                         SEQUENCE LISTING

<110>  University of Sheffield
 
<120>  4792P/WO

<130>  Gene Therapy

<150>  GB2001930.3
<151>  2020-02-12

<160>  8     

<170>  PatentIn version 3.5

<210>  1
<211>  1371
<212>  DNA
<213>  Homo Sapiens

<400>  1
atgaagattg aggaggtgaa gagcactacg aagacgcagc gcatcgcctc ccacagccac       60

gtgaaagggc tggggctgga cgagagcggc ttggccaagc aggcggcctc agggcttgtg      120

ggccaggaga acgcgcgaga ggcatgtggc gtcatagtag aattaatcaa aagcaagaaa      180

atggctggaa gagctgtctt gttggcagga cctcctggaa ctggcaagac agctctggct      240

ctggctattg ctcaggagct gggtagtaag gtccccttct gcccaatggt ggggagtgaa      300

gtttactcaa ctgagatcaa gaagacagag gtgctgatgg agaacttccg cagggccatt      360

gggctgcgaa taaaggagac caaggaagtt tatgaaggtg aagtcacaga gctaactccg      420

tgtgagacag agaatcccat gggaggatat ggcaaaacca ttagccatgt gatcatagga      480

ctcaaaacag ccaaaggaac caaacagttg aaactggacc ccagcatttt tgaaagtttg      540

cagaaagagc gagtagaagc tggagatgtg atttacattg aagccaacag tggggccgtg      600

aagaggcagg gcaggtgtga tacctatgcc acagaattcg accttgaagc tgaagagtat      660

gtccccttgc caaaagggga tgtgcacaaa aagaaagaaa tcatccaaga tgtgaccttg      720

catgacttgg atgtggctaa tgcgcggccc caggggggac aagatatcct gtccatgatg      780

ggccagctaa tgaagccaaa gaagacagaa atcacagaca aacttcgagg ggagattaat      840

aaggtggtga acaagtacat cgaccagggc attgctgagc tggtcccggg tgtgctgttt      900

gttgatgagg tccacatgct ggacattgag tgcttcacct acctgcaccg cgccctggag      960

tcttctatcg ctcccatcgt catctttgca tccaaccgag gcaactgtgt catcagaggc     1020

actgaggaca tcacatcccc tcacggcatc cctcttgacc ttctggaccg agtgatgata     1080

atccggacca tgctgtatac tccacaggaa atgaaacaga tcattaaaat ccgtgcccag     1140

acggaaggaa tcaacatcag tgaggaggca ctgaaccacc tgggggagat tggcaccaag     1200

accacactga ggtactcagt gcagctgctg accccggcca acttgcttgc taaaatcaac     1260

gggaaggaca gcattgagaa agagcatgtc gaagagatca gtgaactttt ctatgatgcc     1320

aagtcctccg ccaaaatcct ggctgaccag caggataagt acatgaagta a              1371


<210>  2
<211>  1392
<212>  DNA
<213>  Homo Sapiens

<400>  2
atggcaaccg ttacagccac aaccaaagtc ccggagatcc gtgatgtaac aaggattgag       60

cgaatcggtg cccactccca catccgggga ctggggctgg acgatgcctt ggagcctcgg      120

caggcttcgc aaggcatggt gggtcagctg gcggcacggc gggcggctgg cgtggtgctg      180

gagatgatcc gggaagggaa gattgccggt cgggcagtcc ttattgctgg ccagccgggc      240

acggggaaga cggccatcgc catgggcatg gcgcaggccc tgggccctga cacgccattc      300

acagccatcg ccggcagtga aatcttctcc ctggagatga gcaagaccga ggcgctgacg      360

caggccttcc ggcggtccat cggcgttcgc atcaaggagg agacggagat catcgaaggg      420

gaggtggtgg agatccagat tgatcgacca gcaacaggga cgggctccaa ggtgggcaaa      480

ctgaccctca agaccacaga gatggagacc atctacgacc tgggcaccaa gatgattgag      540

tccctgacca aggacaaggt ccaggccggg gacgtgatca ccatcgacaa ggcgacgggc      600

aagatctcca agctgggccg ctccttcaca cgcgcccgcg actacgacgc tatgggctcc      660

cagaccaagt tcgtgcagtg cccagatggg gagctccaga aacgcaagga ggtggtgcac      720

accgtgtccc tgcacgagat cgacgtcatc aactctcgca cccagggctt cctggcgctc      780

ttctcaggtg acacagggga gatcaagtca gaagtccgtg agcagatcaa tgccaaggtg      840

gctgagtggc gcgaggaggg caaggcggag atcatccctg gagtgctgtt catcgacgag      900

gtccacatgc tggacatcga gagcttctcc ttcctcaacc gggccctgga gagtgacatg      960

gcgcctgtcc tgatcatggc caccaaccgt ggcatcacgc gaatccgggg caccagctac     1020

cagagccctc acggcatccc catagacctg ctggaccggc tgcttatcgt ctccaccacc     1080

ccctacagcg agaaagacac gaagcagatc ctccgcatcc ggtgcgagga agaagatgtg     1140

gagatgagtg aggacgccta cacggtgctg acccgcatcg ggctggagac gtcactgcgc     1200

tacgccatcc agctcatcac agctgccagc ttggtgtgcc ggaaacgcaa gggtacagaa     1260

gtgcaggtgg atgacatcaa gcgggtctac tcactcttcc tggacgagtc ccgctccacg     1320

cagtacatga aggagtacca ggacgccttc ctcttcaacg aactcaaagg cgagaccatg     1380

gacacctcct aa                                                         1392


<210>  3
<211>  456
<212>  PRT
<213>  Homo Sapiens

<400>  3

Met Lys Ile Glu Glu Val Lys Ser Thr Thr Lys Thr Gln Arg Ile Ala 
1               5                   10                  15      


Ser His Ser His Val Lys Gly Leu Gly Leu Asp Glu Ser Gly Leu Ala 
            20                  25                  30          


Lys Gln Ala Ala Ser Gly Leu Val Gly Gln Glu Asn Ala Arg Glu Ala 
        35                  40                  45              


Cys Gly Val Ile Val Glu Leu Ile Lys Ser Lys Lys Met Ala Gly Arg 
    50                  55                  60                  


Ala Val Leu Leu Ala Gly Pro Pro Gly Thr Gly Lys Thr Ala Leu Ala 
65                  70                  75                  80  


Leu Ala Ile Ala Gln Glu Leu Gly Ser Lys Val Pro Phe Cys Pro Met 
                85                  90                  95      


Val Gly Ser Glu Val Tyr Ser Thr Glu Ile Lys Lys Thr Glu Val Leu 
            100                 105                 110         


Met Glu Asn Phe Arg Arg Ala Ile Gly Leu Arg Ile Lys Glu Thr Lys 
        115                 120                 125             


Glu Val Tyr Glu Gly Glu Val Thr Glu Leu Thr Pro Cys Glu Thr Glu 
    130                 135                 140                 


Asn Pro Met Gly Gly Tyr Gly Lys Thr Ile Ser His Val Ile Ile Gly 
145                 150                 155                 160 


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


Phe Glu Ser Leu Gln Lys Glu Arg Val Glu Ala Gly Asp Val Ile Tyr 
            180                 185                 190         


Ile Glu Ala Asn Ser Gly Ala Val Lys Arg Gln Gly Arg Cys Asp Thr 
        195                 200                 205             


Tyr Ala Thr Glu Phe Asp Leu Glu Ala Glu Glu Tyr Val Pro Leu Pro 
    210                 215                 220                 


Lys Gly Asp Val His Lys Lys Lys Glu Ile Ile Gln Asp Val Thr Leu 
225                 230                 235                 240 


His Asp Leu Asp Val Ala Asn Ala Arg Pro Gln Gly Gly Gln Asp Ile 
                245                 250                 255     


Leu Ser Met Met Gly Gln Leu Met Lys Pro Lys Lys Thr Glu Ile Thr 
            260                 265                 270         


Asp Lys Leu Arg Gly Glu Ile Asn Lys Val Val Asn Lys Tyr Ile Asp 
        275                 280                 285             


Gln Gly Ile Ala Glu Leu Val Pro Gly Val Leu Phe Val Asp Glu Val 
    290                 295                 300                 


His Met Leu Asp Ile Glu Cys Phe Thr Tyr Leu His Arg Ala Leu Glu 
305                 310                 315                 320 


Ser Ser Ile Ala Pro Ile Val Ile Phe Ala Ser Asn Arg Gly Asn Cys 
                325                 330                 335     


Val Ile Arg Gly Thr Glu Asp Ile Thr Ser Pro His Gly Ile Pro Leu 
            340                 345                 350         


Asp Leu Leu Asp Arg Val Met Ile Ile Arg Thr Met Leu Tyr Thr Pro 
        355                 360                 365             


Gln Glu Met Lys Gln Ile Ile Lys Ile Arg Ala Gln Thr Glu Gly Ile 
    370                 375                 380                 


Asn Ile Ser Glu Glu Ala Leu Asn His Leu Gly Glu Ile Gly Thr Lys 
385                 390                 395                 400 


Thr Thr Leu Arg Tyr Ser Val Gln Leu Leu Thr Pro Ala Asn Leu Leu 
                405                 410                 415     


Ala Lys Ile Asn Gly Lys Asp Ser Ile Glu Lys Glu His Val Glu Glu 
            420                 425                 430         


Ile Ser Glu Leu Phe Tyr Asp Ala Lys Ser Ser Ala Lys Ile Leu Ala 
        435                 440                 445             


Asp Gln Gln Asp Lys Tyr Met Lys 
    450                 455     


<210>  4
<211>  463
<212>  PRT
<213>  Homo Sapiens

<400>  4

Met Ala Thr Val Thr Ala Thr Thr Lys Val Pro Glu Ile Arg Asp Val 
1               5                   10                  15      


Thr Arg Ile Glu Arg Ile Gly Ala His Ser His Ile Arg Gly Leu Gly 
            20                  25                  30          


Leu Asp Asp Ala Leu Glu Pro Arg Gln Ala Ser Gln Gly Met Val Gly 
        35                  40                  45              


Gln Leu Ala Ala Arg Arg Ala Ala Gly Val Val Leu Glu Met Ile Arg 
    50                  55                  60                  


Glu Gly Lys Ile Ala Gly Arg Ala Val Leu Ile Ala Gly Gln Pro Gly 
65                  70                  75                  80  


Thr Gly Lys Thr Ala Ile Ala Met Gly Met Ala Gln Ala Leu Gly Pro 
                85                  90                  95      


Asp Thr Pro Phe Thr Ala Ile Ala Gly Ser Glu Ile Phe Ser Leu Glu 
            100                 105                 110         


Met Ser Lys Thr Glu Ala Leu Thr Gln Ala Phe Arg Arg Ser Ile Gly 
        115                 120                 125             


Val Arg Ile Lys Glu Glu Thr Glu Ile Ile Glu Gly Glu Val Val Glu 
    130                 135                 140                 


Ile Gln Ile Asp Arg Pro Ala Thr Gly Thr Gly Ser Lys Val Gly Lys 
145                 150                 155                 160 


Leu Thr Leu Lys Thr Thr Glu Met Glu Thr Ile Tyr Asp Leu Gly Thr 
                165                 170                 175     


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


Ile Thr Ile Asp Lys Ala Thr Gly Lys Ile Ser Lys Leu Gly Arg Ser 
        195                 200                 205             


Phe Thr Arg Ala Arg Asp Tyr Asp Ala Met Gly Ser Gln Thr Lys Phe 
    210                 215                 220                 


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


Thr Val Ser Leu His Glu Ile Asp Val Ile Asn Ser Arg Thr Gln Gly 
                245                 250                 255     


Phe Leu Ala Leu Phe Ser Gly Asp Thr Gly Glu Ile Lys Ser Glu Val 
            260                 265                 270         


Arg Glu Gln Ile Asn Ala Lys Val Ala Glu Trp Arg Glu Glu Gly Lys 
        275                 280                 285             


Ala Glu Ile Ile Pro Gly Val Leu Phe Ile Asp Glu Val His Met Leu 
    290                 295                 300                 


Asp Ile Glu Ser Phe Ser Phe Leu Asn Arg Ala Leu Glu Ser Asp Met 
305                 310                 315                 320 


Ala Pro Val Leu Ile Met Ala Thr Asn Arg Gly Ile Thr Arg Ile Arg 
                325                 330                 335     


Gly Thr Ser Tyr Gln Ser Pro His Gly Ile Pro Ile Asp Leu Leu Asp 
            340                 345                 350         


Arg Leu Leu Ile Val Ser Thr Thr Pro Tyr Ser Glu Lys Asp Thr Lys 
        355                 360                 365             


Gln Ile Leu Arg Ile Arg Cys Glu Glu Glu Asp Val Glu Met Ser Glu 
    370                 375                 380                 


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


Tyr Ala Ile Gln Leu Ile Thr Ala Ala Ser Leu Val Cys Arg Lys Arg 
                405                 410                 415     


Lys Gly Thr Glu Val Gln Val Asp Asp Ile Lys Arg Val Tyr Ser Leu 
            420                 425                 430         


Phe Leu Asp Glu Ser Arg Ser Thr Gln Tyr Met Lys Glu Tyr Gln Asp 
        435                 440                 445             


Ala Phe Leu Phe Asn Glu Leu Lys Gly Glu Thr Met Asp Thr Ser 
    450                 455                 460             


<210>  5
<211>  6
<212>  PRT
<213>  artificial sequence

<220>
<223>  repeat expansion

<400>  5

Gly Gly Gly Gly Cys Cys 
1               5       


<210>  6
<211>  45
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer

<400>  6
actctagagg taccacgtga tcattctcga gggtgctatc caggc                       45


<210>  7
<211>  45
<212>  DNA
<213>  artificial sequence

<220>
<223>  primer

<400>  7
gcctggatag caccctcgag aatgatcacg tggtacctct agagt                       45


<210>  8
<211>  164
<212>  DNA
<213>  artificial sequence

<220>
<223>  promoter sequence

<400>  8
gggcggagtt agggcggagc caatcagcgt gcgccgttcc gaaagttgcc ttttatggct       60

gggcggagaa tgggcggtga acgccgatga ttatataagg acgcgccggg tgtggcacag      120

ctagttccgt cgcagccggg atttgggtcg cggttcttgt ttgt                       164


