                         SEQUENCE LISTING

<110>  Ospedale San Raffaele S.r.l
       Fondazione Telethon
 
<120>  SELECTION BY MEANS OF ARTIFICIAL TRANSACTIVATORS

<130>  P115250PCT

<140>  PCT/EP2019/077657
<141>  2019-10-11

<150>  EP 18199952.5
<151>  2018-10-11

<160>  91    

<170>  PatentIn version 3.5

<210>  1
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  1
tgggggttag acccaatatc agg                                               23


<210>  2
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  2
ccttcctagt ctcctgatat tgg                                               23


<210>  3
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  3
ccaatatcag gagactagga agg                                               23


<210>  4
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  4
agataaggaa tctgcctaac agg                                               23


<210>  5
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  5
tgttaggcag attccttatc tgg                                               23


<210>  6
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  6
tgggggtgtg tcaccagata agg                                               23


<210>  7
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  7
tctccttgcc agaacctcta agg                                               23


<210>  8
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  8
cctctaaggt ttgcttacga tgg                                               23


<210>  9
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  9
aaaccttaga ggttctggca agg                                               23


<210>  10
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  10
taagcaaacc ttagaggttc tgg                                               23


<210>  11
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  11
gtgacctgcc cggttctcag tgg                                               23


<210>  12
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  12
ggggggatgc gtgacctgcc cgg                                               23


<210>  13
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  13
gttctgggag agggtagcgc agg                                               23


<210>  14
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  14
gctgctctga cgcggccgtc tgg                                               23


<210>  15
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  15
gaacctgagc tgctctgacg cgg                                               23


<210>  16
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  16
cggccgcgtc agagcagctc agg                                               23


<210>  17
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  17
ctggtgcgtt tcactgatcc tgg                                               23


<210>  18
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  18
gcttccttac acttcccaag agg                                               23


<210>  19
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  19
gatcagtgaa acgcaccaga cgg                                               23


<210>  20
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  20
ttggtcctga gttctaactt tgg                                               23


<210>  21
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  21
tcccaagagg agaagcagtt tgg                                               23


<210>  22
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  22
cggaggaaca atataaattg ggg                                               23


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

<220>
<223>  target sequence

<400>  23
tcacaggtaa aactgacgca cgg                                               23


<210>  24
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  24
gccagtagcc agccccgtcc tgg                                               23


<210>  25
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  25
gtagccagcc ccgtcctggc agg                                               23


<210>  26
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  26
ggctactggc cttatctcac agg                                               23


<210>  27
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  27
cctaggtgtt caccaggtcg tgg                                               23


<210>  28
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  28
ttgtgagaat ggtgcgtcct agg                                               23


<210>  29
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  29
gagtagaggc ggccacgacc tgg                                               23


<210>  30
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  30
gtgcgtccta ggtgttcacc agg                                               23


<210>  31
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  target sequence

<400>  31
aaagagtccc cagtgctatc tgg                                               23


<210>  32
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  32
tccatcgtaa gcaaacctt                                                    19


<210>  33
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  33
tcccaccccc tgccaagct                                                    19


<210>  34
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  34
tccaaactgc ttctcctct                                                    19


<210>  35
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  35
tccacacgga cacccccct                                                    19


<210>  36
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  36
tccaccatct catgcccct                                                    19


<210>  37
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  37
tctaaggttt gcttacgat                                                    19


<210>  38
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  38
tcctctctgg ctccatcgt                                                    19


<210>  39
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  39
tggtcctgag ttctaactt                                                    19


<210>  40
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TALE binding site, target sequence

<400>  40
tcctccgtgc gtcagtttt                                                    19


<210>  41
<211>  148
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  T6-SK SMArT minimal promoter

<400>  41
tctagaatta gctttaggcg tgtacggtgg gcgcctataa aagcagagct cgtttagtga       60

accgtcagat cgcctggagc aattccacaa cacttttgtc ttataccaac tttccgtacc      120

acttcctacc ctcgtaaaga atccgcgg                                         148


<210>  42
<211>  109
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  minCMV SMArT minimal promoter

<400>  42
gtacggtggg aggcctatat aagcagagct cgtttagtga accgtcagat cgcctggaga       60

cgccatccac gctgttttga cctccataga agacaccggg accgatcca                  109


<210>  43
<211>  720
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  selector eGFP

<400>  43
atggtgagca agggcgagga gctgttcacc ggggtggtgc ccatcctggt cgagctggac       60

ggcgacgtaa acggccacaa gttcagcgtg tccggcgagg gcgagggcga tgccacctac      120

ggcaagctga ccctgaagtt catctgcacc accggcaagc tgcccgtgcc ctggcccacc      180

ctcgtgacca ccctgaccta cggcgtgcag tgcttcagcc gctaccccga ccacatgaag      240

cagcacgact tcttcaagtc cgccatgccc gaaggctacg tccaggagcg caccatcttc      300

ttcaaggacg acggcaacta caagacccgc gccgaggtga agttcgaggg cgacaccctg      360

gtgaaccgca tcgagctgaa gggcatcgac ttcaaggagg acggcaacat cctggggcac      420

aagctggagt acaactacaa cagccacaac gtctatatca tggccgacaa gcagaagaac      480

ggcatcaagg tgaacttcaa gatccgccac aacatcgagg acggcagcgt gcagctcgcc      540

gaccactacc agcagaacac ccccatcggc gacggccccg tgctgctgcc cgacaaccac      600

tacctgagca cccagtccgc cctgagcaaa gaccccaacg agaagcgcga tcacatggtc      660

ctgctggagt tcgtgaccgc cgccgggatc actctcggca tggacgagct gtacaagtaa      720


<210>  44
<211>  843
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  selector NGFR

<400>  44
atgggggcag gtgccaccgg ccgcgccatg gacgggccgc gcctgctgct gttgctgctt       60

ctgggggtgt cccttggagg tgccaaggag gcatgcccca caggcctgta cacacacagc      120

ggtgagtgct gcaaagcctg caacctgggc gagggtgtgg cccagccttg tggagccaac      180

cagaccgtgt gtgagccctg cctggacagc gtgacgttct ccgacgtggt gagcgcgacc      240

gagccgtgca agccgtgcac cgagtgcgtg gggctccaga gcatgtcggc gccgtgcgtg      300

gaggccgacg acgccgtgtg ccgctgcgcc tacggctact accaggatga gacgactggg      360

cgctgcgagg cgtgccgcgt gtgcgaggcg ggctcgggcc tcgtgttctc ctgccaggac      420

aagcagaaca ccgtgtgcga ggagtgcccc gacggcacgt attccgacga ggccaaccac      480

gtggacccgt gcctgccctg caccgtgtgc gaggacaccg agcgccagct ccgcgagtgc      540

acacgctggg ccgacgccga gtgcgaggag atccctggcc gttggattac acggtccaca      600

cccccagagg gctcggacag cacagccccc agcacccagg agcctgaggc acctccagaa      660

caagacctca tagccagcac ggtggcaggt gtggtgacca cagtgatggg cagctcccag      720

cccgtggtga cccgaggcac caccgacaac ctcatccctg tctattgctc catcctggct      780

gctgtggttg tgggccttgt ggcctacata gccttcaaga ggtggaacag ggggatcctc      840

tag                                                                    843


<210>  45
<211>  4284
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  engineered transcriptional transactivator (ETT) Sp-dCas9

<400>  45
atgggcggta ggcgtgtacg gtgggaggtc tatataagca gagctcgttt agtgaaccgt       60

cagatcgcct ggagacgcca tccacgctgt tttgacctcc atagaagaca ccgggaccga      120

tccagcctcc ggactctaga ggatcgaacc cttgccacca tggacaagaa gtactccatt      180

gggctcgcta tcggcacaaa cagcgtcggc tgggccgtca ttacggacga gtacaaggtg      240

ccgagcaaaa aattcaaagt tctgggcaat accgatcgcc acagcataaa gaagaacctc      300

attggcgccc tcctgttcga ctccggggag acggccgaag ccacgcggct caaaagaaca      360

gcacggcgca gatatacccg cagaaagaat cggatctgct acctgcagga gatctttagt      420

aatgagatgg ctaaggtgga tgactctttc ttccataggc tggaggagtc ctttttggtg      480

gaggaggata aaaagcacga gcgccaccca atctttggca atatcgtgga cgaggtggcg      540

taccatgaaa agtacccaac catatatcat ctgaggaaga agcttgtaga cagtactgat      600

aaggctgact tgcggttgat ctatctcgcg ctggcgcata tgatcaaatt tcggggacac      660

ttcctcatcg agggggacct gaacccagac aacagcgatg tcgacaaact ctttatccaa      720

ctggttcaga cttacaatca gcttttcgaa gagaacccga tcaacgcatc cggagttgac      780

gccaaagcaa tcctgagcgc taggctgtcc aaatcccggc ggctcgaaaa cctcatcgca      840

cagctccctg gggagaagaa gaacggcctg tttggtaatc ttatcgccct gtcactcggg      900

ctgaccccca actttaaatc taacttcgac ctggccgaag atgccaagct tcaactgagc      960

aaagacacct acgatgatga tctcgacaat ctgctggccc agatcggcga ccagtacgca     1020

gacctttttt tggcggcaaa gaacctgtca gacgccattc tgctgagtga tattctgcga     1080

gtgaacacgg agatcaccaa agctccgctg agcgctagta tgatcaagcg ctatgatgag     1140

caccaccaag acttgacttt gctgaaggcc cttgtcagac agcaactgcc tgagaagtac     1200

aaggaaattt tcttcgatca gtctaaaaat ggctacgccg gatacattga cggcggagca     1260

agccaggagg aattttacaa atttattaag cccatcttgg aaaaaatgga cggcaccgag     1320

gagctgctgg taaagcttaa cagagaagat ctgttgcgca aacagcgcac tttcgacaat     1380

ggaagcatcc cccaccagat tcacctgggc gaactgcacg ctatcctcag gcggcaagag     1440

gatttctacc cctttttgaa agataacagg gaaaagattg agaaaatcct cacatttcgg     1500

ataccctact atgtaggccc cctcgcccgg ggaaattcca gattcgcgtg gatgactcgc     1560

aaatcagaag agaccatcac tccctggaac ttcgaggaag tcgtggataa gggggcctct     1620

gcccagtcct tcatcgaaag gatgactaac tttgataaaa atctgcctaa cgaaaaggtg     1680

cttcctaaac actctctgct gtacgagtac ttcacagttt ataacgagct caccaaggtc     1740

aaatacgtca cagaagggat gagaaagcca gcattcctgt ctggagagca gaagaaagct     1800

atcgtggacc tcctcttcaa gacgaaccgg aaagttaccg tgaaacagct caaagaagac     1860

tatttcaaaa agattgaatg tttcgactct gttgaaatca gcggagtgga ggatcgcttc     1920

aacgcatccc tgggaacgta tcacgatctc ctgaaaatca ttaaagacaa ggacttcctg     1980

gacaatgagg agaacgagga cattcttgag gacattgtcc tcacccttac gttgtttgaa     2040

gatagggaga tgattgaaga acgcttgaaa acttacgctc atctcttcga cgacaaagtc     2100

atgaaacagc tcaagaggcg ccgatataca ggatgggggc ggctgtcaag aaaactgatc     2160

aatgggatcc gagacaagca gagtggaaag acaatcctgg attttcttaa gtccgatgga     2220

tttgccaacc ggaacttcat gcagttgatc catgatgact ctctcacctt taaggaggac     2280

atccagaaag cacaagtttc tggccagggg gacagtcttc acgagcacat cgctaatctt     2340

gcaggtagcc cagctatcaa aaagggaata ctgcagaccg ttaaggtcgt ggatgaactc     2400

gtcaaagtaa tgggaaggca taagcccgag aatatcgtta tcgagatggc ccgagagaac     2460

caaactaccc agaagggaca gaagaacagt agggaaagga tgaagaggat tgaagagggt     2520

ataaaagaac tggggtccca aatccttaag gaacacccag ttgaaaacac ccagcttcag     2580

aatgagaagc tctacctgta ctacctgcag aacggcaggg acatgtacgt ggatcaggaa     2640

ctggacatca atcggctctc cgactacgac gtggctgcta tcgtgcccca gtcttttctc     2700

aaagatgatt ctattgataa taaagtgttg acaagatccg ataaagctag agggaagagt     2760

gataacgtcc cctcagaaga agttgtcaag aaaatgaaaa attattggcg gcagctgctg     2820

aacgccaaac tgatcacaca acggaagttc gataatctga ctaaggctga acgaggtggc     2880

ctgtctgagt tggataaagc cggcttcatc aaaaggcagc ttgttgagac acgccagatc     2940

accaagcacg tggcccaaat tctcgattca cgcatgaaca ccaagtacga tgaaaatgac     3000

aaactgattc gagaggtgaa agttattact ctgaagtcta agctggtctc agatttcaga     3060

aaggactttc agttttataa ggtgagagag atcaacaatt accaccatgc gcatgatgcc     3120

tacctgaatg cagtggtagg cactgcactt atcaaaaaat atcccaagct tgaatctgaa     3180

tttgtttacg gagactataa agtgtacgat gttaggaaaa tgatcgcaaa gtctgagcag     3240

gaaataggca aggccaccgc taagtacttc ttttacagca atattatgaa ttttttcaag     3300

accgagatta cactggccaa tggagagatt cggaagcgac cacttatcga aacaaacgga     3360

gaaacaggag aaatcgtgtg ggacaagggt agggatttcg cgacagtccg gaaggtcctg     3420

tccatgccgc aggtgaacat cgttaaaaag accgaagtac agaccggagg cttctccaag     3480

gaaagtatcc tcccgaaaag gaacagcgac aagctgatcg cacgcaaaaa agattgggac     3540

cccaagaaat acggcggatt cgattctcct acagtcgctt acagtgtact ggttgtggcc     3600

aaagtggaga aagggaagtc taaaaaactc aaaagcgtca aggaactgct gggcatcaca     3660

atcatggagc gatcaagctt cgaaaaaaac cccatcgact ttctcgaggc gaaaggatat     3720

aaagaggtca aaaaagacct catcattaag cttcccaagt actctctctt tgagcttgaa     3780

aacggccgga aacgaatgct cgctagtgcg ggcgagctgc agaaaggtaa cgagctggca     3840

ctgccctcta aatacgttaa tttcttgtat ctggccagcc actatgaaaa gctcaaaggg     3900

tctcccgaag ataatgagca gaagcagctg ttcgtggaac aacacaaaca ctaccttgat     3960

gagatcatcg agcaaataag cgaattctcc aaaagagtga tcctcgccga cgctaacctc     4020

gataaggtgc tttctgctta caataagcac agggataagc ccatcaggga gcaggcagaa     4080

aacattatcc acttgtttac tctgaccaac ttgggcgcgc ctgcagcctt caagtacttc     4140

gacaccacca tagacagaaa gcggtacacc tctacaaagg aggtcctgga cgccacactg     4200

attcatcagt caattacggg gctctatgaa acaagaatcg acctctctca gctcggtgga     4260

gacagcaggg ctgaccccaa gtga                                            4284


<210>  46
<211>  417
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ETT VP160 domain

<400>  46
gatccggagg cggggcggac gcgctggacg atttcgatct cgacatgctg ggttctgatg       60

ccctcgatga ctttgacctg gatatgttgg gaagcgacgc attggatgac tttgatctgg      120

acatgctcgg ctccgatgct ctggacgatt tcgatctcga tatgttaggg tcagacgcac      180

tggatgattt cgaccttgat atgttgggaa gcgatgccct tgatgatttc gacctggaca      240

tgctcggcag cgacgccctg gacgatttcg atctggacat gctggggtcc gatgccttgg      300

atgattttga cttggatatg ctggggagtg atgccctgga cgactttgac ctggacatgc      360

tgggctccga tgcgctcgat gacttcgatt tggatatgtt gtattgaaag cttctga         417


<210>  47
<211>  1677
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ETT VPR domain

<400>  47
aagaagagga aggtgtcgcc agggatccgt cgacttgacg cgttgatatc aacaagtttg       60

tacaaaaaag caggctacaa agaggccagc ggttccggac gggctgacgc attggacgat      120

tttgatctgg atatgctggg aagtgacgcc ctcgatgatt ttgaccttga catgcttggt      180

tcggatgccc ttgatgactt tgacctcgac atgctcggca gtgacgccct tgatgatttc      240

gacctggaca tgctgattaa ctctagaagt tccggatctc cgaaaaagaa acgcaaagtt      300

ggtagccagt acctgcccga caccgacgac cggcaccgga tcgaggaaaa gcggaagcgg      360

acctacgaga cattcaagag catcatgaag aagtccccct tcagcggccc caccgaccct      420

agacctccac ctagaagaat cgccgtgccc agcagatcca gcgccagcgt gccaaaacct      480

gccccccagc cttacccctt caccagcagc ctgagcacca tcaactacga cgagttccct      540

accatggtgt tccccagcgg ccagatctct caggcctctg ctctggctcc agcccctcct      600

caggtgctgc ctcaggctcc tgctcctgca ccagctccag ccatggtgtc tgcactggct      660

caggcaccag cacccgtgcc tgtgctggct cctggacctc cacaggctgt ggctccacca      720

gcccctaaac ctacacaggc cggcgagggc acactgtctg aagctctgct gcagctgcag      780

ttcgacgacg aggatctggg agccctgctg ggaaacagca ccgatcctgc cgtgttcacc      840

gacctggcca gcgtggacaa cagcgagttc cagcagctgc tgaaccaggg catccctgtg      900

gcccctcaca ccaccgagcc catgctgatg gaataccccg aggccatcac ccggctcgtg      960

acaggcgctc agaggcctcc tgatccagct cctgcccctc tgggagcacc aggcctgcct     1020

aatggactgc tgtctggcga cgaggacttc agctctatcg ccgatatgga tttctcagcc     1080

ttgctgggct ctggcagcgg cagccgggat tccagggaag ggatgttttt gccgaagcct     1140

gaggccggct ccgctattag tgacgtgttt gagggccgcg aggtgtgcca gccaaaacga     1200

atccggccat ttcatcctcc aggaagtcca tgggccaacc gcccactccc cgccagcctc     1260

gcaccaacac caaccggtcc agtacatgag ccagtcgggt cactgacccc ggcaccagtc     1320

cctcagccac tggatccagc gcccgcagtg actcccgagg ccagtcacct gttggaggat     1380

cccgatgaag agacgagcca ggctgtcaaa gcccttcggg agatggccga tactgtgatt     1440

ccccagaagg aagaggctgc aatctgtggc caaatggacc tttcccatcc gcccccaagg     1500

ggccatctgg atgagctgac aaccacactt gagtccatga ccgaggatct gaacctggac     1560

tcacccctga ccccggaatt gaacgagatt ctggatacct tcctgaacga cgagtgcctc     1620

ttgcatgcca tgcatatcag cacaggactg tccatcttcg acacatctct gttttga        1677


<210>  48
<211>  2521
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ETT TALE7 DBD (SMArT)

<400>  48
aaacgggccc tctagactcg agcggccgcg ccaccatggg aaaacctatt cctaatcctc       60

tgctgggcct ggattctacc ggaggcatgg cccctaagaa aaagcggaag gtggacggcg      120

gagtggacct gagaacactg ggatattctc agcagcagca ggagaagatc aagcccaagg      180

tgagatctac agtggcccag caccacgaag ccctggtggg acacggattt acacacgccc      240

acattgtggc cctgtctcag caccctgccg ccctgggaac agtggccgtg aaatatcagg      300

atatgattgc cgccctgcct gaggccacac acgaagccat tgtgggagtg ggaaaacagt      360

ggtctggagc cagagccctg gaagccctgc tgacagtggc cggagaactg agaggacctc      420

ctctgcagct ggatacagga cagctgctga agattgccaa aaggggcgga gtgaccgcgg      480

tggaagccgt gcacgcctgg agaaatgccc tgacgggtgc ccccctgaac ctgaccccgg      540

accaagtggt ggctatcgcc agccacgatg gcggcaagca agcgctcgaa acggtgcagc      600

ggctgttgcc ggtgctgtgc caggaccatg gcctgactcc ggaccaagtg gtggctatcg      660

ccagccacga tggcggcaag caagcgctcg aaacggtgca gcggctgttg ccggtgctgt      720

gccaggacca tggcctgacc ccggaccaag tggtggctat cgccagcaac ggtggcggca      780

agcaagcgct cgaaacggtg cagcggctgt tgccggtgct gtgccaggac catggcctga      840

ctccggacca agtggtggct atcgccagcc acgatggcgg caagcaagcg ctcgaaacgg      900

tgcagcggct gttgccggtg ctgtgccagg accatggcct gaccccggac caagtggtgg      960

ctatcgccag caacggtggc ggcaagcaag cgctcgaaac ggtgcagcgg ctgttgccgg     1020

tgctgtgcca ggaccatggc ctgactccgg accaagtggt ggctatcgcc agccacgatg     1080

gcggcaagca agcgctcgaa acggtgcagc ggctgttgcc ggtgctgtgc caggaccatg     1140

gcctgacccc ggaccaagtg gtggctatcg ccagcaacgg tggcggcaag caagcgctcg     1200

aaacggtgca gcggctgttg ccggtgctgt gccaggacca tggcctgacc ccggaccaag     1260

tggtggctat cgccagcaat cacggcggca agcaagcgct cgaaacggtg cagcggctgt     1320

tgccggtgct gtgccaggac catggcctga ccccggacca agtggtggct atcgccagca     1380

atcacggcgg caagcaagcg ctcgaaacgg tgcagcggct gttgccggtg ctgtgccagg     1440

accatggcct gactccggac caagtggtgg ctatcgccag ccacgatggc ggcaagcaag     1500

cgctcgaaac ggtgcagcgg ctgttgccgg tgctgtgcca ggaccatggc ctgaccccgg     1560

accaagtggt ggctatcgcc agcaacggtg gcggcaagca agcgctcgaa acggtgcagc     1620

ggctgttgcc ggtgctgtgc caggaccatg gcctgactcc ggaccaagtg gtggctatcg     1680

ccagccacga tggcggcaag caagcgctcg aaacggtgca gcggctgttg ccggtgctgt     1740

gccaggacca tggcctgact ccggaccaag tggtggctat cgccagccac gatggcggca     1800

agcaagcgct cgaaacggtg cagcggctgt tgccggtgct gtgccaggac catggcctga     1860

ccccggacca agtggtggct atcgccagca acattggcgg caagcaagcg ctcgaaacgg     1920

tgcagcggct gttgccggtg ctgtgccagg accatggcct gaccccggac caagtggtgg     1980

ctatcgccag caacggtggc ggcaagcaag cgctcgaaac ggtgcagcgg ctgttgccgg     2040

tgctgtgcca ggaccatggc ctgactccgg accaagtggt ggctatcgcc agccacgatg     2100

gcggcaagca agcgctcgaa acggtgcagc ggctgttgcc ggtgctgtgc caggaccatg     2160

gcctgacccc ggaccaagtg gtggctatcg ccagcaatca cggcggcaag caagcgctcg     2220

aaacggtgca gcggctgttg ccggtgctgt gccaggacca tggcctgacc ccggaccaag     2280

tggtggctat cgccagcaac ggtggcggca agcaagcgct cgaaagcatt gtggcccagc     2340

tgagccggcc tgatccggcg ttggccgcgt tgaccaacga tcacctggtg gccctggcct     2400

gtctgggagg cagacctgcc ctggatgccg tgaaaaaagg actgcctcac gcccctgccc     2460

tgatcaagag aacaaataga agaatccccg agcggacctc tcacagagtg gccggatcac     2520

a                                                                     2521


<210>  49
<211>  2391
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  ETT TALE3 DBD (SMArTER)

<400>  49
gtggacctga gaacactggg atattctcag cagcagcagg agaagatcaa gcccaaggtg       60

agatctacag tggcccagca ccacgaagcc ctggtgggac acggatttac acacgcccac      120

attgtggccc tgtctcagca ccctgccgcc ctgggaacag tggccgtgaa atatcaggat      180

atgattgccg ccctgcctga ggccacacac gaagccattg tgggagtggg aaaacagtgg      240

tctggagcca gagccctgga agccctgctg acagtggccg gagaactgag aggacctcct      300

ctgcagctgg atacaggaca gctgctgaag attgccaaaa ggggcggagt gaccgcggtg      360

gaagccgtgc acgcctggag aaatgccctg acgggtgccc ccctgaacct gaccccggac      420

caagtggtgg ctatcgccag caacaatggc ggcaagcaag cgctcgaaac ggtgcagcgg      480

ctgttgccgg tgctgtgcca ggaccatggc ctgaccccgg accaagtggt ggctatcgcc      540

agcaacggtg gcggcaagca agcgctcgaa acggtgcagc ggctgttgcc ggtgctgtgc      600

caggaccatg gcctgactcc ggaccaagtg gtggctatcg ccagccacga tggcggcaag      660

caagcgctcg aaacggtgca gcggctgttg ccggtgctgt gccaggacca tggcctgacc      720

ccggaccaag tggtggctat cgccagcaac attggcggca agcaagcgct cgaaacggtg      780

cagcggctgt tgccggtgct gtgccaggac catggcctga ctccggacca agtggtggct      840

atcgccagcc acgatggcgg caagcaagcg ctcgaaacgg tgcagcggct gttgccggtg      900

ctgtgccagg accatggcct gaccccggac caagtggtgg ctatcgccag caacattggc      960

ggcaagcaag cgctcgaaac ggtgcagcgg ctgttgccgg tgctgtgcca ggaccatggc     1020

ctgactccgg accaagtggt ggctatcgcc agccacgatg gcggcaagca agcgctcgaa     1080

acggtgcagc ggctgttgcc ggtgctgtgc caggaccatg gcctgacccc ggaccaagtg     1140

gtggctatcg ccagcaacat tggcggcaag caagcgctcg aaacggtgca gcggctgttg     1200

ccggtgctgt gccaggacca tggcctgacc ccggaccaag tggtggctat cgccagcaat     1260

cacggcggca agcaagcgct cgaaacggtg cagcggctgt tgccggtgct gtgccaggac     1320

catggcctga ctccggacca agtggtggct atcgccagcc acgatggcgg caagcaagcg     1380

ctcgaaacgg tgcagcggct gttgccggtg ctgtgccagg accatggcct gaccccggac     1440

caagtggtgg ctatcgccag caacattggc ggcaagcaag cgctcgaaac ggtgcagcgg     1500

ctgttgccgg tgctgtgcca ggaccatggc ctgactccgg accaagtggt ggctatcgcc     1560

agccacgatg gcggcaagca agcgctcgaa acggtgcagc ggctgttgcc ggtgctgtgc     1620

caggaccatg gcctgacccc ggaccaagtg gtggctatcg ccagcaacat tggcggcaag     1680

caagcgctcg aaacggtgca gcggctgttg ccggtgctgt gccaggacca tggcctgacc     1740

ccggaccaag tggtggctat cgccagcaac ggtggcggca agcaagcgct cgaaacggtg     1800

cagcggctgt tgccggtgct gtgccaggac catggcctga ccccggacca agtggtggct     1860

atcgccagca acattggcgg caagcaagcg ctcgaaacgg tgcagcggct gttgccggtg     1920

ctgtgccagg accatggcct gaccccggac caagtggtgg ctatcgccag caacggtggc     1980

ggcaagcaag cgctcgaaac ggtgcagcgg ctgttgccgg tgctgtgcca ggaccatggc     2040

ctgaccccgg accaagtggt ggctatcgcc agcaacggtg gcggcaagca agcgctcgaa     2100

acggtgcagc ggctgttgcc ggtgctgtgc caggaccatg gcctgacccc ggaccaagtg     2160

gtggctatcg ccagcaacgg tggcggcaag caagcgctcg aaagcattgt ggcccagctg     2220

agccggcctg atccggcgtt ggccgcgttg accaacgatc acctggtggc cctggcctgt     2280

ctgggaggca gacctgccct ggatgccgtg aaaaaaggac tgcctcacgc ccctgccctg     2340

atcaagagaa caaatagaag aatccccgag cggacctctc acagagtggc c              2391


<210>  50
<211>  801
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Left homology arm of AAVS1-targeting constructs

<400>  50
ccactgtggg gtggagggga cagataaaag tacccagaac cagagccaca ttaaccggcc       60

ctgggaatat aaggtggtcc cagctcgggg acacaggatc cctggaggca gcaaacatgc      120

tgtcctgaag tggacatagg ggcccgggtt ggaggaagaa gactagctga gctctcggac      180

ccctggaaga tgccatgaca gggggctgga agagctagca cagactagag aggtaagggg      240

ggtaggggag ctgcccaaat gaaaggagtg agaggtgacc cgaatccaca ggagaacggg      300

gtgtccaggc aaagaaagca agaggatgga gaggtggcta aagccaggga gacggggtac      360

tttggggttg tccagaaaaa cggtgatgat gcaggcctac aagaagggga ggcgggacgc      420

aagggagaca tccgtcggag aaggccatcc taagaaacga gagatggcac aggccccaga      480

aggagaagga aaagggaacc cagcgagtga agacggcatg gggttgggtg agggaggaga      540

gatgcccgga gaggacccag acacggggag gatccgctca gaggacatca cgtggtgcag      600

cgccgagaag gaagtgctcc ggaaagagca tccttgggca gcaacacagc agagagcaag      660

gggaagaggg agtggaggaa gacggaacct gaaggaggcg gcagggaagg atctgggcca      720

gccgtagagg tgacccaggc cacaagctgc agacagaaag cggcacaggc ccaggggaga      780

gaatgctggt cagagaaagc a                                                801


<210>  51
<211>  256
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SV40polyA of AAVS1-targeting constructs

<400>  51
gagatccaga catgataaga tacattgatg agtttggaca aaccaaaact agaatgcagt       60

gaaaaaaatg ccttatttgt gaaatttgtg atgctattgc cttatttgta accattataa      120

gctgcaataa acaagttaac aacaacaatt gcattcattt tatgtttcag gttcaggggg      180

aggtgtggga ggttttttaa agcaagtaaa acctctacaa atgtggtatg gctgattatg      240

atcagatctc tcgagg                                                      256


<210>  52
<211>  147
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Shortened left homology arm of AAVS1-targeting constructs

<400>  52
gcaaggagag agatggctcc aggaaatggg ggtgtgtcac cagataagga atctgcctaa       60

caggaggtgg gggttagacc caatatcagg agactaggaa ggaggaggcc taaggatggg      120

gcttttctgt caccaatcct gtcccta                                          147


<210>  53
<211>  290
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Left homology arm of IL2RG-targeting constructs

<400>  53
agaggaaacg tgtgggtggg gaggggtagt gggtgaggga cccaggttcc tgacacagac       60

agactacacc cagggaatga agagcaagcg ccatgttgaa gccatcatta ccattcacat      120

ccctcttatt cctgcagctg cccctgctgg gagtggggct gaacacgaca attctgacgc      180

ccaatgggaa tgaagacacc acagctggtg ggaaatctgg gactggaggg ggctggtgag      240

aagggtggct gtgggaaggg gccgtacaga gatctggtgc ctgccactgg                 290


<210>  54
<211>  1076
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  IL2RG recoded corrective cDNA (for intron 1 gene correction 
       strategy) of IL2RG-targeting constructs

<400>  54
atttctttct gaccaccatg cccaccgaca gcctgagcgt gagcaccctg cccctgcccg       60

aggtgcagtg cttcgtgttc aacgtggagt acatgaactg cacctggaac agcagcagcg      120

agccccagcc caccaatctg accctgcact actggtacaa gaacagcgac aacgacaagg      180

tgcagaagtg cagccactac ctgttcagcg aggaaatcac cagcggctgc cagctgcaga      240

agaaagagat ccacctgtac cagaccttcg tggtgcagct gcaggacccc cgggagcccc      300

gcaggcaggc cacccagatg ctgaagctgc agaacctggt gatcccctgg gcccctgaga      360

acctgacact gcacaagctg tccgagagcc agctggaact gaactggaac aaccgcttcc      420

tgaaccactg cctggaacac ctggtgcagt accggaccga ctgggaccac agctggaccg      480

agcagagcgt ggactaccgg cacaagttca gcctgcccag cgtggacggc cagaagcggt      540

acaccttcag agtgcggagc cggttcaacc ccctgtgcgg cagcgcccag cactggtccg      600

agtggagcca ccccatccac tggggcagca acaccagcaa agagaacccc ttcctgttcg      660

ccctggaagc cgtggtgatc agcgtgggca gcatgggcct gatcatctcc ctgctgtgcg      720

tgtacttctg gctggaacgg accatgccca gaatccccac cctgaagaac ctggaagatc      780

tggtgaccga gtaccacggc aacttcagcg cctggtccgg cgtgagcaag ggcctggccg      840

agagcctgca gcccgactac agcgagcggc tgtgcctggt gtccgagatc ccccccaaag      900

gcggagccct gggcgaaggc cctggcgcca gcccctgcaa ccagcacagc ccctactggg      960

cccctccttg ctacaccctg aagcccgaga cccgggccaa gcgatccgga tccggagcca     1020

ccaacttcag cctgctgaag caggccggcg acgtggagga gaaccccggc ccctga         1076


<210>  55
<211>  81
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Furin site + self-cleaving 2A peptide of IL2RG-targeting 
       constructs

<400>  55
cgggccaagc gatccggatc cggagccacc aacttcagcc tgctgaagca ggccggcgac       60

gtggaggaga accccggccc c                                                 81


<210>  56
<211>  267
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Right homology arm of IL2RG-targeting constructs

<400>  56
tacaatcatg tgggcagaat tgaaaagtgg agtgggaagg gcaaggggga gggttccctg       60

cctcacgcta cttcttcttt ctttcttgtt tgtttgtttc tttctttctt ttgaggcagg      120

gtctcactat gttgcctagg ctggtctcaa actcctggcc tctagtgatc ctcctgcctc      180

agcctttcaa agcaccagga ttacagacat gagccaccgt gcttggcctc ctccttctga      240

ccatcatttc tctttccctc cctgcct                                          267


<210>  57
<211>  516
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  PGK promoter of IL2RG-targeting constructs

<400>  57
ccacggggtt ggggttgcgc cttttccaag gcagccctgg gtttgcgcag ggacgcggct       60

gctctgggcg tggttccggg aaacgcagcg gcgccgaccc tgggtctcgc acattcttca      120

cgtccgttcg cagcgtcacc cggatcttcg ccgctaccct tgtgggcccc ccggcgacgc      180

ttcctgctcc gcccctaagt cgggaaggtt ccttgcggtt cgcggcgtgc cggacgtgac      240

aaacggaagc cgcacgtctc actagtaccc tcgcagacgg acagcgccag ggagcaatgg      300

cagcgcgccg accgcgatgg gctgtggcca atagcggctg ctcagcgggg cgcgccgaga      360

gcagcggccg ggaaggggcg gtgcgggagg cggggtgtgg ggcggtagtg tgggccctgt      420

tcctgcccgc gcggtgttcc gcattctgca agcctccgga gcgcacgtcg gcagtcggct      480

ccctcgttga ccgaatcacc gacctctctc cccagg                                516


<210>  58
<211>  1041
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  tTA and 3' UTR

<400>  58
atgtctagac tggacaagag caaagtcata aactctgctc tggaattact caatgaagtc       60

ggtatcgaag gcctgacgac aaggaaactc gctcaaaagc tgggagttga gcagcctacc      120

ctgtactggc acgtgaagaa caagcgggcc ctgctcgatg ccctggcaat cgagatgctg      180

gacaggcatc atacccactt ctgccccctg gaaggcgagt catggcaaga ctttctgcgg      240

aacaacgcca agtcattccg ctgtgctctc ctctcacatc gcgacggggc taaagtgcat      300

ctcggcaccc gcccaacaga gaaacagtac gaaaccctgg aaaatcagct cgcgttcctg      360

tgtcagcaag gcttctccct ggagaacgca ctgtacgctc tgtccgccgt gggccacttt      420

acactgggct gcgtattgga ggatcaggag catcaagtag caaaagagga aagagagaca      480

cctaccaccg attctatgcc cccacttctg agacaagcaa ttgagctgtt cgaccatcag      540

ggagccgaac ctgccttcct tttcggcctg gaactaatca tatgtggcct ggagaaacag      600

ctaaagtgcg aaagcggcgg gccggccgac gcccttgacg attttgactt agacatgctc      660

ccagccgatg cccttgacga ctttgacctt gatatgctgc ctgctgacgc tcttgacgat      720

tttgaccttg acatgctccc cgggtaaaag ctcgctttct tgctgtccaa tttctattaa      780

aggttccttt gttccctaag tccaactact aaactggggg atattatgaa gggccttgag      840

catctggatt ctgcctaata aaaaacattt attttcattg ctgcgctaga agctcgcttt      900

cttgctgtcc aatttctatt aaaggttcct ttgttcccta agtccaacta ctaaactggg      960

ggatattatg aagggccttg agcatctgga ttctgcctaa taaaaaacat ttattttcat     1020

tgctgcggga cattcttaat t                                               1041


<210>  59
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Tet operon (TetO) sequence

<400>  59
tccctatcag tgatagaga                                                    19


<210>  60
<211>  16
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  spacer sequence

<400>  60
acgatgtcga gtttac                                                       16


<210>  61
<211>  796
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Left homology arm of AAV6 AAVS1

<400>  61
tgctttctct gaccagcatt ctctcccctg ggcctgtgcc gctttctgtc tgcagcttgt       60

ggcctgggtc acctctacgg ctggcccaga tccttccctg ccgcctcctt caggttccgt      120

cttcctccac tccctcttcc ccttgctctc tgctgtgttg ctgcccaagg atgctctttc      180

cggagcactt ccttctcggc gctgcaccac gtgatgtcct ctgagcggat cctccccgtg      240

tctgggtcct ctccgggcat ctctcctccc tcacccaacc ccatgccgtc ttcactcgct      300

gggttccctt ttccttctcc ttctggggcc tgtgccatct ctcgtttctt aggatggcct      360

tctccgacgg atgtctccct tgcgtcccgc ctccccttct tgtaggcctg catcatcacc      420

gtttttctgg acaaccccaa agtaccccgt ctccctggct ttagccacct ctccatcctc      480

ttgctttctt tgcctggaca ccccgttctc ctgtggattc gggtcacctc tcactccttt      540

catttgggca gctcccctac cccccttacc tctctagtct gtgctagctc ttccagcccc      600

ctgtcatggc atcttccagg ggtccgagag ctcagctagt cttcttcctc caacccgggc      660

ccctatgtcc acttcaggac agcatgtttg ctgcctccag ggatcctgtg tccccgagct      720

gggaccacct tatattccca gggccggtta atgtggctct ggttctgggt acttttatct      780

gtcccctcca ccccac                                                      796


<210>  62
<211>  258
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Right homology arm of AAV6 AAVS1

<400>  62
tagggacagg attggtgaca gaaaagcccc atccttaggc ctcctccttc ctagtctcct       60

gatattgggt ctaaccccca cctcctgtta ggcagattcc ttatctggtg acacaccccc      120

atttcctgga gccatctctc tccttgccag aacctctaag gtttgcttac gatggagcca      180

gagaggatcc tgggagggag agcttggcag ggggtgggag ggaagggggg gatgcgtgac      240

ctgcccggtt ctcagtgg                                                    258


<210>  63
<211>  496
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Left homology arm of AAV6 CD40LG

<400>  63
tgctttaaaa gtaagctatt tttttatgga gacagctttt ttcttttaaa tttccagcta       60

ggcaagaaga gcgtcaattt gatctaaaat ttcataatgc ttcagattaa catagacatg      120

gataagtccc agaatttgca gtcttttagt aaaagtagca ttttctgtgt aattcttcac      180

aagcactgat tgtagttgca ggatgctcag tctccctctg agatgtttta catttttaaa      240

tggttagact tgcaggaaca aaagagcaga gtaacttagt aggctgtttt gcattcttag      300

gaaaagaaaa ccatcaggac ttattttgtt ttcatgtatt ttttcacttc cactgaggag      360

tataattggc tggtgttgac aaaataccaa tcatagatgt aaaggagaaa gttgattagt      420

tttctggctg ttcctaaaat tctggatgca ggaactgtgg ctagaaagca tctggatgat      480

tgcactttac cttagg                                                      496


<210>  64
<211>  499
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Right homology arm of AAV6 CD40LG

<400>  64
cagggatact tgagtgtcct ctcttaggat ctggacctag aattaatgtc atgagatttt       60

tctaacagga taaggtgagg tagtgagggc tgaagtcatc cactgggtta tccaaatatt      120

aggtttcact gctgacaaaa gagggggctt ctggtctggt tggttatttg tgtttggcct      180

gatgtgctct gtcaatcaaa tgtatggaca taggcctagc ttctaaaggg gcaatagtga      240

cctcagtgga ctgatattta ccgtactatt tacatgtgct cttaattaca gcagaagctg      300

ccagctaact gaatcttgtt ttgaatctaa aaaatctact cttaaagcaa gaaaatggta      360

taaaattagt tgataatgca agtgaattct gtacatttaa ttattctaag acattggaaa      420

ataaaatatc ttgttacttt gaggataaaa gatgatttct ttaaaaatgc aaatgttttc      480

tacaaatact aaagttaaa                                                   499


<210>  65
<211>  371
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TETO7-SK sequence

<400>  65
ccctatcagt gatagagaac gtatgaagag tttactccct atcagtgata gagaacgtat       60

gcagacttta ctccctatca gtgatagaga acgtataagg agtttactcc ctatcagtga      120

tagagaacgt atgaccagtt tactccctat cagtgataga gaacgtatct acagtttact      180

ccctatcagt gatagagaac gtatatccag tttactccct atcagtgata gagaacgtat      240

aagctttagg cgtgtacggt gggcgcctat aaaagcagag ctcgtttagt gaaccgtcag      300

atcgcctgga gcaattccac aacacttttg tcttatacca actttccgta ccacttccta      360

ccctcgtaaa g                                                           371


<210>  66
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Tet operon (TetO) sequence

<400>  66
ccctatcagt gatagaga                                                     18


<210>  67
<211>  843
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  delta LNGFR codon optimized for human expression

<400>  67
atgggagctg gtgctaccgg cagagctatg gatggaccta gactgctgct cctgctgctg       60

ctcggagttt ctcttggcgg agccaaagag gcctgtccta ccggcctgta tacacactct      120

ggcgagtgct gcaaggcctg caatcttgga gaaggcgtgg cacagccttg cggcgctaat      180

cagacagtgt gcgagccttg cctggacagc gtgaccttta gcgacgtggt gtctgccacc      240

gagccatgca agccttgtac cgagtgtgtg ggcctgcaga gcatgtctgc cccttgtgtg      300

gaagccgacg atgccgtgtg tagatgcgcc tacggctact accaggacga gacaacaggc      360

agatgcgagg cctgtagagt gtgtgaagcc ggctctggac tggtgttcag ctgccaagac      420

aagcagaaca ccgtgtgcga ggaatgcccc gatggcacct atagcgacga ggccaaccat      480

gtggatccct gcctgccttg tactgtgtgc gaagataccg agcggcagct gcgcgagtgt      540

acaagatggg ctgatgccga gtgcgaagag atccccggca gatggatcac cagaagcaca      600

cctccagagg gcagcgatag cacagcccct tctacacaag agcccgaggc tcctcctgag      660

caggatctga ttgcctctac agtggccggc gtggtcacaa cagtgatggg atcttctcag      720

cccgtggtca ccagaggcac caccgacaat ctgatccccg tgtactgtag catcctggcc      780

gccgtggttg tgggactcgt ggcctatatc gccttcaagc ggtggaaccg gggcatcctg      840

taa                                                                    843


<210>  68
<211>  1077
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CXCR4 WT sequence

<400>  68
gccaccatgt ctattcctct gcccctgctg cagatctaca ccagcgacaa ctacaccgag       60

gaaatgggca gcggcgacta cgacagcatg aaggaaccct gcttccggga agagaacgcc      120

aacttcaaca agatcttcct gcccacaatc tacagcatca tctttctgac cggcatcgtg      180

ggcaacggac tcgtgatcct cgtgatgggc taccagaaaa agctgcggag catgaccgac      240

aagtaccggc tgcacctgag cgtggccgac ctgctgttcg tgatcaccct gcctttctgg      300

gccgtggacg ccgtggccaa ttggtacttc ggcaacttcc tgtgcaaggc cgtgcacgtg      360

atctacacag tgaacctgta cagcagcgtg ctgatcctgg ccttcatcag cctggacaga      420

tacctggcca tcgtgcacgc caccaacagc cagcggccta gaaagctgct ggccgagaag      480

gtggtgtacg tgggcgtgtg gattcccgcc ctgctgctga ccatccccga cttcatcttc      540

gccaacgtgt ccgaggccga cgaccggtac atctgcgacc ggttctaccc caacgacctg      600

tgggtggtgg tgttccagtt ccagcacatc atggtgggac tgatcctgcc tggcatcgtg      660

attctgagct gctactgcat catcatcagc aagctgagcc acagcaaggg ccaccagaag      720

cggaaggccc tgaaaaccac cgtgatcctg attctggctt tcttcgcctg ctggctgccc      780

tactacatcg gcatcagcat cgacagcttc atcctgctgg aaatcatcaa gcagggctgc      840

gagttcgaga acaccgtgca caagtggatc agcattaccg aggccctggc ctttttccac      900

tgctgcctga accctatcct gtacgccttc ctgggcgcca agttcaagac ctctgcccag      960

cacgccctga ccagcgtgtc cagaggaagc agcctgaaga tcctgagcaa gggcaagaga     1020

ggcggccaca gctccgtgtc tacagagagc gagagcagca gcttccacag cagctga        1077


<210>  69
<211>  1077
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CXCR4 WHIM sequence

<400>  69
gccaccatgt ctattcctct gcccctgctg cagatctaca ccagcgacaa ctacaccgag       60

gaaatgggca gcggcgacta cgacagcatg aaggaaccct gcttccggga agagaacgcc      120

aacttcaaca agatcttcct gcccacaatc tacagcatca tctttctgac cggcatcgtg      180

ggcaacggac tcgtgatcct cgtgatgggc taccagaaaa agctgcggag catgaccgac      240

aagtaccggc tgcacctgag cgtggccgac ctgctgttcg tgatcaccct gcctttctgg      300

gccgtggacg ccgtggccaa ttggtacttc ggcaacttcc tgtgcaaggc cgtgcacgtg      360

atctacacag tgaacctgta cagcagcgtg ctgatcctgg ccttcatcag cctggacaga      420

tacctggcca tcgtgcacgc caccaacagc cagcggccta gaaagctgct ggccgagaag      480

gtggtgtacg tgggcgtgtg gattcccgcc ctgctgctga ccatccccga cttcatcttc      540

gccaacgtgt ccgaggccga cgaccggtac atctgcgacc ggttctaccc caacgacctg      600

tgggtggtgg tgttccagtt ccagcacatc atggtgggac tgatcctgcc tggcatcgtg      660

attctgagct gctactgcat catcatcagc aagctgagcc acagcaaggg ccaccagaag      720

cggaaggccc tgaaaaccac cgtgatcctg attctggctt tcttcgcctg ctggctgccc      780

tactacatcg gcatcagcat cgacagcttc atcctgctgg aaatcatcaa gcagggctgc      840

gagttcgaga acaccgtgca caagtggatc agcattaccg aggccctggc ctttttccac      900

tgctgcctga accctatcct gtacgccttc ctgggcgcca agttcaagac ctctgcccag      960

cacgccctga ccagcgtgtc cagaggaagc agcctgaaga tcctgagcaa gggcaagtga     1020

ggcggccaca gctccgtgtc tacagagagc gagagcagca gcttccacag cagctga        1077


<210>  70
<211>  744
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  reverse-tTA (rtTA)

<400>  70
atgtctagac tggacaagag caaagtcata aactctgctc tggaattact caatggagtc       60

ggtatcgaag gcctgacgac aaggaaactc gctcaaaagc tgggagttga gcagcctacc      120

ctgtactggc acgtgaagaa caagcgggcc ctgctcgatg ccctgccaat cgagatgctg      180

gacaggcatc atacccactt ctgccccctg gaaggcgagt catggcaaga ctttctgcgg      240

aacaacgcca agtcattccg ctgtgctctc ctctcacatc gcgacggggc taaagtgcat      300

ctcggcaccc gcccaacaga gaaacagtac gaaaccctgg aaaatcagct cgcgttcctg      360

tgtcagcaag gcttctccct ggagaacgca ctgtacgctc tgtccgccgt gggccacttt      420

acactgggct gcgtattgga ggaacaggag catcaagtag caaaagagga aagagagaca      480

cctaccaccg attctatgcc cccacttctg agacaagcaa ttgagctgtt cgaccggcag      540

ggagccgaac ctgccttcct tttcggcctg gaactaatca tatgtggcct ggagaaacag      600

ctaaagtgcg aaagcggcgg gccggccgac gcccttgacg attttgactt agacatgctc      660

ccagccgatg cccttgacga ctttgacctt gatatgctgc ctgctgacgc tcttgacgat      720

tttgaccttg acatgctccc cggg                                             744


<210>  71
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  genomic sequence, Guide ID AAVS1gRNA

<400>  71
gtcaccaatc ctgtccctag tgg                                               23


<210>  72
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  genomic sequence, Guide ID IL2RG gRNA

<400>  72
actggccatt acaatcatgt ggg                                               23


<210>  73
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  genomic sequence, Guide ID CD40LG gRNA

<400>  73
tggatgattg cactttatca ggg                                               23


<210>  74
<211>  618
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TetR sequence

<400>  74
atgtctagac tggacaagag caaagtcata aactctgctc tggaattact caatgaagtc       60

ggtatcgaag gcctgacgac aaggaaactc gctcaaaagc tgggagttga gcagcctacc      120

ctgtactggc acgtgaagaa caagcgggcc ctgctcgatg ccctggcaat cgagatgctg      180

gacaggcatc atacccactt ctgccccctg gaaggcgagt catggcaaga ctttctgcgg      240

aacaacgcca agtcattccg ctgtgctctc ctctcacatc gcgacggggc taaagtgcat      300

ctcggcaccc gcccaacaga gaaacagtac gaaaccctgg aaaatcagct cgcgttcctg      360

tgtcagcaag gcttctccct ggagaacgca ctgtacgctc tgtccgccgt gggccacttt      420

acactgggct gcgtattgga ggatcaggag catcaagtag caaaagagga aagagagaca      480

cctaccaccg attctatgcc cccacttctg agacaagcaa ttgagctgtt cgaccatcag      540

ggagccgaac ctgccttcct tttcggcctg gaactaatca tatgtggcct ggagaaacag      600

ctaaagtgcg aaagcggc                                                    618


<210>  75
<211>  36
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  VP16 sequence

<400>  75
gccgacgccc ttgacgattt tgacttagac atgctc                                 36


<210>  76
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  TetO sequence

<400>  76
ccctatcagt gatagaga                                                     18


<210>  77
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NHEJ AAVS1 FW primer

<400>  77
cttcaggaca gcatgtttgc                                                   20


<210>  78
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NHEJ AAVS1 RV primer

<400>  78
acaggaggtg ggggttagac                                                   20


<210>  79
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NHEJ Intron 1 IL2RG FW primer

<400>  79
caccctctgt aaagccctgg                                                   20


<210>  80
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  NHEJ Intron 1 IL2RG RV primer

<400>  80
aagaaatcta gattggggag                                                   20


<210>  81
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Intron 1 IL2RG 3' integration junction ddPCR FW primer

<400>  81
ctagattggg gagaaaatga                                                   20


<210>  82
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Intron 1 IL2RG 3' integration junction ddPCR RV primer

<400>  82
gtgggaaggg gccgtacag                                                    19


<210>  83
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Intron 1 IL2RG 3' integration junction ddPCR probe (FAM)

<400>  83
gtagctccta tgctaggcgt agcc                                              24


<210>  84
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAVS1 3' integration junction ddPCR FW (SK) primer

<400>  84
caccgtacac gcctaaagct a                                                 21


<210>  85
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAVS1 3' integration junction ddPCR FW (mCMV) primer

<400>  85
cttatatagg cctcccaccg t                                                 21


<210>  86
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAVS1 3' integration junction ddPCR RV primer

<400>  86
tcttgggaag tgtaaggaag                                                   20


<210>  87
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAVS1 3' integration junction ddPCR probe (FAM)

<400>  87
ccagataagg aatctgccta                                                   20


<210>  88
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAVS1 3' integration junction ddPCR FW primer

<400>  88
gattgggaag acaatagcag                                                   20


<210>  89
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CD40LG 3' integration junction ddPCR FW primer

<400>  89
ttaggagggg gtctgataca                                                   20


<210>  90
<211>  25
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CD40LG 3' integration junction ddPCR RV primer

<400>  90
tcctcgatct gtgggaggaa gagaa                                             25


<210>  91
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CD40LG 3' integration junction ddPCR Probe (FAM)

<400>  91
tcagtctccc tctgagatgt                                                   20


