                         SEQUENCE LISTING

<110>  uniQure IP B.V.
 
<120>  targeting misspliced transcripts in genetic disorders

<130>  p6093701pct

<150>  EP 19197533.3
<151>  2019-09-16

<160>  51    

<170>  PatentIn version 3.5

<210>  1
<211>  16
<212>  RNA
<213>  artificial sequence

<220>
<223>  flanking sequence miRNA

<400>  1
cuugggaaug gcaagg                                                       16


<210>  2
<211>  16
<212>  RNA
<213>  artificial sequence

<220>
<223>  flanking miRNA sequence

<400>  2
cucuugcuau cccaga                                                       16


<210>  3
<211>  7762
<212>  DNA
<213>  Homo sapiens

<400>  3
gctgccggga cgggtccaag atggacggcc gctcaggttc tgcttttacc tgcggcccag       60

agccccattc attgccccgg tgctgagcgg cgccgcgagt cggcccgagg cctccgggga      120

ctgccgtgcc gggcgggaga ccgccatggc gaccctggaa aagctgatga aggccttcga      180

gtccctcaag tccttccagc agcagcagca gcagcagcag cagcagcagc agcagcagca      240

gcagcagcag cagcaacagc cgccaccgcc gccgccgccg ccgccgcctc ctcagcttcc      300

tcagccgccg ccgcaggcac agccgctgct gcctcagccg cagccgcccc cgccgccgcc      360

cccgccgcca cccggcccgg ctgtggctga ggagccgctg caccgaccgt gagtttgggc      420

ccgctgcagc tccctgtccc ggcgggtccc aggctacggc ggggatggcg gtaaccctgc      480

agcctgcggg ccggcgacac gaacccccgg ccccgcagag acagagtgac ccagcaaccc      540

agagcccatg agggacaccc gccccctcct ggggcgaggc cttcccccac ttcagccccg      600

ctccctcact tgggtcttcc cttgtcctct cgcgagggga ggcagagcct tgttggggcc      660

tgtcctgaat tcaccgaggg gagtcacggc ctcagccctc tcgcccttcg caggatgcga      720

agagttgggg cgagaacttg tttcttttta tttgcgagaa accagggcgg gggttctttt      780

aactgcgttg tgaagagaac ttggaggagc cgagatttgc tcagtgccac ttccctcttc      840

tagtctgaga gggaagaggg ctgggggcgc gggacacttc gagaggaggc ggggtttgga      900

gctggagaga tgtgggggca gtggatgaca taatgctttt aggacgcctc ggcgggagtg      960

gcggggcagg gggggggcgg ggagtgaggg cgcgtccaat gggagatttc ttttcctagt     1020

ggcacttaaa acagcctgag atttgaggct cttcctacat tgtcaggaca tttcatttag     1080

ttcatgatca cggtggtagt aacacgattt taagcaccac ctaagagatc tgctcatcta     1140

agcctaagtt ggtctgcagg cgtttgaatg agttgtggtt gccaagtaaa gtggtgaact     1200

tacgtggtga ttaatgaaat tatcttaaat attaggaaga gttgattgaa gttttttgcc     1260

tatgtgtgtt gggaataaaa ccaacacgtt gctgatgggg aggttaattg ccgagggatg     1320

aatgaggtgt acattttacc agtattccag tcaggcttgc cagaatacgg ggggtccgca     1380

gactccgtgg gcatctcaga tgtgccagtg aaagggtttc tgtttgcttc attgctgaca     1440

gcttgttact ttttggaagc taggggtttc tgttgcttgt tcttggggag aatttttgaa     1500

acaggaaaag agagaccatt aaaacatcta gcggaacccc aggactttcc ctggaagtct     1560

gtgtgtcgag tgtacagtag gagttaggaa gtactctggt gcagttcagg cctttctctt     1620

acctctcagt attctatttc cgatctggat gtgtcccaga tggcatttgg taagaatatc     1680

tctgttaaga ctgattaatt tttagtaata tttcttgttc tttgtttctg ttatgatcct     1740

tgtctcgtct tcaaagttta attagaaaat gattcggaga gcagtgttag cttatttgtt     1800

ggaataaaat ttaggaataa attattctaa aggatggaaa aactttttgg atatttggag     1860

aaattttaaa acaatttggc ttatctcttc agtaagtaat ttctcatcca gaaatttact     1920

gtagtgcttt tctaggaggt aggtgtcata aaagttcaca cattgcatgt atcttgtgta     1980

aacactaaac agggctcctg atgggaagga agacctttct gctgggctgc ttcagacact     2040

tgatcattct aaaaatatgc cttctctttc ttatgctgat ttgacagaac ctgcatttgc     2100

ttatcttcaa aatatgggta tcaagaaatt tcctttgctg ccttgacaaa ggagatagat     2160

tttgtttcat tactttaagg taatatatga ttaccttatt taaaaaattt aatcaggact     2220

ggcaaggtgg cttacacctt taatccgagc actttgggag gcctaggtgg acgaatcacc     2280

tgaggtcagg agtttgagac cagcctggct aacatggtga aaccctgtct ctactaaaaa     2340

tacaaaaatt agctggtcat ggtggcacgt gcctgtaatc caagctacct gggaggctga     2400

ggcaggaaaa tcgcttgaac ccgggaggca gagtctgcag tgagttgaga tcacgccact     2460

gcactccagc ctgggtgaca gagcgagact ctatctcaaa aaaaattttt tttaatgtat     2520

tatttttgca taagtaatac attgacatga tacaaattct gtaattacaa aagggcaata     2580

attaaaatat cttccttcca cccctttcct ctgagtacct aactttgtcc ccaagaacaa     2640

gcactatttc agttcctcat gtatcctgcc agatataacc tgttcatatt gtaagataga     2700

tttaaaatgc tctaaaaaca aaagtagttt agaataatat atatctatat attttttgag     2760

atgtagtctc acattgtcac ccaggctgga gtgcagtgat acaatctcgg ctcactgcag     2820

tctctgcctc ccaggttcaa atgcttctcc tgcctcagcc ttctgagtag ctgggattac     2880

aggcgcccac caccatgtcc agctaatttt tgtattttta gtagagatgg ggtttcacca     2940

tgttggccag gctggtcttg aactcctgac cttgtgatct gtccacctcg gcctcccaaa     3000

gtgctgggat tacaggtgtg agccaccatg cctggctaga ataataactt ttaaaggttc     3060

ttagcatgct ctgaaatcaa ctgcattagg tttatttata gttttatagt tattttaaat     3120

aaaatgcata tttgtcatat ttctctgtat tttgctgttg agaaaggagg tattcactaa     3180

ttttgagtaa caaacactgc tcacaaagtt tggattttgg cagttctgtt cacgtgcttc     3240

agccaaaaaa tcctcttctc aaagtaagat tgatgaaagc aatttagaaa gtatctgttc     3300

tgtttttatg gctcttgctc tttggtgtgg aactgtggtg tcacgccatg catgggcctc     3360

agtttatgag tgtttgtgct ctgctcagca tacaggatgc aggagttcct tatggggctg     3420

gctgcaggct cagcaaatct agcatgcttg ggagggtcct cacagtaatt aggaggcaat     3480

taatacttgc ttctggcagt ttcttattct ccttcagatt cctatctggt gtttccctga     3540

ctttattcat tcatcagtaa atatttacta aacatgtact atgtgcctgg cactgttata     3600

ggtgcagggc tcagcagtga gcagacaaag ctctgccctc gtgaagcttt cattctaatg     3660

aaggacatag acagtaagca agatagataa gtaaaatata cagtacgtta atacgtggag     3720

gaacttcaaa gcagggaagg ggatagggaa atgtcagggt taatcgagtg ttaacttatt     3780

tttattttta aaaaaattgt taagggcttt ccagcaaaac ccagaaagcc tgctagacaa     3840

attccaaaag agctgtagca ctaagtgttg acatttttat tttattttgt tttgttttgt     3900

tttttttgag acagttcttg ctctatcagc caggctggag tgcactagtg tgatcttggc     3960

tcactgcaac ctctgcctct tgggttcaag tgattctcat gcctcagcct cctgtttagc     4020

tgggattata gacatgcact gccatgcctg ggtaattttt tttttttccc ccgagacgga     4080

gtcttgctct gtcgcccagg ctggagtgca gtggcgcgat ctcagctcac tgcaagctcc     4140

gcttcccgag ttcacgccat tctcctgcct cagtctccca agtagctggg actacaggcg     4200

cctgccacca cgtccagcta atttttttgt atttttaata gagacggggt ttcaccgtgt     4260

tagccaggat gatcttgatc tcctgacctc gtcatccgcc gaccttgtga tccgcccacc     4320

tcggcctccc aaagtgctgg gattacaggc atgagccact gtgcccggcc acgcctgggt     4380

aatttttgta tttttagtag agatggggtt ttgccatgat gagcaggctg gtctcgaact     4440

cccggcctca tgtgatctgc ctgccttggc ctcccaaagt gctaggatta caggcatgag     4500

ccaccatacc tggccagtgt tgatatttta aatacggtgt tcagggaagg tccactgaga     4560

agacagcttt tttttttttt ttttttgggg ttggggggca aggtcttgct ctttaaccca     4620

ggctggaatg cagtatcact atcgtagctc acttcagcct tgaactcctg ggctcaagtg     4680

atcctcccac ctcaacctca caatgtgttg ggactatagg tgtgagccat cacacctggc     4740

cagatgatgg cttttgagta aagacctcaa gcgagttaag agtctagtgt aagggtgtat     4800

gaagtagtgg tattccagat ggggggaaca ggtccaaaat cttcctgttt caggaatagc     4860

aaggatgtca ttttagttgg gtgaattgag tgagggggac atttgtagta agaagtaagg     4920

tccaagaggt caagggagtg ccatatcaga ccaatactac ttgccttgta gatggaataa     4980

agatattggc atttatgtga gtgagatggg atgtcactgg aggattagag cagaggagta     5040

gcatgatctg aatttcaatc ttaagtgaac tctggctgac aacagagtga aggggaacac     5100

cggcaaaagc agaaaccagt taggaagcca ctgcagtgct cagataagca tggtgggttc     5160

tgtcagggta ccggctgtcg gctgtgggca gtgtgaggaa tgactgactg gattttgaat     5220

gcggaaccaa ctgcacttgt tgaactctgc taagtataac aatttagcag tagcttgcgt     5280

tatcaggttt gtattcagct gcaagtaaca gaaaatcctg ctgcaatagc ttaaactggt     5340

aacaagcaag agcttatcag aagacaaaaa taagtctggg gaaattcaac aataagttaa     5400

ggaacccagg ctctttcttt tttttttttt tgaaacggag tttcgctctt gtcacccggg     5460

ctggagtgca atgatgtgat ctcagctcac taaaacctct acctcctggg ttcaagtgat     5520

tcttctgcct cagcctccca agtaactggg attacaggcg tataccacca tgcccagcta     5580

atttttgtgt ttttagtaga gatggggttt caccatgttg gccaggctgg tctcgaactt     5640

ctgacctcag gtgatccact cgcctcagcc tgccaaagtg ctgggattac aggtttgggc     5700

cactgcaccc ggtcagaacc caggctcttt cttatactta ccttgcaaac ccttgttctc     5760

attttttccc tttgtatttt tattgttgaa ttgtaatagt tctttatata ttctggatac     5820

tggattctta tcagatagat gatttgtaaa aactctccct tcctttggat tgtcttttta     5880

ctttcttgat agtgtctttt gaagtgtaaa agtttttaat tttgatgaag tcgagtttat     5940

ctattttgtc tttggttgct gtgcttcaag tgtcatatct aagaaatcat tgtctaatcc     6000

aaagtcaaaa aggtttactc ctatgttttc ttctaagaat tttagagttt tacatttaag     6060

tctgatccat tttgagttaa tttttatata tggttcaggt agaagtccaa ctttattctt     6120

ttccatgtgg ttattcagtt gtcccagcac tgtttgttga agagactatt ctttccccat     6180

ggaattatct tagtaccctt gttgaaaatt aatcgtcctt aattgtataa atttatttct     6240

agactgtcag ttctacctgt tggtctttat gtcgatcctg tgccagtacc atacagtctt     6300

gattactgaa gtttgtgtca cagtttaaat tcatgaaatg tgagttctcc aactttgttc     6360

cttttcaaga ttgatttggc catgctgggt cccttgcatt tccgtacgaa ttgtaggatc     6420

agcttgtcag tttcaacaaa gaagccaagt aggattctga gagggattgt gttgaatctg     6480

tagatcaact tggggagtat tcgcatctta acaatattgt cttccaccta tgaacatggg     6540

caaactttgt gtaaatggtc agattgtaag tatttcgggc tgtgtgggca cagtgtctct     6600

gtcacagcta cgcggctctg ccattgtagc atgaaagtag ccataagcaa tatgtatgag     6660

tgtctgtgtt ccaatagaat tttattaatg acaaggaagt ttgaatttca tataattttc     6720

acctgtcatg agatagtatt tgattatttt ggtcaaccat ttaaaaatgt aaaaacattt     6780

cttagcttgt gaactagcca aaaatatgca ggttatagtt ttcccactcc taggttaaaa     6840

tatgatagga ccacatttgg aaagcatttc tttttttttt tttttttttt tttttgagac     6900

ggagtttcac tcttgttgcc caggctggag tgcagtggcg cgatctcggc tcactgcaac     6960

ctctgcctcc caggttcaag acattctcct gcacggcctc cctagtagct gggattacag     7020

gcatgcgcca ccacacccag ctaattttgt atttttagta gagacggggt ttctccatgt     7080

tggtcaggct ggtcttgaac tcctgacctc aggtgatcca cccgcctcag cctcccaaag     7140

tgctgggatt acagggtgtg agccaccaca ccctgctgga aagcatttct tttttggctg     7200

tttttgtttt ttttttaaac tagttttgaa aattataaaa gttacacata tacattataa     7260

aaatatcttc aagcagcaca gatgaaaaac aaagcccttc ttgcaagtct gtcatctttg     7320

tctaacttcc taagaacaaa agtgtttctt gtgtcttctt cccagatttt aatatgcata     7380

tacaagcatt taaatgtgtc attttttgtt tgcttgactg agatcacatt acatatgtat     7440

ttttttactt aacaatgtgt catagatatt gttccatagc agtacctgta attcttatta     7500

attgctatgt aatattttag aatttctttt taaaagagga cttttggaga tgtaaaggca     7560

aaggtctcac atttttgtgg ctgtagaatg tgctggtgac atattctctc taccttgaga     7620

agtccccatc cccatcacct ccatttcctg taaataagtc aaccacttga taaactacct     7680

ttgaatggat ccacactcaa aacatttagt cttattcaga caacaaggag gaaaaataaa     7740

ataccttata aagcaaaaaa aa                                              7762


<210>  4
<211>  196
<212>  RNA
<213>  homo sapiens

<400>  4
gcugccggga cggguccaag auggacggcc gcucagguuc ugcuuuuacc ugcggcccag       60

agccccauuc auugccccgg ugcugagcgg cgccgcgagu cggcccgagg ccuccgggga      120

cugccgugcc gggcgggaga ccgccauggc gacccuggaa aagcugauga aggccuucga      180

gucccucaag uccuuc                                                      196


<210>  5
<211>  23
<212>  RNA
<213>  homo sapiens

<400>  5
gagaccgcca uggcgacccu gga                                               23


<210>  6
<211>  23
<212>  RNA
<213>  homo sapiens

<400>  6
agaccgccau ggcgacccug gaa                                               23


<210>  7
<211>  24
<212>  RNA
<213>  homo sapiens

<400>  7
gaugaaggcc uucgaguccc ucaa                                              24


<210>  8
<211>  24
<212>  RNA
<213>  homo sapiens

<400>  8
ggccuucgag ucccucaagu ccuu                                              24


<210>  9
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting HTT mRNA

<400>  9
aaggacuuga gggacucgaa ga                                                22


<210>  10
<211>  21
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting HTT mRNA

<400>  10
aaggacuuga gggacucgaa g                                                 21


<210>  11
<211>  88
<212>  PRT
<213>  homo sapiens

<400>  11

Met Ala Thr Leu Glu Lys Leu Met Lys Ala Phe Glu Ser Leu Lys Ser 
1               5                   10                  15      


Phe Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln 
            20                  25                  30          


Gln Gln Gln Gln Gln Gln Pro Pro Pro Pro Pro Pro Pro Pro Pro Pro 
        35                  40                  45              


Pro Gln Leu Pro Gln Pro Pro Pro Gln Ala Gln Pro Leu Leu Pro Gln 
    50                  55                  60                  


Pro Gln Pro Pro Pro Pro Pro Pro Pro Pro Pro Pro Gly Pro Ala Val 
65                  70                  75                  80  


Ala Glu Glu Pro Leu His Arg Pro 
                85              


<210>  12
<211>  119
<212>  DNA
<213>  homo sapiens

<400>  12
acagcagcaa aagcagcaac agcagcagca gcagcagcag cagggggacc tatcaggaca       60

gagttcacat ccatgtgaaa ggccagccac cagttcagga gcacttggga gtgatctag       119


<210>  13
<211>  95
<212>  DNA
<213>  homo sapiens

<400>  13
gtgatgctat gagtgaagaa gacatgcttc aggcagctgt gaccatgtct ttagaaactg       60

tcagaaatga tttgaaaaca gaaggaaaaa aataa                                  95


<210>  14
<211>  56
<212>  PRT
<213>  homo sapiens

<400>  14

Gly Asp Leu Ser Gly Gln Ser Ser His Pro Cys Glu Arg Pro Ala Thr 
1               5                   10                  15      


Ser Ser Gly Ala Leu Gly Ser Asp Leu Gly Asp Ala Met Ser Glu Glu 
            20                  25                  30          


Asp Met Leu Gln Ala Ala Val Thr Met Ser Leu Glu Thr Val Arg Asn 
        35                  40                  45              


Asp Leu Lys Thr Glu Gly Lys Lys 
    50                  55      


<210>  15
<211>  190
<212>  DNA
<213>  homo sapiens

<400>  15
gtaaggcctg ctcaccattc atcatgttcg ctaccttcac actttatctg acatacgagc       60

tccatgtgat ttttgcttta cattattctt cattccctct ttaatcatat taagaatctt      120

aagtaaattt gtaatctact aaatttccct ggattaagga gcagttacca aaagaaaaaa      180

aaaaaaaaaa                                                             190


<210>  16
<211>  59
<212>  PRT
<213>  homo sapiens

<400>  16

Gly Asp Leu Ser Gly Gln Ser Ser His Pro Cys Glu Arg Pro Ala Thr 
1               5                   10                  15      


Ser Ser Gly Ala Leu Gly Ser Asp Leu Gly Lys Ala Cys Ser Pro Phe 
            20                  25                  30          


Ile Met Phe Ala Thr Phe Thr Leu Tyr Leu Thr Tyr Glu Leu His Val 
        35                  40                  45              


Ile Phe Ala Leu His Tyr Ser Ser Phe Pro Leu 
    50                  55                  


<210>  17
<211>  22
<212>  RNA
<213>  homo sapiens

<400>  17
aacacugguu uacaguuaga aa                                                22


<210>  18
<211>  22
<212>  RNA
<213>  homo sapiens

<400>  18
aauuaggaaa acagugguuu aa                                                22


<210>  19
<211>  22
<212>  RNA
<213>  homo sapiens

<400>  19
aaguaugcaa gguaguucca ga                                                22


<210>  20
<211>  22
<212>  RNA
<213>  homo sapiens

<400>  20
uacuucagaa gagcuucgga ag                                                22


<210>  21
<211>  22
<212>  RNA
<213>  homo sapiens

<400>  21
gagacgagaa gccuacuuug aa                                                22


<210>  22
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting ATXN3 mRNA

<400>  22
uuucuaacug uaaaccagug uu                                                22


<210>  23
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting ATXN3 mRNA

<400>  23
uuaaaccacu guuuuccuaa uu                                                22


<210>  24
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting ATXN3 mRNA

<400>  24
ucuggaacua ccuugcauac uu                                                22


<210>  25
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting ATXN3 mRNA

<400>  25
cuuccgaagc ucuucugaag ua                                                22


<210>  26
<211>  22
<212>  RNA
<213>  artificial sequence

<220>
<223>  polynucleotide sequence targeting ATXN3 mRNA

<400>  26
uucaaaguag gcuucucguc uc                                                22


<210>  27
<211>  17
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer -19 fw

<400>  27
aggaaccgct gcaccga                                                      17


<210>  28
<211>  27
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 431 rv

<400>  28
gagacctcct aaaagcatta tgtcatc                                           27


<210>  29
<211>  22
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer ex2 fw

<400>  29
aagaaggaac tctcagccac ca                                                22


<210>  30
<211>  23
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer ex2 rv

<400>  30
ctgagagact gtgccacaat gtt                                               23


<210>  31
<211>  23
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 347 fw

<400>  31
tcctcatcag gcctaagagc tgg                                               23


<210>  32
<211>  22
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 785 rv

<400>  32
tgaaaactga gcaccaccaa tg                                                22


<210>  33
<211>  18
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 5'UTR fw

<400>  33
cttggttccg cttctgcc                                                     18


<210>  34
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 5'UTR rv

<400>  34
tggagcctac tggcactacg                                                   20


<210>  35
<211>  24
<212>  DNA
<213>  artificial sequence

<220>
<223>  5'UTR p

<400>  35
cagagcccca ttcattgcct tgct                                              24


<210>  36
<211>  17
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 135 fw

<400>  36
cttgcggggt ctctggc                                                      17


<210>  37
<211>  18
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 200 rv

<400>  37
tcagcgagtc cctggctg                                                     18


<210>  38
<211>  25
<212>  DNA
<213>  artificial sequence

<220>
<223>  155 p

<400>  38
cctcagagga gacagagccg ggtca                                             25


<210>  39
<211>  27
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 431 rv

<400>  39
gagacctcct aaaagcatta tgtcatc                                           27


<210>  40
<211>  25
<212>  DNA
<213>  artificial sequence

<220>
<223>  371 p

<400>  40
agtgcaggac agcgtgagag atgtg                                             25


<210>  41
<211>  37
<212>  DNA
<213>  artificial sequence

<220>
<223>  Ex2 p

<400>  41
agaaagaccg tgtgaatcat tgtctaacaa tatgtga                                37


<210>  42
<211>  52
<212>  DNA
<213>  artificial sequence

<220>
<223>  QT 3'RACE

<400>  42
ccagtgagca gagtgacgag gactcgagct caagcttttt tttttttttt tt               52


<210>  43
<211>  18
<212>  DNA
<213>  artificial sequence

<220>
<223>  Q0

<400>  43
ccagtgagca gagtgacg                                                     18


<210>  44
<211>  18
<212>  DNA
<213>  artificial sequence

<220>
<223>  Qi

<400>  44
gaggactcga gctcaagc                                                     18


<210>  45
<211>  24
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 571 fw

<400>  45
aaccaggttt taagcatagc caga                                              24


<210>  46
<211>  30
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer 622 fw

<400>  46
agttggatga gttgtatttg tcaagtacat                                        30


<210>  47
<211>  513
<212>  DNA
<213>  Mus musculus


<220>
<221>  misc_feature
<222>  (4)..(6)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (297)..(297)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (383)..(383)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (509)..(509)
<223>  n is a, c, g, or t

<400>  47
cgtnnnattt cttaggtgtg attattaata aaaaactata tgtgtgcata tatatgaaag       60

agtcgactta tacttaactg cctatcgatt ttttgttcta tataaaacgg atacattggt      120

ggtgctcagt tttcaccggg gaatgaattt tactagtgtt gcagacaggc ttgttttaga      180

acataggcca ctctgactct gactttgtgc cagtaaaagt tcctgtttag ttctttgctg      240

acatcttata gatctttgga agctagctgc ttgtgactgg agagaatatt gaaacanaag      300

agagaccatg agtcacagtg ctctaagaga aaagagacgc tcaaaacatt tcctggaaat      360

ccatgctgag tgttgagccc tgngctctct tgcagctcag tcctttctct caactctggg      420

cattttattt ctaatctgga tttgtataat taataaggag aacttttggg aacaacctac      480

taaagaatgt catcattaaa actcattana atc                                   513


<210>  48
<211>  79
<212>  DNA
<213>  mus musculus


<220>
<221>  misc_feature
<222>  (5)..(5)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (7)..(7)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (9)..(9)
<223>  n is a, c, g, or t

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  n is a, c, g, or t

<400>  48
ggtgnantnt attangtgtg attattataa aaaactatat gtgtgcatat aaaaaaaaaa       60

aaaaaaaaaa aaaaaaaaa                                                    79


<210>  49
<211>  319
<212>  DNA
<213>  mus musculus


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

<220>
<221>  misc_feature
<222>  (8)..(8)
<223>  n is a, c, g, or t

<400>  49
gccncccngt gagcaggctt tccggcccgg gccctcgtct tgcggggtct ctggcctccc       60

tcagaggaga cagagccggg tcaggccagc cagggactcg ctgaggggcg tcacgactcc      120

agtgccttcg ccgttcccag tttgcgaagt tagggaacga acttgtttct ctcttctgga      180

gaaactgggg cggtggcgca catgactgtt gtgaagagaa cttggagagg cagagatctc      240

tagggttacc tcctcatcag gcctaagagc tgggagtgca ggacagcgtg agagatgtgc      300

gggtagtgga tgacataat                                                   319


<210>  50
<211>  22
<212>  DNA
<213>  artificial sequence

<220>
<223>  DNA sequence corresponding with sequence targeting HTT mRNA

<400>  50
aaggacttga gggactcgaa ga                                                22


<210>  51
<211>  50
<212>  PRT
<213>  Homo Sapiens

<400>  51

Pro Pro Pro Pro Pro Pro Pro Pro Pro Pro Pro Gln Leu Pro Gln Pro 
1               5                   10                  15      


Pro Pro Gln Ala Gln Pro Leu Leu Pro Gln Pro Gln Pro Pro Pro Pro 
            20                  25                  30          


Pro Pro Pro Pro Pro Pro Gly Pro Ala Val Ala Glu Glu Pro Leu His 
        35                  40                  45              


Arg Pro 
    50  


