                         SEQUENCE LISTING

<110>  Benitec Biopharma Limited
 
<120>  Reagents for treatment of Oculopharyngeal Muscular Dystrophy 
       (OPMD) and use thereof

<130>  177475

<150>  US 62/322,745
<151>  2016-04-14

<160>  29    

<170>  PatentIn version 3.5

<210>  1
<211>  23
<212>  RNA
<213>  Homo sapiens

<400>  1
uugaggagaa gauggaggcu gau                                               23


<210>  2
<211>  21
<212>  RNA
<213>  Homo sapiens

<400>  2
aggaagaagc ugagaagcua a                                                 21


<210>  3
<211>  27
<212>  RNA
<213>  Homo sapiens

<400>  3
gagguagaga agcagaugaa uaugagu                                           27


<210>  4
<211>  23
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for ssRNA1 and dsRNA1

<400>  4
aucagccucc aucuucuccu caa                                               23


<210>  5
<211>  23
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for dsRNA1

<400>  5
uugaggagaa gauggaggcu gau                                               23


<210>  6
<211>  21
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for ssRNA2 and dsRNA2

<400>  6
uuagcuucuc agcuucuucc u                                                 21


<210>  7
<211>  21
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for dsRNA2

<400>  7
aggaagaagc ugagaagcua a                                                 21


<210>  8
<211>  27
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for ssRNA3 and dsRNA3

<400>  8
acucauauuc aucugcuucu cuaccuc                                           27


<210>  9
<211>  27
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for dsRNA3

<400>  9
gagguagaga agcagaugaa uaugagu                                           27


<210>  10
<211>  23
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for shRNA1 and shRNA2

<400>  10
aucagccucc aucuucuccu caa                                               23


<210>  11
<211>  23
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for shRNA1 and shRNA2

<400>  11
uugaggagaa gauggaggcu gau                                               23


<210>  12
<211>  21
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for shRNA3 and shRNA4

<400>  12
uuagcuucuc agcuucuucc u                                                 21


<210>  13
<211>  21
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for shRNA3 and shRNA4

<400>  13
aggaagaagc ugagaagcua a                                                 21


<210>  14
<211>  27
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector sequence for shRNA5 and shRNA6

<400>  14
acucauauuc aucugcuucu cuaccuc                                           27


<210>  15
<211>  27
<212>  RNA
<213>  artificial sequence

<220>
<223>  Effector complement sequence for shRNA5 and shRNA6

<400>  15
gagguagaga agcagaugaa uaugagu                                           27


<210>  16
<211>  49
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA1 sequence

<400>  16
gaggagaaga uggaggcuga ucaagagaau cagccuccau cuucuccuc                   49


<210>  17
<211>  49
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA2 sequence

<400>  17
aucagccucc aucuucuccu ccaagagaga ggagaagaug gaggcugau                   49


<210>  18
<211>  47
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA3 sequence

<400>  18
ggaagaagcu gagaagcuaa caagagauua gcuucucagc uucuucc                     47


<210>  19
<211>  47
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA4 sequence

<400>  19
uuagcuucuc agcuucuucc caagagagga agaagcugag aagcuaa                     47


<210>  20
<211>  60
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA5 sequence

<400>  20
gagguagaga agcagaugaa uaugagucaa gagacucaua uucaucugcu ucucuaccuc       60


<210>  21
<211>  60
<212>  RNA
<213>  artificial sequence

<220>
<223>  shRNA6 sequence

<400>  21
cucauauuca ucugcuucuc uaccuccaag agagagguag agaagcagau gaauaugagu       60


<210>  22
<211>  1068
<212>  DNA
<213>  artificial sequence

<220>
<223>  OPMD Triple construct short

<400>  22
ctcgagacgc gtggtaccga gctcgctagc agcgcttacg tatttaaatg gcaggaagag       60

ggcctatttc ccatgattcc ttcatatttg catatacgat acaaggctgt tagagagata      120

attagaatta atttgactgt aaacacaaag atattagtac aaaatacgtg acgtagaaag      180

taataatttc ttgggtagtt tgcagtttta aaattatgtt ttaaaatgga ctatcatatg      240

cttaccgtaa cttgaaagta tttcgatttc ttggctttat atatcttgtg gaaaggacga      300

aacaccgagg agaagatgga ggctgatcaa gagaatcagc ctccatcttc tcctcttttt      360

tatgcgcacg tttaaacagg gcggtgcggc tcaggctctg ccccgcctcc ggggctattt      420

gcatacgacc atttccagta attcccagca gccaccgtag ctatatttgg tagaacaacg      480

agcactttct caactccagt caataactac gttagttgca ttacacattg ggctaatata      540

aatagaggtt aaatctctag gtcatttaag agaagtcggc ctatgtgtac agacatttgt      600

tccaggggct ttaaatagct ggtggtggaa ctcaatattc ggaagaagct gagaagctaa      660

caagagatta gcttctcagc ttcttccttt tttgtatacg atacctatca attcgaacgc      720

tgacgtcatc aacccgctcc aaggaatcgc gggcccagtg tcactaggcg ggaacaccca      780

gcgcgcgtgc gccctggcag gaagatggct gtgagggaca ggggagtggc gccctgcaat      840

atttgcatgt cgctatgtgt tctgggaaat caccataaac gtgaaatgtc tttggatttg      900

ggaatcttat aagttctgta tgagaccaca gatccccgag gtagagaagc agatgaatat      960

gagttcaaga gactcatatt catctgcttc tctacctctt tttgacacac gtcctgcagc     1020

gttaaccaat tgccacgggg ttggggtacc acgcgtgagc tcctcgag                  1068


<210>  23
<211>  1527
<212>  DNA
<213>  artificial sequence

<220>
<223>  OPMD Triple construct long

<400>  23
ctcgagacgc gtggtaccga gctcgctagc agcgcttacg tatttaaatg gcaggaagag       60

ggcctatttc ccatgattcc ttcatatttg catatacgat acaaggctgt tagagagata      120

attagaatta atttgactgt aaacacaaag atattagtac aaaatacgtg acgtagaaag      180

taataatttc ttgggtagtt tgcagtttta aaattatgtt ttaaaatgga ctatcatata      240

agatgggctt accgtaactt gaaagtattt cgatttcttg gctttatata tcttgtggaa      300

aggacgaaac accgaggaga agatggaggc tgatcaagag aatcagcctc catcttctcc      360

tcttttttac atcaggttgt ttttctgttt ttacatcagg ttgtttttct gtttggtttt      420

ttttttacac cacgtttata cgccggtgca cggtttacca ctgaaaacac ctttcatcta      480

caggtgatat cttttaacac aaataaaatg tagtagtcca tgcgcacgtt taaacaaggg      540

cggtgcggct caggctctgc cccgcctccg gggctatttg catacgacca tttccagtaa      600

ttcccagcag ccaccgtagc tatatttggt agaacaacga gcactttctc aactccagtc      660

aataactacg ttagttgcat tacacattgg gctaatataa atagaggtta aatctctagg      720

tcatttaaga gaagtcggcc tatgtgtaca gacatttgtt ccaggggctt taaatagctg      780

gtggtggaac tcaatattcg gaagaagctg agaagctaac aagagattag cttctcagct      840

tcttcctttt ttccattttc cctcccagaa acggaatctt gctctgttgc ccaggctgga      900

atgcaatggc gcgatcttag cttgttgcaa cctccgcctc ccgggttgaa gcgattctcc      960

tgcctcagcc tcccgagtag ctgggattat aaacatgcgc cagtatacga tacctatcaa     1020

ttcgaacgct gacgtcatca acccgctcca aggaatcgcg ggcccagtgt cactaggcgg     1080

gaacacccag cgcgcgtgcg ccctggcagg aagatggctg tgagggacag gggagtggcg     1140

ccctgcaata tttgcatgtc gctatgtgtt ctgggaaatc accataaacg tgaaatgtct     1200

ttggatttgg gaatcttata agttctgtat gagaccacag atccccgagg tagagaagca     1260

gatgaatatg agttcaagag actcatattc atctgcttct ctacctcttt ttaaacaaaa     1320

cgaaaccggg ccgggcgcgg tggttcacgc ctataatccc tgcactttgg gaggccgagg     1380

cgggcggatc acaaggtcag gaggtcgaga ccatccaggc taacacggtg aaaccccccc     1440

ccatctctac taaaaaaaaa aagacacacg tcctgcagcg ttaaccaatt gccacggggt     1500

tggggtacca cgcgtgagct cctcgag                                         1527


<210>  24
<211>  950
<212>  DNA
<213>  artificial sequence

<220>
<223>  Human codon-optimized PABpN1 cDNA sequence (with kozak sequence 
       but without myc-tag)

<400>  24
ccgctgccgc cgctgctgct gccgcagccg gcgctgccgg cggaagaggc agcggccctg       60

gcagacggcg gcatctggtc cctggcgccg gaggggaggc cggcgaaggc gcccctggcg      120

gagccggcga ctacggcaac ggcctggaaa gcgaggaact ggaacccgag gaactgctgc      180

tggaacctga gcccgagcca gagcccgagg aagagccccc taggccaaga gccccccctg      240

gcgccccagg accaggacca ggctctgggg caccaggctc tcaggaagag gaagaagagc      300

ccggcctcgt cgagggagac ccaggcgatg gcgctatcga agatcccgag ctggaagcca      360

tcaaggccag agtgcgggag atggaagagg aggccgaaaa attgaaagag ctgcagaacg      420

aagtcgaaaa acaaatgaac atgtcccccc ctcctggaaa tgctggccct gtgatcatga      480

gcatcgagga aaagatggaa gccgacgccc ggtctatcta cgtgggcaac gtggactacg      540

gcgccaccgc cgaagaactg gaagcccact ttcacggctg tggcagcgtg aaccgggtga      600

ccatcctgtg cgacaagttc agcggccacc ccaagggctt cgcctacatc gagttcagcg      660

acaaagaaag cgtgcggacc tctctggctc tcgacgagtc tctgttcagg ggaaggcaga      720

tcaaggtcat ccccaagcgg accaacaggc ccggcatcag caccaccgac agaggcttcc      780

ctagggctag gtacagagcc cggaccacca actacaacag cagcagaagc cggttctaca      840

gcggcttcaa ttctcggcct agaggcagag tgtaccgggg cagggccagg gccacctcct      900

ggtacagccc ctactgaacc tcctggtaca gcccctactg atgagatatc                 950


<210>  25
<211>  306
<212>  PRT
<213>  artificial sequence

<220>
<223>  Human codon-optimized PABpN1 amino acid sequence (without 
       Myc-tag)

<400>  25

Met Ala Ala Ala Ala Ala Ala Ala Ala Ala Ala Gly Ala Ala Gly Gly 
1               5                   10                  15      


Arg Gly Ser Gly Pro Gly Arg Arg Arg His Leu Val Pro Gly Ala Gly 
            20                  25                  30          


Gly Glu Ala Gly Glu Gly Ala Pro Gly Gly Ala Gly Asp Tyr Gly Asn 
        35                  40                  45              


Gly Leu Glu Ser Glu Glu Leu Glu Pro Glu Glu Leu Leu Leu Glu Pro 
    50                  55                  60                  


Glu Pro Glu Pro Glu Pro Glu Glu Glu Pro Pro Arg Pro Arg Ala Pro 
65                  70                  75                  80  


Pro Gly Ala Pro Gly Pro Gly Pro Gly Ser Gly Ala Pro Gly Ser Gln 
                85                  90                  95      


Glu Glu Glu Glu Glu Pro Gly Leu Val Glu Gly Asp Pro Gly Asp Gly 
            100                 105                 110         


Ala Ile Glu Asp Pro Glu Leu Glu Ala Ile Lys Ala Arg Val Arg Glu 
        115                 120                 125             


Met Glu Glu Glu Ala Glu Lys Leu Lys Glu Leu Gln Asn Glu Val Glu 
    130                 135                 140                 


Lys Gln Met Asn Met Ser Pro Pro Pro Gly Asn Ala Gly Pro Val Ile 
145                 150                 155                 160 


Met Ser Ile Glu Glu Lys Met Glu Ala Asp Ala Arg Ser Ile Tyr Val 
                165                 170                 175     


Gly Asn Val Asp Tyr Gly Ala Thr Ala Glu Glu Leu Glu Ala His Phe 
            180                 185                 190         


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


Ser Gly His Pro Lys Gly Phe Ala Tyr Ile Glu Phe Ser Asp Lys Glu 
    210                 215                 220                 


Ser Val Arg Thr Ser Leu Ala Leu Asp Glu Ser Leu Phe Arg Gly Arg 
225                 230                 235                 240 


Gln Ile Lys Val Ile Pro Lys Arg Thr Asn Arg Pro Gly Ile Ser Thr 
                245                 250                 255     


Thr Asp Arg Gly Phe Pro Arg Ala Arg Tyr Arg Ala Arg Thr Thr Asn 
            260                 265                 270         


Tyr Asn Ser Ser Arg Ser Arg Phe Tyr Ser Gly Phe Asn Ser Arg Pro 
        275                 280                 285             


Arg Gly Arg Val Tyr Arg Gly Arg Ala Arg Ala Thr Ser Trp Tyr Ser 
    290                 295                 300                 


Pro Tyr 
305     


<210>  26
<211>  947
<212>  DNA
<213>  artificial sequence

<220>
<223>  Human codon-optimized PABpN1 cDNA sequence (with kozak sequence 
       and Myc-tag)

<400>  26
ccgctgccgc cgctgctgct gccgcagccg gcgctgccgg cggaagaggc agcggccctg       60

gcagacggcg gcatctggtc cctggcgccg gaggggaggc cggcgaaggc gcccctggcg      120

gagccggcga ctacggcaac ggcctggaaa gcgaggaact ggaacccgag gaactgctgc      180

tggaacctga gcccgagcca gagcccgagg aagagccccc taggccaaga gccccccctg      240

gcgccccagg accaggacca ggctctgggg caccaggctc tcaggaagag gaagaagagc      300

ccggcctcgt cgagggagac ccaggcgatg gcgctatcga agatcccgag ctggaagcca      360

tcaaggccag agtgcgggag atggaagagg aggccgaaaa attgaaagag ctgcagaacg      420

aagtcgaaaa acaaatgaac atgtcccccc ctcctggaaa tgctggccct gtgatcatga      480

gcatcgagga aaagatggaa gccgacgccc ggtctatcta cgtgggcaac gtggactacg      540

gcgccaccgc cgaagaactg gaagcccact ttcacggctg tggcagcgtg aaccgggtga      600

ccatcctgtg cgacaagttc agcggccacc ccaagggctt cgcctacatc gagttcagcg      660

acaaagaaag cgtgcggacc tctctggctc tcgacgagtc tctgttcagg ggaaggcaga      720

tcaaggtcat ccccaagcgg accaacaggc ccggcatcag caccaccgac agaggcttcc      780

ctagggctag gtacagagcc cggaccacca actacaacag cagcagaagc cggttctaca      840

gcggcttcaa ttctcggcct agaggcagag tgtaccgggg cagggccagg gccacctcct      900

ggtacagccc ctacgaacag aagctgatca gcgaggaaga tctgtga                    947


<210>  27
<211>  316
<212>  PRT
<213>  artificial sequence

<220>
<223>  Human codon-optimized PABpN1 amino acid sequence (with Myc-tag)

<400>  27

Met Ala Ala Ala Ala Ala Ala Ala Ala Ala Ala Gly Ala Ala Gly Gly 
1               5                   10                  15      


Arg Gly Ser Gly Pro Gly Arg Arg Arg His Leu Val Pro Gly Ala Gly 
            20                  25                  30          


Gly Glu Ala Gly Glu Gly Ala Pro Gly Gly Ala Gly Asp Tyr Gly Asn 
        35                  40                  45              


Gly Leu Glu Ser Glu Glu Leu Glu Pro Glu Glu Leu Leu Leu Glu Pro 
    50                  55                  60                  


Glu Pro Glu Pro Glu Pro Glu Glu Glu Pro Pro Arg Pro Arg Ala Pro 
65                  70                  75                  80  


Pro Gly Ala Pro Gly Pro Gly Pro Gly Ser Gly Ala Pro Gly Ser Gln 
                85                  90                  95      


Glu Glu Glu Glu Glu Pro Gly Leu Val Glu Gly Asp Pro Gly Asp Gly 
            100                 105                 110         


Ala Ile Glu Asp Pro Glu Leu Glu Ala Ile Lys Ala Arg Val Arg Glu 
        115                 120                 125             


Met Glu Glu Glu Ala Glu Lys Leu Lys Glu Leu Gln Asn Glu Val Glu 
    130                 135                 140                 


Lys Gln Met Asn Met Ser Pro Pro Pro Gly Asn Ala Gly Pro Val Ile 
145                 150                 155                 160 


Met Ser Ile Glu Glu Lys Met Glu Ala Asp Ala Arg Ser Ile Tyr Val 
                165                 170                 175     


Gly Asn Val Asp Tyr Gly Ala Thr Ala Glu Glu Leu Glu Ala His Phe 
            180                 185                 190         


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


Ser Gly His Pro Lys Gly Phe Ala Tyr Ile Glu Phe Ser Asp Lys Glu 
    210                 215                 220                 


Ser Val Arg Thr Ser Leu Ala Leu Asp Glu Ser Leu Phe Arg Gly Arg 
225                 230                 235                 240 


Gln Ile Lys Val Ile Pro Lys Arg Thr Asn Arg Pro Gly Ile Ser Thr 
                245                 250                 255     


Thr Asp Arg Gly Phe Pro Arg Ala Arg Tyr Arg Ala Arg Thr Thr Asn 
            260                 265                 270         


Tyr Asn Ser Ser Arg Ser Arg Phe Tyr Ser Gly Phe Asn Ser Arg Pro 
        275                 280                 285             


Arg Gly Arg Val Tyr Arg Gly Arg Ala Arg Ala Thr Ser Trp Tyr Ser 
    290                 295                 300                 


Pro Tyr Glu Gln Lys Leu Ile Ser Glu Glu Asp Leu 
305                 310                 315     


<210>  28
<211>  945
<212>  DNA
<213>  artificial sequence

<220>
<223>  Mutant human PABpN1 cDNA sequence (with FLAG tag)

<400>  28
atggcggcgg cggcggcggc ggcagcagca gcgggggctg cgggcggtcg gggctccggg       60

ccggggcggc ggcgccatct tgtgcccggg gccggtgggg aggccgggga gggggccccg      120

gggggcgcag gggactacgg gaacggcctg gagtctgagg aactggagcc tgaggagctg      180

ctgctggagc ccgagccgga gcccgagccc gaagaggagc cgccccggcc ccgcgccccc      240

ccgggagctc cgggccctgg gcctggttcg ggagcccccg gcagccaaga ggaggaggag      300

gagccgggac tggtcgaggg tgacccgggg gacggcgcca ttgaggaccc ggagctggaa      360

gctatcaaag ctcgagtcag ggagatggag gaagaagctg agaagctaaa ggagctacag      420

aacgaggtag agaagcagat gaatatgagt ccacctccag gcaatgctgg cccggtgatc      480

atgtccattg aggagaagat ggaggctgat gcccgttcca tctatgttgg caatgtggac      540

tatggtgcaa cagcagaaga gctggaagct cactttcatg gctgtggttc agtcaaccgt      600

gttaccatac tgtgtgacaa atttagtggc catcccaaag ggtttgcgta tatagagttc      660

tcagacaaag agtcagtgag gacttccttg gccttagatg agtccctatt tagaggaagg      720

caaatcaagg tgatcccaaa acgaaccaac agaccaggca tcagcacaac agaccggggt      780

tttccacgag cccgctaccg cgcccggacc accaactaca acagctcccg ctctcgattc      840

tacagtggtt ttaacagcag gccccggggt cgcgtctaca ggggccgggc tagagcgaca      900

tcatggtatt ccccttacga ctacaaggac gacgatgaca agtga                      945


<210>  29
<211>  314
<212>  PRT
<213>  artificial sequence

<220>
<223>  Mutant human PABpN1 amino acid sequence (with FLAG tag)

<400>  29

Met Ala Ala Ala Ala Ala Ala Ala Ala Ala Ala Gly Ala Ala Gly Gly 
1               5                   10                  15      


Arg Gly Ser Gly Pro Gly Arg Arg Arg His Leu Val Pro Gly Ala Gly 
            20                  25                  30          


Gly Glu Ala Gly Glu Gly Ala Pro Gly Gly Ala Gly Asp Tyr Gly Asn 
        35                  40                  45              


Gly Leu Glu Ser Glu Glu Leu Glu Pro Glu Glu Leu Leu Leu Glu Pro 
    50                  55                  60                  


Glu Pro Glu Pro Glu Pro Glu Glu Glu Pro Pro Arg Pro Arg Ala Pro 
65                  70                  75                  80  


Pro Gly Ala Pro Gly Pro Gly Pro Gly Ser Gly Ala Pro Gly Ser Gln 
                85                  90                  95      


Glu Glu Glu Glu Glu Pro Gly Leu Val Glu Gly Asp Pro Gly Asp Gly 
            100                 105                 110         


Ala Ile Glu Asp Pro Glu Leu Glu Ala Ile Lys Ala Arg Val Arg Glu 
        115                 120                 125             


Met Glu Glu Glu Ala Glu Lys Leu Lys Glu Leu Gln Asn Glu Val Glu 
    130                 135                 140                 


Lys Gln Met Asn Met Ser Pro Pro Pro Gly Asn Ala Gly Pro Val Ile 
145                 150                 155                 160 


Met Ser Ile Glu Glu Lys Met Glu Ala Asp Ala Arg Ser Ile Tyr Val 
                165                 170                 175     


Gly Asn Val Asp Tyr Gly Ala Thr Ala Glu Glu Leu Glu Ala His Phe 
            180                 185                 190         


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


Ser Gly His Pro Lys Gly Phe Ala Tyr Ile Glu Phe Ser Asp Lys Glu 
    210                 215                 220                 


Ser Val Arg Thr Ser Leu Ala Leu Asp Glu Ser Leu Phe Arg Gly Arg 
225                 230                 235                 240 


Gln Ile Lys Val Ile Pro Lys Arg Thr Asn Arg Pro Gly Ile Ser Thr 
                245                 250                 255     


Thr Asp Arg Gly Phe Pro Arg Ala Arg Tyr Arg Ala Arg Thr Thr Asn 
            260                 265                 270         


Tyr Asn Ser Ser Arg Ser Arg Phe Tyr Ser Gly Phe Asn Ser Arg Pro 
        275                 280                 285             


Arg Gly Arg Val Tyr Arg Gly Arg Ala Arg Ala Thr Ser Trp Tyr Ser 
    290                 295                 300                 


Pro Tyr Asp Tyr Lys Asp Asp Asp Asp Lys 
305                 310                 


