa cccactcgtg cacccaactg atcttcagca tcttttactt     5040

tcaccagcgt ttctgggtga gcaaaaacag gaaggcaaaa tgccgcaaaa aagggaataa     5100

gggcgacacg gaaatgttga atactcatac tcttcctttt tcaatattat tgaagcattt     5160

atcagggtta ttgtctcatg agcggataca tatttgaatg tatttagaaa aataaacaaa     5220

taggggttcc gcgcacattt ccccgaaaag tgccacctga cgtc                      5264


<210>  65
<211>  5100
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide sequence of TREX2-encoding plasmid pExodus CMV.Trex2

<400>  65
gacggatcgg gagatctccc gatcccctat ggtgcactct cagtacaatc tgctctgatg       60

ccgcatagtt aagccagtat ctgctccctg cttgtgtgtt ggaggtcgct gagtagtgcg      120

cgagcaaaat ttaagctaca acaaggcaag gcttgaccga caattgcatg aagaatctgc      180

ttagggttag gcgttttgcg ctgcttcgcg atgtacgggc cagatatacg cgttgacatt      240

gattattgac tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata      300

tggagttccg cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc      360

cccgcccatt gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc      420

attgacgtca atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt      480

atcatatgcc aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt      540

atgcccagta catgacctta tgggactttc ctacttggca gtacatctac gtattagtca      600

tcgctattac catggtgatg cggttttggc agtacatcaa tgggcgtgga tagcggtttg      660

actcacgggg atttccaagt ctccacccca ttgacgtcaa tgggagtttg ttttggcacc      720

aaaatcaacg ggactttcca aaatgtcgta acaactccgc cccattgacg caaatgggcg      780

gtaggcgtgt acggtgggag gtctatataa gcagagctct ctggctaact agagaaccca      840

ctgcttactg gcttatcgaa attaatacga ctcactatag ggagacccaa gctggctagc      900

atgtctgagc cacctcgggc tgagaccttt gtattcctgg acctagaagc cactgggctc      960

ccaaacatgg accctgagat tgcagagata tccctttttg ctgttcaccg ctcttccctg     1020

gagaacccag aacgggatga ttctggttcc ttggtgctgc cccgtgttct ggacaagctc     1080

acactgtgca tgtgcccgga gcgccccttt actgccaagg ccagtgagat tactggtttg     1140

agcagcgaaa gcctgatgca ctgcgggaag gctggtttca atggcgctgt ggtaaggaca     1200

ctgcagggct tcctaagccg ccaggagggc cccatctgcc ttgtggccca caatggcttc     1260

gattatgact tcccactgct gtgcacggag ctacaacgtc tgggtgccca tctgccccaa     1320

gacactgtct gcctggacac actgcctgca ttgcggggcc tggaccgtgc tcacagccac     1380

ggcaccaggg ctcaaggccg caaaagctac agcctggcca gtctcttcca ccgctacttc     1440

caggctgaac ccagtgctgc ccattcagca gaaggtgatg tgcacaccct gcttctgatc     1500

ttcctgcatc gtgctcctga gctgctcgcc tgggcagatg agcaggcccg cagctgggct     1560

catattgagc ccatgtacgt gccacctgat ggtccaagcc tcgaagcctg aattctgcag     1620

atatccagca cagtggcggc cgctcgagtc tagagggccc gtttaaaccc gctgatcagc     1680

ctcgactgtg ccttctagtt gccagccatc tgttgtttgc ccctcccccg tgccttcctt     1740

gaccctggaa ggtgccactc ccactgtcct ttcctaataa aatgaggaaa ttgcatcgca     1800

ttgtctgagt aggtgtcatt ctattctggg gggtggggtg gggcaggaca gcaaggggga     1860

ggattgggaa gacaatagca ggcatgctgg ggatgcggtg ggctctatgg cttctgaggc     1920

ggaaagaacc agctggggct ctagggggta tccccacgcg ccctgtagcg gcgcattaag     1980

cgcggcgggt gtggtggtta cgcgcagcgt gaccgctaca cttgccagcg ccctagcgcc     2040

cgctcctttc gctttcttcc cttcctttct cgccacgttc gccggctttc cccgtcaagc     2100

tctaaatcgg gggctccctt tagggttccg atttagtgct ttacggcacc tcgaccccaa     2160

aaaacttgat tagggtgatg gttcacgtag tgggccatcg ccctgataga cggtttttcg     2220

ccctttgacg ttggagtcca cgttctttaa tagtggactc ttgttccaaa ctggaacaac     2280

actcaaccct atctcggtct attcttttga tttataaggg attttgccga tttcggccta     2340

ttggttaaaa aatgagctga tttaacaaaa atttaacgcg aattaattct gtggaatgtg     2400

tgtcagttag ggtgtggaaa gtccccaggc tccccagcag gcagaagtat gcaaagccta     2460

tcaggacata gcgttggcta cccgtgatat tgctgaagag cttggcggcg aatgggctga     2520

ccgcttcctc gtgctttacg gtatcgccgc tcccgattcg cagcgcatcg ccttctatcg     2580

ccttcttgac gagttcttct gagcgggact ctggggttcg aaatgaccga ccaagcgacg     2640

cccaacctgc catcacgaga tttcgattcc accgccgcct tctatgaaag gttgggcttc     2700

ggaatcgttt tccgggacgc cggctggatg atcctccagc gcggggatct catgctggag     2760

ttcttcgccc accccaactt gtttattgca gcttataatg gttacaaata aagcaatagc     2820

atcacaaatt tcacaaataa agcatttttt tcactgcatt ctagttgtgg tttgtccaaa     2880

ctcatcaatg tatcttatca tgtctgtata ccgtcgacct ctagctagag cttggcgtaa     2940

tcatggtcat agctgtttcc tgtgtgaaat tgttatccgc tcacaattcc acacaacata     3000

cgagccggaa gcataaagtg taaagcctgg ggtgcctaat gagtgagcta actcacatta     3060

attgcgttgc gctcactgcc cgctttccag tcgggaaacc tgtcgtgcca gctgcattaa     3120

tgaatcggcc aacgcgcggg gagaggcggt ttgcgtattg ggcgctcttc cgcttcctcg     3180

ctcactgact cgctgcgctc ggtcgttcgg ctgcggcgag cggtatcagc tcactcaaag     3240

gcggtaatac ggttatccac agaatcaggg gataacgcag gaaagaacat gtgagcaaaa     3300

ggccagcaaa aggccaggaa ccgtaaaaag gccgcgttgc tggcgttttt ccataggctc     3360

cgcccccctg acgagcatca caaaaatcga cgctcaagtc agaggtggcg aaacccgaca     3420

ggactataaa gataccaggc gtttccccct ggaagctccc tcgtgcgctc tcctgttccg     3480

accctgccgc ttaccggata cctgtccgcc tttctccctt cgggaagcgt ggcgctttct     3540

catagctcac gctgtaggta tctcagttcg gtgtaggtcg ttcgctccaa gctgggctgt     3600

gtgcacgaac cccccgttca gcccgaccgc tgcgccttat ccggtaacta tcgtcttgag     3660

tccaacccgg taagacacga cttatcgcca ctggcagcag ccactggtaa caggattagc     3720

agagcgaggt atgtaggcgg tgctacagag ttcttgaagt ggtggcctaa ctacggctac     3780

actagaagaa cagtatttgg tatctgcgct ctgctgaagc cagttacctt cggaaaaaga     3840

gttggtagct cttgatccgg caaacaaacc accgctggta gcggtttttt tgtttgcaag     3900

cagcagatta cgcgcagaaa aaaaggatct caagaagatc ctttgatctt ttctacgggg     3960

tctgacgctc agtggaacga aaactcacgt taagggattt tggtcatgag attatcaaaa     4020

aggatcttca cctagatcct tttaaattaa aaatgaagtt ttaaatcaat ctaaagtata     4080

tatgagtaaa cttggtctga cagttaccaa tgcttaatca gtgaggcacc tatctcagcg     4140

atctgtctat ttcgttcatc catagttgcc tgactccccg tcgtgtagat aactacgata     4200

cgggagggct taccatctgg ccccagtgct gcaatgatac cgcgagaccc acgctcaccg     4260

gctccagatt tatcagcaat aaaccagcca gccggaaggg ccgagcgcag aagtggtcct     4320

gcaactttat ccgcctccat ccagtctatt aattgttgcc gggaagctag agtaagtagt     4380

tcgccagtta atagtttgcg caacgttgtt gccattgcta caggcatcgt ggtgtcacgc     4440

tcgtcgtttg gtatggcttc attcagctcc ggttcccaac gatcaaggcg agttacatga     4500

tcccccatgt tgtgcaaaaa agcggttagc tccttcggtc ctccgatcgt tgtcagaagt     4560

aagttggccg cagtgttatc actcatggtt atggcagcac tgcataattc tcttactgtc     4620

atgccatccg taagatgctt ttctgtgact ggtgagtact caaccaagtc attctgagaa     4680

tagtgtatgc ggcgaccgag ttgctcttgc ccggcgtcaa tacgggataa taccgcgcca     4740

catagcagaa ctttaaaagt gctcatcatt ggaaaacgtt cttcggggcg aaaactctca     4800

aggatcttac cgctgttgag atccagttcg atgtaaccca ctcgtgcacc caactgatct     4860

tcagcatctt ttactttcac cagcgtttct gggtgagcaa aaacaggaag gcaaaatgcc     4920

gcaaaaaagg gaataagggc gacacggaaa tgttgaatac tcatactctt cctttttcaa     4980

tattattgaa gcatttatca gggttattgt ctcatgagcg gatacatatt tgaatgtatt     5040

tagaaaaata aacaaatagg ggttccgcgc acatttcccc gaaaagtgcc acctgacgtc     5100


<210>  66
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PCR Primer

<400>  66
gctggaatgg ttgcagtaac                                                   20


<210>  67
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PCR Primer

<400>  67
caaacagcca ttcaccagtg                                                   20


<210>  68
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PCR Primer

<400>  68
ctgccagctc tctaagtctc c                                                 21


<210>  69
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PCR Primer

<400>  69
tgcaacacgc acagaacact c                                                 21


