                         SEQUENCE LISTING

<110>  GENZYME CORPORATION
       PASSINI, Marco A.
       SHIHABUDDIN, Lamya
       CHENG, Seng H.
 
<120>  GENE THERAPY FOR NEURODEGENERATIVE DISORDERS

<130>  0800-0056.40

<150>  US 61/174,982
<151>  2009-05-02

<150>  US 61/268,059
<151>  2009-06-08

<160>  2     

<170>  PatentIn version 3.5

<210>  1
<211>  885
<212>  DNA
<213>  Homo sapiens


<220>
<221>  misc_feature
<223>  cDNA sequence of human survival motor neuron (SMN1) gene

<400>  1
atggcgatga gcagcggcgg cagtggtggc ggcgtcccgg agcaggagga ttccgtgctg       60

ttccggcgcg gcacaggcca gagcgatgat tctgacattt gggatgatac agcactgata      120

aaagcatatg ataaagctgt ggcttcattt aagcatgctc taaagaatgg tgacatttgt      180

gaaacttcgg gtaaaccaaa aaccacacct aaaagaaaac ctgctaagaa gaataaaagc      240

caaaagaaga atactgcagc ttccttacaa cagtggaaag ttggggacaa atgttctgcc      300

atttggtcag aagacggttg catttaccca gctaccattg cttcaattga ttttaagaga      360

gaaacctgtg ttgtggttta cactggatat ggaaatagag aggagcaaaa tctgtccgat      420

ctactttccc caatctgtga agtagctaat aatatagaac aaaatgctca agagaatgaa      480

aatgaaagcc aagtttcaac agatgaaagt gagaactcca ggtctcctgg aaataaatca      540

gataacatca agcccaaatc tgctccatgg aactcttttc tccctccacc accccccatg      600

ccagggccaa gactgggacc aggaaagcca ggtctaaaat tcaatggccc accaccgcca      660

ccgccaccac caccacccca cttactatca tgctggctgc ctccatttcc ttctggacca      720

ccaataattc ccccaccacc tcccatatgt ccagattctc ttgatgatgc tgatgctttg      780

ggaagtatgt taatttcatg gtacatgagt ggctatcata ctggctatta tatgggtttc      840

agacaaaatc aaaaagaagg aaggtgctca cattccttaa attaa                      885


<210>  2
<211>  294
<212>  PRT
<213>  Homo sapiens


<220>
<221>  misc_feature
<223>  amino acid sequence of human survival motor neuron (SMN1) gene

<400>  2

Met Ala Met Ser Ser Gly Gly Ser Gly Gly Gly Val Pro Glu Gln Glu 
1               5                   10                  15      


Asp Ser Val Leu Phe Arg Arg Gly Thr Gly Gln Ser Asp Asp Ser Asp 
            20                  25                  30          


Ile Trp Asp Asp Thr Ala Leu Ile Lys Ala Tyr Asp Lys Ala Val Ala 
        35                  40                  45              


Ser Phe Lys His Ala Leu Lys Asn Gly Asp Ile Cys Glu Thr Ser Gly 
    50                  55                  60                  


Lys Pro Lys Thr Thr Pro Lys Arg Lys Pro Ala Lys Lys Asn Lys Ser 
65                  70                  75                  80  


Gln Lys Lys Asn Thr Ala Ala Ser Leu Gln Gln Trp Lys Val Gly Asp 
                85                  90                  95      


Lys Cys Ser Ala Ile Trp Ser Glu Asp Gly Cys Ile Tyr Pro Ala Thr 
            100                 105                 110         


Ile Ala Ser Ile Asp Phe Lys Arg Glu Thr Cys Val Val Val Tyr Thr 
        115                 120                 125             


Gly Tyr Gly Asn Arg Glu Glu Gln Asn Leu Ser Asp Leu Leu Ser Pro 
    130                 135                 140                 


Ile Cys Glu Val Ala Asn Asn Ile Glu Gln Asn Ala Gln Glu Asn Glu 
145                 150                 155                 160 


Asn Glu Ser Gln Val Ser Thr Asp Glu Ser Glu Asn Ser Arg Ser Pro 
                165                 170                 175     


Gly Asn Lys Ser Asp Asn Ile Lys Pro Lys Ser Ala Pro Trp Asn Ser 
            180                 185                 190         


Phe Leu Pro Pro Pro Pro Pro Met Pro Gly Pro Arg Leu Gly Pro Gly 
        195                 200                 205             


Lys Pro Gly Leu Lys Phe Asn Gly Pro Pro Pro Pro Pro Pro Pro Pro 
    210                 215                 220                 


Pro Pro His Leu Leu Ser Cys Trp Leu Pro Pro Phe Pro Ser Gly Pro 
225                 230                 235                 240 


Pro Ile Ile Pro Pro Pro Pro Pro Ile Cys Pro Asp Ser Leu Asp Asp 
                245                 250                 255     


Ala Asp Ala Leu Gly Ser Met Leu Ile Ser Trp Tyr Met Ser Gly Tyr 
            260                 265                 270         


His Thr Gly Tyr Tyr Met Gly Phe Arg Gln Asn Gln Lys Glu Gly Arg 
        275                 280                 285             


Cys Ser His Ser Leu Asn 
    290                 


