                         SEQUENCE LISTING

<110>  Oxford University Innovation Limited
 
<120>  Treatment

<130>  N417623WO

<150>  GB1914296.7
<151>  2019-10-03

<160>  35    

<170>  PatentIn version 3.5

<210>  1
<211>  365
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Polypeptide sequence of an AR variant

<400>  1

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


Tyr Gly Ala Leu Thr Cys Gly Ser Cys Lys Val Phe Phe Lys Arg Ala 
            20                  25                  30          


Ala Glu Gly Lys Gln Lys Tyr Leu Cys Ala Ser Arg Asn Asp Cys Thr 
        35                  40                  45              


Ile Asp Lys Phe Arg Arg Lys Asn Cys Pro Ser Cys Arg Leu Arg Lys 
    50                  55                  60                  


Cys Tyr Glu Ala Gly Met Thr Leu Gly Ala Arg Lys Leu Lys Lys Leu 
65                  70                  75                  80  


Gly Asn Leu Lys Leu Gln Glu Glu Gly Glu Ala Ser Ser Thr Thr Ser 
                85                  90                  95      


Pro Thr Glu Glu Thr Thr Gln Lys Leu Thr Val Ser His Ile Glu Gly 
            100                 105                 110         


Tyr Glu Cys Gln Pro Ile Phe Leu Asn Val Leu Glu Ala Ile Glu Pro 
        115                 120                 125             


Gly Val Val Cys Ala Gly His Asp Asn Asn Gln Pro Asp Ser Phe Ala 
    130                 135                 140                 


Ala Leu Leu Ser Ser Leu Asn Glu Leu Gly Glu Arg Gln Leu Val His 
145                 150                 155                 160 


Val Val Lys Trp Ala Lys Ala Leu Pro Gly Phe Arg Asn Leu His Val 
                165                 170                 175     


Asp Asp Gln Met Ala Val Ile Gln Tyr Ser Trp Met Gly Leu Met Val 
            180                 185                 190         


Phe Ala Met Gly Trp Arg Ser Phe Thr Asn Val Asn Ser Arg Met Leu 
        195                 200                 205             


Tyr Phe Ala Pro Asp Leu Val Phe Asn Glu Tyr Arg Met His Lys Ser 
    210                 215                 220                 


Arg Met Tyr Ser Gln Cys Val Arg Met Arg His Leu Ser Gln Glu Phe 
225                 230                 235                 240 


Gly Trp Leu Gln Ile Thr Pro Gln Glu Phe Leu Cys Met Lys Ala Leu 
                245                 250                 255     


Leu Leu Phe Ser Ile Ile Pro Val Asp Gly Leu Lys Asn Gln Lys Phe 
            260                 265                 270         


Phe Asp Glu Leu Arg Met Asn Tyr Ile Lys Glu Leu Asp Arg Ile Ile 
        275                 280                 285             


Ala Cys Lys Arg Lys Asn Pro Thr Ser Cys Ser Arg Arg Phe Tyr Gln 
    290                 295                 300                 


Leu Thr Lys Leu Leu Asp Ser Val Gln Pro Ile Ala Arg Glu Leu His 
305                 310                 315                 320 


Gln Phe Thr Phe Asp Leu Leu Ile Lys Ser His Met Val Ser Val Asp 
                325                 330                 335     


Phe Pro Glu Met Met Ala Glu Ile Ile Ser Val Gln Val Pro Lys Ile 
            340                 345                 350         


Leu Ser Gly Lys Val Lys Pro Ile Tyr Phe His Thr Gln 
        355                 360                 365 


<210>  2
<211>  1098
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  cDNA encoding the AR variant of SEQ ID NO: 1

<400>  2
ccccagaaga cctgcctgat ctgtggagat gaagcttctg ggtgtcacta tggagctctc       60

acatgtggaa gctgcaaggt cttcttcaaa agagccgctg aagggaaaca gaagtacctg      120

tgcgccagca gaaatgattg cactattgat aaattccgaa ggaaaaattg tccatcttgt      180

cgtcttcgga aatgttatga agcagggatg actctgggag cccggaagct gaagaaactt      240

ggtaatctga aactacagga ggaaggagag gcttccagca ccaccagccc cactgaggag      300

acaacccaga agctgacagt gtcacacatt gaaggctatg aatgtcagcc catctttctg      360

aatgtcctgg aagccattga gccaggtgta gtgtgtgctg gacacgacaa caaccagccc      420

gactcctttg cagccttgct ctctagcctc aatgaactgg gagagagaca gcttgtacac      480

gtggtcaagt gggccaaggc cttgcctggc ttccgcaact tacacgtgga cgaccagatg      540

gctgtcattc agtactcctg gatggggctc atggtgtttg ccatgggctg gcgatccttc      600

accaatgtca actccaggat gctctacttc gcccctgatc tggttttcaa tgagtaccgc      660

atgcacaagt cccggatgta cagccagtgt gtccgaatga ggcacctctc tcaagagttt      720

ggatggctcc aaatcacccc ccaggaattc ctgtgcatga aagcactgct actcttcagc      780

attattccag tggatgggct gaaaaatcaa aaattctttg atgaacttcg aatgaactac      840

atcaaggaac tcgatcgtat cattgcatgc aaaagaaaaa atcccacatc ctgctcaaga      900

cgcttctacc agctcaccaa gctcctggac tccgtgcagc ctattgcgag agagctgcat      960

cagttcactt ttgacctgct aatcaagtca cacatggtga gcgtggactt tccggaaatg     1020

atggcagaga tcatctctgt gcaagtgccc aagatccttt ctgggaaagt caagcccatc     1080

tatttccaca cccagtga                                                   1098


<210>  3
<211>  388
<212>  PRT
<213>  Homo sapiens

<400>  3

Met Ile Leu Trp Leu His Ser Leu Glu Thr Ala Arg Asp His Val Leu 
1               5                   10                  15      


Pro Ile Asp Tyr Tyr Phe Pro Pro Gln Lys Thr Cys Leu Ile Cys Gly 
            20                  25                  30          


Asp Glu Ala Ser Gly Cys His Tyr Gly Ala Leu Thr Cys Gly Ser Cys 
        35                  40                  45              


Lys Val Phe Phe Lys Arg Ala Ala Glu Gly Lys Gln Lys Tyr Leu Cys 
    50                  55                  60                  


Ala Ser Arg Asn Asp Cys Thr Ile Asp Lys Phe Arg Arg Lys Asn Cys 
65                  70                  75                  80  


Pro Ser Cys Arg Leu Arg Lys Cys Tyr Glu Ala Gly Met Thr Leu Gly 
                85                  90                  95      


Ala Arg Lys Leu Lys Lys Leu Gly Asn Leu Lys Leu Gln Glu Glu Gly 
            100                 105                 110         


Glu Ala Ser Ser Thr Thr Ser Pro Thr Glu Glu Thr Thr Gln Lys Leu 
        115                 120                 125             


Thr Val Ser His Ile Glu Gly Tyr Glu Cys Gln Pro Ile Phe Leu Asn 
    130                 135                 140                 


Val Leu Glu Ala Ile Glu Pro Gly Val Val Cys Ala Gly His Asp Asn 
145                 150                 155                 160 


Asn Gln Pro Asp Ser Phe Ala Ala Leu Leu Ser Ser Leu Asn Glu Leu 
                165                 170                 175     


Gly Glu Arg Gln Leu Val His Val Val Lys Trp Ala Lys Ala Leu Pro 
            180                 185                 190         


Gly Phe Arg Asn Leu His Val Asp Asp Gln Met Ala Val Ile Gln Tyr 
        195                 200                 205             


Ser Trp Met Gly Leu Met Val Phe Ala Met Gly Trp Arg Ser Phe Thr 
    210                 215                 220                 


Asn Val Asn Ser Arg Met Leu Tyr Phe Ala Pro Asp Leu Val Phe Asn 
225                 230                 235                 240 


Glu Tyr Arg Met His Lys Ser Arg Met Tyr Ser Gln Cys Val Arg Met 
                245                 250                 255     


Arg His Leu Ser Gln Glu Phe Gly Trp Leu Gln Ile Thr Pro Gln Glu 
            260                 265                 270         


Phe Leu Cys Met Lys Ala Leu Leu Leu Phe Ser Ile Ile Pro Val Asp 
        275                 280                 285             


Gly Leu Lys Asn Gln Lys Phe Phe Asp Glu Leu Arg Met Asn Tyr Ile 
    290                 295                 300                 


Lys Glu Leu Asp Arg Ile Ile Ala Cys Lys Arg Lys Asn Pro Thr Ser 
305                 310                 315                 320 


Cys Ser Arg Arg Phe Tyr Gln Leu Thr Lys Leu Leu Asp Ser Val Gln 
                325                 330                 335     


Pro Ile Ala Arg Glu Leu His Gln Phe Thr Phe Asp Leu Leu Ile Lys 
            340                 345                 350         


Ser His Met Val Ser Val Asp Phe Pro Glu Met Met Ala Glu Ile Ile 
        355                 360                 365             


Ser Val Gln Val Pro Lys Ile Leu Ser Gly Lys Val Lys Pro Ile Tyr 
    370                 375                 380                 


Phe His Thr Gln 
385             


<210>  4
<211>  1167
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  cDNA sequence of human AR2

<400>  4
atgatactct ggcttcacag tttggagact gccagggacc atgttttgcc cattgactat       60

tactttccac cccagaagac ctgcctgatc tgtggagatg aagcttctgg gtgtcactat      120

ggagctctca catgtggaag ctgcaaggtc ttcttcaaaa gagccgctga agggaaacag      180

aagtacctgt gcgccagcag aaatgattgc actattgata aattccgaag gaaaaattgt      240

ccatcttgtc gtcttcggaa atgttatgaa gcagggatga ctctgggagc ccggaagctg      300

aagaaacttg gtaatctgaa actacaggag gaaggagagg cttccagcac caccagcccc      360

actgaggaga caacccagaa gctgacagtg tcacacattg aaggctatga atgtcagccc      420

atctttctga atgtcctgga agccattgag ccaggtgtag tgtgtgctgg acacgacaac      480

aaccagcccg actcctttgc agccttgctc tctagcctca atgaactggg agagagacag      540

cttgtacacg tggtcaagtg ggccaaggcc ttgcctggct tccgcaactt acacgtggac      600

gaccagatgg ctgtcattca gtactcctgg atggggctca tggtgtttgc catgggctgg      660

cgatccttca ccaatgtcaa ctccaggatg ctctacttcg cccctgatct ggttttcaat      720

gagtaccgca tgcacaagtc ccggatgtac agccagtgtg tccgaatgag gcacctctct      780

caagagtttg gatggctcca aatcaccccc caggaattcc tgtgcatgaa agcactgcta      840

ctcttcagca ttattccagt ggatgggctg aaaaatcaaa aattctttga tgaacttcga      900

atgaactaca tcaaggaact cgatcgtatc attgcatgca aaagaaaaaa tcccacatcc      960

tgctcaagac gcttctacca gctcaccaag ctcctggact ccgtgcagcc tattgcgaga     1020

gagctgcatc agttcacttt tgacctgcta atcaagtcac acatggtgag cgtggacttt     1080

ccggaaatga tggcagagat catctctgtg caagtgccca agatcctttc tgggaaagtc     1140

aagcccatct atttccacac ccagtga                                         1167


<210>  5
<211>  555
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  NTD of human AR

<400>  5

Met Glu Val Gln Leu Gly Leu Gly Arg Val Tyr Pro Arg Pro Pro Ser 
1               5                   10                  15      


Lys Thr Tyr Arg Gly Ala Phe Gln Asn Leu Phe Gln Ser Val Arg Glu 
            20                  25                  30          


Val Ile Gln Asn Pro Gly Pro Arg His Pro Glu Ala Ala Ser Ala Ala 
        35                  40                  45              


Pro Pro Gly Ala Ser Leu Leu Leu Leu Gln Gln Gln Gln Gln Gln Gln 
    50                  55                  60                  


Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln 
65                  70                  75                  80  


Glu Thr Ser Pro Arg Gln Gln Gln Gln Gln Gln Gly Glu Asp Gly Ser 
                85                  90                  95      


Pro Gln Ala His Arg Arg Gly Pro Thr Gly Tyr Leu Val Leu Asp Glu 
            100                 105                 110         


Glu Gln Gln Pro Ser Gln Pro Gln Ser Ala Leu Glu Cys His Pro Glu 
        115                 120                 125             


Arg Gly Cys Val Pro Glu Pro Gly Ala Ala Val Ala Ala Ser Lys Gly 
    130                 135                 140                 


Leu Pro Gln Gln Leu Pro Ala Pro Pro Asp Glu Asp Asp Ser Ala Ala 
145                 150                 155                 160 


Pro Ser Thr Leu Ser Leu Leu Gly Pro Thr Phe Pro Gly Leu Ser Ser 
                165                 170                 175     


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


Leu Leu Gln Gln Gln Gln Gln Glu Ala Val Ser Glu Gly Ser Ser Ser 
        195                 200                 205             


Gly Arg Ala Arg Glu Ala Ser Gly Ala Pro Thr Ser Ser Lys Asp Asn 
    210                 215                 220                 


Tyr Leu Gly Gly Thr Ser Thr Ile Ser Asp Asn Ala Lys Glu Leu Cys 
225                 230                 235                 240 


Lys Ala Val Ser Val Ser Met Gly Leu Gly Val Glu Ala Leu Glu His 
                245                 250                 255     


Leu Ser Pro Gly Glu Gln Leu Arg Gly Asp Cys Met Tyr Ala Pro Leu 
            260                 265                 270         


Leu Gly Val Pro Pro Ala Val Arg Pro Thr Pro Cys Ala Pro Leu Ala 
        275                 280                 285             


Glu Cys Lys Gly Ser Leu Leu Asp Asp Ser Ala Gly Lys Ser Thr Glu 
    290                 295                 300                 


Asp Thr Ala Glu Tyr Ser Pro Phe Lys Gly Gly Tyr Thr Lys Gly Leu 
305                 310                 315                 320 


Glu Gly Glu Ser Leu Gly Cys Ser Gly Ser Ala Ala Ala Gly Ser Ser 
                325                 330                 335     


Gly Thr Leu Glu Leu Pro Ser Thr Leu Ser Leu Tyr Lys Ser Gly Ala 
            340                 345                 350         


Leu Asp Glu Ala Ala Ala Tyr Gln Ser Arg Asp Tyr Tyr Asn Phe Pro 
        355                 360                 365             


Leu Ala Leu Ala Gly Pro Pro Pro Pro Pro Pro Pro Pro His Pro His 
    370                 375                 380                 


Ala Arg Ile Lys Leu Glu Asn Pro Leu Asp Tyr Gly Ser Ala Trp Ala 
385                 390                 395                 400 


Ala Ala Ala Ala Gln Cys Arg Tyr Gly Asp Leu Ala Ser Leu His Gly 
                405                 410                 415     


Ala Gly Ala Ala Gly Pro Gly Ser Gly Ser Pro Ser Ala Ala Ala Ser 
            420                 425                 430         


Ser Ser Trp His Thr Leu Phe Thr Ala Glu Glu Gly Gln Leu Tyr Gly 
        435                 440                 445             


Pro Cys Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly 
    450                 455                 460                 


Gly Gly Gly Gly Gly Gly Gly Gly Gly Glu Ala Gly Ala Val Ala Pro 
465                 470                 475                 480 


Tyr Gly Tyr Thr Arg Pro Pro Gln Gly Leu Ala Gly Gln Glu Ser Asp 
                485                 490                 495     


Phe Thr Ala Pro Asp Val Trp Tyr Pro Gly Gly Met Val Ser Arg Val 
            500                 505                 510         


Pro Tyr Pro Ser Pro Thr Cys Val Lys Ser Glu Met Gly Pro Trp Met 
        515                 520                 525             


Asp Ser Tyr Ser Gly Pro Tyr Gly Asp Met Arg Leu Glu Thr Ala Arg 
    530                 535                 540                 


Asp His Val Leu Pro Ile Asp Tyr Tyr Phe Pro 
545                 550                 555 


<210>  6
<211>  920
<212>  PRT
<213>  Homo sapiens

<400>  6

Met Glu Val Gln Leu Gly Leu Gly Arg Val Tyr Pro Arg Pro Pro Ser 
1               5                   10                  15      


Lys Thr Tyr Arg Gly Ala Phe Gln Asn Leu Phe Gln Ser Val Arg Glu 
            20                  25                  30          


Val Ile Gln Asn Pro Gly Pro Arg His Pro Glu Ala Ala Ser Ala Ala 
        35                  40                  45              


Pro Pro Gly Ala Ser Leu Leu Leu Leu Gln Gln Gln Gln Gln Gln Gln 
    50                  55                  60                  


Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln Gln 
65                  70                  75                  80  


Glu Thr Ser Pro Arg Gln Gln Gln Gln Gln Gln Gly Glu Asp Gly Ser 
                85                  90                  95      


Pro Gln Ala His Arg Arg Gly Pro Thr Gly Tyr Leu Val Leu Asp Glu 
            100                 105                 110         


Glu Gln Gln Pro Ser Gln Pro Gln Ser Ala Leu Glu Cys His Pro Glu 
        115                 120                 125             


Arg Gly Cys Val Pro Glu Pro Gly Ala Ala Val Ala Ala Ser Lys Gly 
    130                 135                 140                 


Leu Pro Gln Gln Leu Pro Ala Pro Pro Asp Glu Asp Asp Ser Ala Ala 
145                 150                 155                 160 


Pro Ser Thr Leu Ser Leu Leu Gly Pro Thr Phe Pro Gly Leu Ser Ser 
                165                 170                 175     


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


Leu Leu Gln Gln Gln Gln Gln Glu Ala Val Ser Glu Gly Ser Ser Ser 
        195                 200                 205             


Gly Arg Ala Arg Glu Ala Ser Gly Ala Pro Thr Ser Ser Lys Asp Asn 
    210                 215                 220                 


Tyr Leu Gly Gly Thr Ser Thr Ile Ser Asp Asn Ala Lys Glu Leu Cys 
225                 230                 235                 240 


Lys Ala Val Ser Val Ser Met Gly Leu Gly Val Glu Ala Leu Glu His 
                245                 250                 255     


Leu Ser Pro Gly Glu Gln Leu Arg Gly Asp Cys Met Tyr Ala Pro Leu 
            260                 265                 270         


Leu Gly Val Pro Pro Ala Val Arg Pro Thr Pro Cys Ala Pro Leu Ala 
        275                 280                 285             


Glu Cys Lys Gly Ser Leu Leu Asp Asp Ser Ala Gly Lys Ser Thr Glu 
    290                 295                 300                 


Asp Thr Ala Glu Tyr Ser Pro Phe Lys Gly Gly Tyr Thr Lys Gly Leu 
305                 310                 315                 320 


Glu Gly Glu Ser Leu Gly Cys Ser Gly Ser Ala Ala Ala Gly Ser Ser 
                325                 330                 335     


Gly Thr Leu Glu Leu Pro Ser Thr Leu Ser Leu Tyr Lys Ser Gly Ala 
            340                 345                 350         


Leu Asp Glu Ala Ala Ala Tyr Gln Ser Arg Asp Tyr Tyr Asn Phe Pro 
        355                 360                 365             


Leu Ala Leu Ala Gly Pro Pro Pro Pro Pro Pro Pro Pro His Pro His 
    370                 375                 380                 


Ala Arg Ile Lys Leu Glu Asn Pro Leu Asp Tyr Gly Ser Ala Trp Ala 
385                 390                 395                 400 


Ala Ala Ala Ala Gln Cys Arg Tyr Gly Asp Leu Ala Ser Leu His Gly 
                405                 410                 415     


Ala Gly Ala Ala Gly Pro Gly Ser Gly Ser Pro Ser Ala Ala Ala Ser 
            420                 425                 430         


Ser Ser Trp His Thr Leu Phe Thr Ala Glu Glu Gly Gln Leu Tyr Gly 
        435                 440                 445             


Pro Cys Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly 
    450                 455                 460                 


Gly Gly Gly Gly Gly Gly Gly Gly Gly Glu Ala Gly Ala Val Ala Pro 
465                 470                 475                 480 


Tyr Gly Tyr Thr Arg Pro Pro Gln Gly Leu Ala Gly Gln Glu Ser Asp 
                485                 490                 495     


Phe Thr Ala Pro Asp Val Trp Tyr Pro Gly Gly Met Val Ser Arg Val 
            500                 505                 510         


Pro Tyr Pro Ser Pro Thr Cys Val Lys Ser Glu Met Gly Pro Trp Met 
        515                 520                 525             


Asp Ser Tyr Ser Gly Pro Tyr Gly Asp Met Arg Leu Glu Thr Ala Arg 
    530                 535                 540                 


Asp His Val Leu Pro Ile Asp Tyr Tyr Phe Pro Pro Gln Lys Thr Cys 
545                 550                 555                 560 


Leu Ile Cys Gly Asp Glu Ala Ser Gly Cys His Tyr Gly Ala Leu Thr 
                565                 570                 575     


Cys Gly Ser Cys Lys Val Phe Phe Lys Arg Ala Ala Glu Gly Lys Gln 
            580                 585                 590         


Lys Tyr Leu Cys Ala Ser Arg Asn Asp Cys Thr Ile Asp Lys Phe Arg 
        595                 600                 605             


Arg Lys Asn Cys Pro Ser Cys Arg Leu Arg Lys Cys Tyr Glu Ala Gly 
    610                 615                 620                 


Met Thr Leu Gly Ala Arg Lys Leu Lys Lys Leu Gly Asn Leu Lys Leu 
625                 630                 635                 640 


Gln Glu Glu Gly Glu Ala Ser Ser Thr Thr Ser Pro Thr Glu Glu Thr 
                645                 650                 655     


Thr Gln Lys Leu Thr Val Ser His Ile Glu Gly Tyr Glu Cys Gln Pro 
            660                 665                 670         


Ile Phe Leu Asn Val Leu Glu Ala Ile Glu Pro Gly Val Val Cys Ala 
        675                 680                 685             


Gly His Asp Asn Asn Gln Pro Asp Ser Phe Ala Ala Leu Leu Ser Ser 
    690                 695                 700                 


Leu Asn Glu Leu Gly Glu Arg Gln Leu Val His Val Val Lys Trp Ala 
705                 710                 715                 720 


Lys Ala Leu Pro Gly Phe Arg Asn Leu His Val Asp Asp Gln Met Ala 
                725                 730                 735     


Val Ile Gln Tyr Ser Trp Met Gly Leu Met Val Phe Ala Met Gly Trp 
            740                 745                 750         


Arg Ser Phe Thr Asn Val Asn Ser Arg Met Leu Tyr Phe Ala Pro Asp 
        755                 760                 765             


Leu Val Phe Asn Glu Tyr Arg Met His Lys Ser Arg Met Tyr Ser Gln 
    770                 775                 780                 


Cys Val Arg Met Arg His Leu Ser Gln Glu Phe Gly Trp Leu Gln Ile 
785                 790                 795                 800 


Thr Pro Gln Glu Phe Leu Cys Met Lys Ala Leu Leu Leu Phe Ser Ile 
                805                 810                 815     


Ile Pro Val Asp Gly Leu Lys Asn Gln Lys Phe Phe Asp Glu Leu Arg 
            820                 825                 830         


Met Asn Tyr Ile Lys Glu Leu Asp Arg Ile Ile Ala Cys Lys Arg Lys 
        835                 840                 845             


Asn Pro Thr Ser Cys Ser Arg Arg Phe Tyr Gln Leu Thr Lys Leu Leu 
    850                 855                 860                 


Asp Ser Val Gln Pro Ile Ala Arg Glu Leu His Gln Phe Thr Phe Asp 
865                 870                 875                 880 


Leu Leu Ile Lys Ser His Met Val Ser Val Asp Phe Pro Glu Met Met 
                885                 890                 895     


Ala Glu Ile Ile Ser Val Gln Val Pro Lys Ile Leu Ser Gly Lys Val 
            900                 905                 910         


Lys Pro Ile Tyr Phe His Thr Gln 
        915                 920 


<210>  7
<211>  759
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Polypeptide sequence of an AR variant

<400>  7

Met Glu Val Gln Leu Gly Leu Gly Arg Val Tyr Pro Arg Pro Pro Ser 
1               5                   10                  15      


Lys Thr Tyr Arg Gly Ala Phe Gln Asn Leu Phe Gln Ser Val Arg Glu 
            20                  25                  30          


Val Ile Gln Asn Pro Gly Pro Arg His Pro Glu Ala Ala Ser Ala Ala 
        35                  40                  45              


Pro Pro Arg Glu Ala Ser Gly Ala Pro Thr Ser Ser Lys Asp Asn Tyr 
    50                  55                  60                  


Leu Gly Gly Thr Ser Thr Ile Ser Asp Asn Ala Lys Glu Leu Cys Lys 
65                  70                  75                  80  


Ala Val Ser Val Ser Met Gly Leu Gly Val Glu Ala Leu Glu His Leu 
                85                  90                  95      


Ser Pro Gly Glu Gln Leu Arg Gly Asp Cys Met Tyr Ala Pro Leu Leu 
            100                 105                 110         


Gly Val Pro Pro Ala Val Arg Pro Thr Pro Cys Ala Pro Leu Ala Glu 
        115                 120                 125             


Cys Lys Gly Ser Leu Leu Asp Asp Ser Ala Gly Lys Ser Thr Glu Asp 
    130                 135                 140                 


Thr Ala Glu Tyr Ser Pro Phe Lys Gly Gly Tyr Thr Lys Gly Leu Glu 
145                 150                 155                 160 


Gly Glu Ser Leu Gly Cys Ser Gly Ser Ala Ala Ala Gly Ser Ser Gly 
                165                 170                 175     


Thr Leu Glu Leu Pro Ser Thr Leu Ser Leu Tyr Lys Ser Gly Ala Leu 
            180                 185                 190         


Asp Glu Ala Ala Ala Tyr Gln Ser Arg Asp Tyr Tyr Asn Phe Pro Leu 
        195                 200                 205             


Ala Leu Ala Gly Pro Pro Pro Pro Pro Pro Pro Pro His Pro His Ala 
    210                 215                 220                 


Arg Ile Lys Leu Glu Asn Pro Leu Asp Tyr Gly Ser Ala Trp Ala Ala 
225                 230                 235                 240 


Ala Ala Ala Gln Cys Arg Tyr Gly Asp Leu Ala Ser Leu His Gly Ala 
                245                 250                 255     


Gly Ala Ala Gly Pro Gly Ser Gly Ser Pro Ser Ala Ala Ala Ser Ser 
            260                 265                 270         


Ser Trp His Thr Leu Phe Thr Ala Glu Glu Gly Gln Leu Tyr Gly Pro 
        275                 280                 285             


Cys Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly 
    290                 295                 300                 


Gly Gly Gly Gly Gly Gly Gly Gly Glu Ala Gly Ala Val Ala Pro Tyr 
305                 310                 315                 320 


Gly Tyr Thr Arg Pro Pro Gln Gly Leu Ala Gly Gln Glu Ser Asp Phe 
                325                 330                 335     


Thr Ala Pro Asp Val Trp Tyr Pro Gly Gly Met Val Ser Arg Val Pro 
            340                 345                 350         


Tyr Pro Ser Pro Thr Cys Val Lys Ser Glu Met Gly Pro Trp Met Asp 
        355                 360                 365             


Ser Tyr Ser Gly Pro Tyr Gly Asp Met Arg Leu Glu Thr Ala Arg Asp 
    370                 375                 380                 


His Val Leu Pro Ile Asp Tyr Tyr Phe Pro Pro Gln Lys Thr Cys Leu 
385                 390                 395                 400 


Ile Cys Gly Asp Glu Ala Ser Gly Cys His Tyr Gly Ala Leu Thr Cys 
                405                 410                 415     


Gly Ser Cys Lys Val Phe Phe Lys Arg Ala Ala Glu Gly Lys Gln Lys 
            420                 425                 430         


Tyr Leu Cys Ala Ser Arg Asn Asp Cys Thr Ile Asp Lys Phe Arg Arg 
        435                 440                 445             


Lys Asn Cys Pro Ser Cys Arg Leu Arg Lys Cys Tyr Glu Ala Gly Met 
    450                 455                 460                 


Thr Leu Gly Ala Arg Lys Leu Lys Lys Leu Gly Asn Leu Lys Leu Gln 
465                 470                 475                 480 


Glu Glu Gly Glu Ala Ser Ser Thr Thr Ser Pro Thr Glu Glu Thr Thr 
                485                 490                 495     


Gln Lys Leu Thr Val Ser His Ile Glu Gly Tyr Glu Cys Gln Pro Ile 
            500                 505                 510         


Phe Leu Asn Val Leu Glu Ala Ile Glu Pro Gly Val Val Cys Ala Gly 
        515                 520                 525             


His Asp Asn Asn Gln Pro Asp Ser Phe Ala Ala Leu Leu Ser Ser Leu 
    530                 535                 540                 


Asn Glu Leu Gly Glu Arg Gln Leu Val His Val Val Lys Trp Ala Lys 
545                 550                 555                 560 


Ala Leu Pro Gly Phe Arg Asn Leu His Val Asp Asp Gln Met Ala Val 
                565                 570                 575     


Ile Gln Tyr Ser Trp Met Gly Leu Met Val Phe Ala Met Gly Trp Arg 
            580                 585                 590         


Ser Phe Thr Asn Val Asn Ser Arg Met Leu Tyr Phe Ala Pro Asp Leu 
        595                 600                 605             


Val Phe Asn Glu Tyr Arg Met His Lys Ser Arg Met Tyr Ser Gln Cys 
    610                 615                 620                 


Val Arg Met Arg His Leu Ser Gln Glu Phe Gly Trp Leu Gln Ile Thr 
625                 630                 635                 640 


Pro Gln Glu Phe Leu Cys Met Lys Ala Leu Leu Leu Phe Ser Ile Ile 
                645                 650                 655     


Pro Val Asp Gly Leu Lys Asn Gln Lys Phe Phe Asp Glu Leu Arg Met 
            660                 665                 670         


Asn Tyr Ile Lys Glu Leu Asp Arg Ile Ile Ala Cys Lys Arg Lys Asn 
        675                 680                 685             


Pro Thr Ser Cys Ser Arg Arg Phe Tyr Gln Leu Thr Lys Leu Leu Asp 
    690                 695                 700                 


Ser Val Gln Pro Ile Ala Arg Glu Leu His Gln Phe Thr Phe Asp Leu 
705                 710                 715                 720 


Leu Ile Lys Ser His Met Val Ser Val Asp Phe Pro Glu Met Met Ala 
                725                 730                 735     


Glu Ile Ile Ser Val Gln Val Pro Lys Ile Leu Ser Gly Lys Val Lys 
            740                 745                 750         


Pro Ile Tyr Phe His Thr Gln 
        755                 


<210>  8
<211>  709
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Polypeptide sequence of an AR variant

<400>  8

Arg Glu Ala Ser Gly Ala Pro Thr Ser Ser Lys Asp Asn Tyr Leu Gly 
1               5                   10                  15      


Gly Thr Ser Thr Ile Ser Asp Asn Ala Lys Glu Leu Cys Lys Ala Val 
            20                  25                  30          


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


Gly Glu Gln Leu Arg Gly Asp Cys Met Tyr Ala Pro Leu Leu Gly Val 
    50                  55                  60                  


Pro Pro Ala Val Arg Pro Thr Pro Cys Ala Pro Leu Ala Glu Cys Lys 
65                  70                  75                  80  


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


Glu Tyr Ser Pro Phe Lys Gly Gly Tyr Thr Lys Gly Leu Glu Gly Glu 
            100                 105                 110         


Ser Leu Gly Cys Ser Gly Ser Ala Ala Ala Gly Ser Ser Gly Thr Leu 
        115                 120                 125             


Glu Leu Pro Ser Thr Leu Ser Leu Tyr Lys Ser Gly Ala Leu Asp Glu 
    130                 135                 140                 


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


Ala Gly Pro Pro Pro Pro Pro Pro Pro Pro His Pro His Ala Arg Ile 
                165                 170                 175     


Lys Leu Glu Asn Pro Leu Asp Tyr Gly Ser Ala Trp Ala Ala Ala Ala 
            180                 185                 190         


Ala Gln Cys Arg Tyr Gly Asp Leu Ala Ser Leu His Gly Ala Gly Ala 
        195                 200                 205             


Ala Gly Pro Gly Ser Gly Ser Pro Ser Ala Ala Ala Ser Ser Ser Trp 
    210                 215                 220                 


His Thr Leu Phe Thr Ala Glu Glu Gly Gln Leu Tyr Gly Pro Cys Gly 
225                 230                 235                 240 


Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly Gly 
                245                 250                 255     


Gly Gly Gly Gly Gly Gly Glu Ala Gly Ala Val Ala Pro Tyr Gly Tyr 
            260                 265                 270         


Thr Arg Pro Pro Gln Gly Leu Ala Gly Gln Glu Ser Asp Phe Thr Ala 
        275                 280                 285             


Pro Asp Val Trp Tyr Pro Gly Gly Met Val Ser Arg Val Pro Tyr Pro 
    290                 295                 300                 


Ser Pro Thr Cys Val Lys Ser Glu Met Gly Pro Trp Met Asp Ser Tyr 
305                 310                 315                 320 


Ser Gly Pro Tyr Gly Asp Met Arg Leu Glu Thr Ala Arg Asp His Val 
                325                 330                 335     


Leu Pro Ile Asp Tyr Tyr Phe Pro Pro Gln Lys Thr Cys Leu Ile Cys 
            340                 345                 350         


Gly Asp Glu Ala Ser Gly Cys His Tyr Gly Ala Leu Thr Cys Gly Ser 
        355                 360                 365             


Cys Lys Val Phe Phe Lys Arg Ala Ala Glu Gly Lys Gln Lys Tyr Leu 
    370                 375                 380                 


Cys Ala Ser Arg Asn Asp Cys Thr Ile Asp Lys Phe Arg Arg Lys Asn 
385                 390                 395                 400 


Cys Pro Ser Cys Arg Leu Arg Lys Cys Tyr Glu Ala Gly Met Thr Leu 
                405                 410                 415     


Gly Ala Arg Lys Leu Lys Lys Leu Gly Asn Leu Lys Leu Gln Glu Glu 
            420                 425                 430         


Gly Glu Ala Ser Ser Thr Thr Ser Pro Thr Glu Glu Thr Thr Gln Lys 
        435                 440                 445             


Leu Thr Val Ser His Ile Glu Gly Tyr Glu Cys Gln Pro Ile Phe Leu 
    450                 455                 460                 


Asn Val Leu Glu Ala Ile Glu Pro Gly Val Val Cys Ala Gly His Asp 
465                 470                 475                 480 


Asn Asn Gln Pro Asp Ser Phe Ala Ala Leu Leu Ser Ser Leu Asn Glu 
                485                 490                 495     


Leu Gly Glu Arg Gln Leu Val His Val Val Lys Trp Ala Lys Ala Leu 
            500                 505                 510         


Pro Gly Phe Arg Asn Leu His Val Asp Asp Gln Met Ala Val Ile Gln 
        515                 520                 525             


Tyr Ser Trp Met Gly Leu Met Val Phe Ala Met Gly Trp Arg Ser Phe 
    530                 535                 540                 


Thr Asn Val Asn Ser Arg Met Leu Tyr Phe Ala Pro Asp Leu Val Phe 
545                 550                 555                 560 


Asn Glu Tyr Arg Met His Lys Ser Arg Met Tyr Ser Gln Cys Val Arg 
                565                 570                 575     


Met Arg His Leu Ser Gln Glu Phe Gly Trp Leu Gln Ile Thr Pro Gln 
            580                 585                 590         


Glu Phe Leu Cys Met Lys Ala Leu Leu Leu Phe Ser Ile Ile Pro Val 
        595                 600                 605             


Asp Gly Leu Lys Asn Gln Lys Phe Phe Asp Glu Leu Arg Met Asn Tyr 
    610                 615                 620                 


Ile Lys Glu Leu Asp Arg Ile Ile Ala Cys Lys Arg Lys Asn Pro Thr 
625                 630                 635                 640 


Ser Cys Ser Arg Arg Phe Tyr Gln Leu Thr Lys Leu Leu Asp Ser Val 
                645                 650                 655     


Gln Pro Ile Ala Arg Glu Leu His Gln Phe Thr Phe Asp Leu Leu Ile 
            660                 665                 670         


Lys Ser His Met Val Ser Val Asp Phe Pro Glu Met Met Ala Glu Ile 
        675                 680                 685             


Ile Ser Val Gln Val Pro Lys Ile Leu Ser Gly Lys Val Lys Pro Ile 
    690                 695                 700                 


Tyr Phe His Thr Gln 
705                 


<210>  9
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-1 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  9
ccaaacugug aagccagagu                                                   20


<210>  10
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-1 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  10
ggcuucacag uuugg                                                        15


<210>  11
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-2 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  11
uccaaacugu gaagccagag                                                   20


<210>  12
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-2 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  12
gcuucacagu uugga                                                        15


<210>  13
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-3 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  13
cuccaaacug ugaagccaga                                                   20


<210>  14
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-3 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  14
cuucacaguu uggag                                                        15


<210>  15
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-4 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  15
ucuccaaacu gugaagccag                                                   20


<210>  16
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-4 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  16
uucacaguuu ggaga                                                        15


<210>  17
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-5 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  17
gucuccaaac ugugaagcca                                                   20


<210>  18
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-5 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  18
ucacaguuug gagac                                                        15


<210>  19
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-6 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  19
agucuccaaa cugugaagcc                                                   20


<210>  20
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-6 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  20
cacaguuugg agacu                                                        15


<210>  21
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-7 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (2)..(2)
<223>  2'F

<220>
<221>  misc_feature
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (4)..(4)
<223>  2'F

<220>
<221>  misc_feature
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (6)..(6)
<223>  2'F

<220>
<221>  misc_feature
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (8)..(8)
<223>  2'F

<220>
<221>  misc_feature
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (10)..(10)
<223>  2'F

<220>
<221>  misc_feature
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (12)..(12)
<223>  2'F

<220>
<221>  misc_feature
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (14)..(14)
<223>  2'F

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (16)..(16)
<223>  2'F

<220>
<221>  misc_feature
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (18)..(18)
<223>  2'F

<220>
<221>  misc_feature
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (20)..(20)
<223>  2'F

<400>  21
cagucuccaa acugugaagc                                                   20


<210>  22
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-7 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  22
acaguuugga gacug                                                        15


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

<220>
<223>  siRNA SBMA-8 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  23
gcagucucca aacugugaag                                                   20


<210>  24
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-8 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  24
caguuuggag acugc                                                        15


<210>  25
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-9 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  25
ggcagucucc aaacugugaa                                                   20


<210>  26
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-9 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  26
aguuuggaga cugcc                                                        15


<210>  27
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-10 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  27
uggcagucuc caaacuguga                                                   20


<210>  28
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-10 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  28
guuuggagac ugcca                                                        15


<210>  29
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-11 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' phosphate

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  29
cuggcagucu ccaaacugug                                                   20


<210>  30
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA SBMA-11 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' chol

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  30
uuuggagacu gccag                                                        15


<210>  31
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA AR-2 sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' PC-DCA (Phosphocholine Docosanoic Acid)

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  31
ggcuucacag uuugg                                                        15


<210>  32
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  siRNA AR-2 antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' VP (Vinylphosphonate)

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  32
ucaaacugug aagccagagu                                                   20


<210>  33
<211>  15
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Mock sense strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'F

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'F

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'F

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'F

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'F

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'F

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'F

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'OMe

<220>
<221>  misc_feature
<222>  (15)..(15)
<223>  3' PC-DCA (Phosphocholine Docosanoic Acid)

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'F

<400>  33
caguaaagag auuaa                                                        15


<210>  34
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  Mock antisense strand


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  5' VP (Vinylphosphonate)

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'F

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'F

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'F

<220>
<221>  modified_base
<222>  (7)..(7)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (8)..(8)
<223>  2'F

<220>
<221>  modified_base
<222>  (9)..(9)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (10)..(10)
<223>  2'F

<220>
<221>  modified_base
<222>  (11)..(11)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'F

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'F

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'F

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'F

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'OMe

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'F

<400>  34
uuaaucucuu uacugauaua                                                   20


<210>  35
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Polypeptide sequence at the N-terminus of AR2 resulting from the 
       alternative exon 1b

<400>  35

Met Ile Leu Trp Leu His Ser 
1               5           


