
                                SEQUENCE LISTING

<110> GENZYME CORPORATION

<120> ADENO-ASSOCIATED VIRAL VECTORS FOR
  TREATING MUCOLIPIDOSIS TYPE II
  

<130> 159792012640

<140> Not Yet Assigned    
<141> Concurrently Herewith            

<150> US 62/267,502       
<151> 2015-12-15  

<160> 2

<170> FastSEQ for Windows Version 4.0

<210> 1
<211> 1256
<212> PRT
<213> Homo sapiens

<400> 1
Met Leu Phe Lys Leu Leu Gln Arg Gln Thr Tyr Thr Cys Leu Ser His
 1               5                  10                  15      
Arg Tyr Gly Leu Tyr Val Cys Phe Leu Gly Val Val Val Thr Ile Val
            20                  25                  30          
Ser Ala Phe Gln Phe Gly Glu Val Val Leu Glu Trp Ser Arg Asp Gln
        35                  40                  45              
Tyr His Val Leu Phe Asp Ser Tyr Arg Asp Asn Ile Ala Gly Lys Ser
    50                  55                  60                  
Phe Gln Asn Arg Leu Cys Leu Pro Met Pro Ile Asp Val Val Tyr Thr
65                  70                  75                  80  
Trp Val Asn Gly Thr Asp Leu Glu Leu Leu Lys Glu Leu Gln Gln Val
                85                  90                  95      
Arg Glu Gln Met Glu Glu Glu Gln Lys Ala Met Arg Glu Ile Leu Gly
            100                 105                 110         
Lys Asn Thr Thr Glu Pro Thr Lys Lys Ser Glu Lys Gln Leu Glu Cys
        115                 120                 125             
Leu Leu Thr His Cys Ile Lys Val Pro Met Leu Val Leu Asp Pro Ala
    130                 135                 140                 
Leu Pro Ala Asn Ile Thr Leu Lys Asp Leu Pro Ser Leu Tyr Pro Ser
145                 150                 155                 160 
Phe His Ser Ala Ser Asp Ile Phe Asn Val Ala Lys Pro Lys Asn Pro
                165                 170                 175     
Ser Thr Asn Val Ser Val Val Val Phe Asp Ser Thr Lys Asp Val Glu
            180                 185                 190         
Asp Ala His Ser Gly Leu Leu Lys Gly Asn Ser Arg Gln Thr Val Trp
        195                 200                 205             
Arg Gly Tyr Leu Thr Thr Asp Lys Glu Val Pro Gly Leu Val Leu Met
    210                 215                 220                 
Gln Asp Leu Ala Phe Leu Ser Gly Phe Pro Pro Thr Phe Lys Glu Thr
225                 230                 235                 240 
Asn Gln Leu Lys Thr Lys Leu Pro Glu Asn Leu Ser Ser Lys Val Lys
                245                 250                 255     
Leu Leu Gln Leu Tyr Ser Glu Ala Ser Val Ala Leu Leu Lys Leu Asn
            260                 265                 270         
Asn Pro Lys Asp Phe Gln Glu Leu Asn Lys Gln Thr Lys Lys Asn Met
        275                 280                 285             
Thr Ile Asp Gly Lys Glu Leu Thr Ile Ser Pro Ala Tyr Leu Leu Trp
    290                 295                 300                 
Asp Leu Ser Ala Ile Ser Gln Ser Lys Gln Asp Glu Asp Ile Ser Ala
305                 310                 315                 320 
Ser Arg Phe Glu Asp Asn Glu Glu Leu Arg Tyr Ser Leu Arg Ser Ile
                325                 330                 335     
Glu Arg His Ala Pro Trp Val Arg Asn Ile Phe Ile Val Thr Asn Gly
            340                 345                 350         
Gln Ile Pro Ser Trp Leu Asn Leu Asp Asn Pro Arg Val Thr Ile Val
        355                 360                 365             
Thr His Gln Asp Val Phe Arg Asn Leu Ser His Leu Pro Thr Phe Ser
    370                 375                 380                 
Ser Pro Ala Ile Glu Ser His Ile His Arg Ile Glu Gly Leu Ser Gln
385                 390                 395                 400 
Lys Phe Ile Tyr Leu Asn Asp Asp Val Met Phe Gly Lys Asp Val Trp
                405                 410                 415     
Pro Asp Asp Phe Tyr Ser His Ser Lys Gly Gln Lys Val Tyr Leu Thr
            420                 425                 430         
Trp Pro Val Pro Asn Cys Ala Glu Gly Cys Pro Gly Ser Trp Ile Lys
        435                 440                 445             
Asp Gly Tyr Cys Asp Lys Ala Cys Asn Asn Ser Ala Cys Asp Trp Asp
    450                 455                 460                 
Gly Gly Asp Cys Ser Gly Asn Ser Gly Gly Ser Arg Tyr Ile Ala Gly
465                 470                 475                 480 
Gly Gly Gly Thr Gly Ser Ile Gly Val Gly Gln Pro Trp Gln Phe Gly
                485                 490                 495     
Gly Gly Ile Asn Ser Val Ser Tyr Cys Asn Gln Gly Cys Ala Asn Ser
            500                 505                 510         
Trp Leu Ala Asp Lys Phe Cys Asp Gln Ala Cys Asn Val Leu Ser Cys
        515                 520                 525             
Gly Phe Asp Ala Gly Asp Cys Gly Gln Asp His Phe His Glu Leu Tyr
    530                 535                 540                 
Lys Val Ile Leu Leu Pro Asn Gln Thr His Tyr Ile Ile Pro Lys Gly
545                 550                 555                 560 
Glu Cys Leu Pro Tyr Phe Ser Phe Ala Glu Val Ala Lys Arg Gly Val
                565                 570                 575     
Glu Gly Ala Tyr Ser Asp Asn Pro Ile Ile Arg His Ala Ser Ile Ala
            580                 585                 590         
Asn Lys Trp Lys Thr Ile His Leu Ile Met His Ser Gly Met Asn Ala
        595                 600                 605             
Thr Thr Ile His Phe Asn Leu Thr Phe Gln Asn Thr Asn Asp Glu Glu
    610                 615                 620                 
Phe Lys Met Gln Ile Thr Val Glu Val Asp Thr Arg Glu Gly Pro Lys
625                 630                 635                 640 
Leu Asn Ser Thr Ala Gln Lys Gly Tyr Glu Asn Leu Val Ser Pro Ile
                645                 650                 655     
Thr Leu Leu Pro Glu Ala Glu Ile Leu Phe Glu Asp Ile Pro Lys Glu
            660                 665                 670         
Lys Arg Phe Pro Lys Phe Lys Arg His Asp Val Asn Ser Thr Arg Arg
        675                 680                 685             
Ala Gln Glu Glu Val Lys Ile Pro Leu Val Asn Ile Ser Leu Leu Pro
    690                 695                 700                 
Lys Asp Ala Gln Leu Ser Leu Asn Thr Leu Asp Leu Gln Leu Glu His
705                 710                 715                 720 
Gly Asp Ile Thr Leu Lys Gly Tyr Asn Leu Ser Lys Ser Ala Leu Leu
                725                 730                 735     
Arg Ser Phe Leu Met Asn Ser Gln His Ala Lys Ile Lys Asn Gln Ala
            740                 745                 750         
Ile Ile Thr Asp Glu Thr Asn Asp Ser Leu Val Ala Pro Gln Glu Lys
        755                 760                 765             
Gln Val His Lys Ser Ile Leu Pro Asn Ser Leu Gly Val Ser Glu Arg
    770                 775                 780                 
Leu Gln Arg Leu Thr Phe Pro Ala Val Ser Val Lys Val Asn Gly His
785                 790                 795                 800 
Asp Gln Gly Gln Asn Pro Pro Leu Asp Leu Glu Thr Thr Ala Arg Phe
                805                 810                 815     
Arg Val Glu Thr His Thr Gln Lys Thr Ile Gly Gly Asn Val Thr Lys
            820                 825                 830         
Glu Lys Pro Pro Ser Leu Ile Val Pro Leu Glu Ser Gln Met Thr Lys
        835                 840                 845             
Glu Lys Lys Ile Thr Gly Lys Glu Lys Glu Asn Ser Arg Met Glu Glu
    850                 855                 860                 
Asn Ala Glu Asn His Ile Gly Val Thr Glu Val Leu Leu Gly Arg Lys
865                 870                 875                 880 
Leu Gln His Tyr Thr Asp Ser Tyr Leu Gly Phe Leu Pro Trp Glu Lys
                885                 890                 895     
Lys Lys Tyr Phe Gln Asp Leu Leu Asp Glu Glu Glu Ser Leu Lys Thr
            900                 905                 910         
Gln Leu Ala Tyr Phe Thr Asp Ser Lys Asn Thr Gly Arg Gln Leu Lys
        915                 920                 925             
Asp Thr Phe Ala Asp Ser Leu Arg Tyr Val Asn Lys Ile Leu Asn Ser
    930                 935                 940                 
Lys Phe Gly Phe Thr Ser Arg Lys Val Pro Ala His Met Pro His Met
945                 950                 955                 960 
Ile Asp Arg Ile Val Met Gln Glu Leu Gln Asp Met Phe Pro Glu Glu
                965                 970                 975     
Phe Asp Lys Thr Ser Phe His Lys Val Arg His Ser Glu Asp Met Gln
            980                 985                 990         
Phe Ala Phe Ser Tyr Phe Tyr Tyr Leu Met Ser Ala Val Gln Pro Leu
        995                 1000                1005            
Asn Ile Ser Gln Val Phe Asp Glu Val Asp Thr Asp Gln Ser Gly Val
    1010                1015                1020                
Leu Ser Asp Arg Glu Ile Arg Thr Leu Ala Thr Arg Ile His Glu Leu
1025                1030                1035               1040 
Pro Leu Ser Leu Gln Asp Leu Thr Gly Leu Glu His Met Leu Ile Asn
                1045                1050                1055    
Cys Ser Lys Met Leu Pro Ala Asp Ile Thr Gln Leu Asn Asn Ile Pro
            1060                1065                1070        
Pro Thr Gln Glu Ser Tyr Tyr Asp Pro Asn Leu Pro Pro Val Thr Lys
        1075                1080                1085            
Ser Leu Val Thr Asn Cys Lys Pro Val Thr Asp Lys Ile His Lys Ala
    1090                1095                1100                
Tyr Lys Asp Lys Asn Lys Tyr Arg Phe Glu Ile Met Gly Glu Glu Glu
1105                1110                1115               1120 
Ile Ala Phe Lys Met Ile Arg Thr Asn Val Ser His Val Val Gly Gln
                1125                1130                1135    
Leu Asp Asp Ile Arg Lys Asn Pro Arg Lys Phe Val Cys Leu Asn Asp
            1140                1145                1150        
Asn Ile Asp His Asn His Lys Asp Ala Gln Thr Val Lys Ala Val Leu
        1155                1160                1165            
Arg Asp Phe Tyr Glu Ser Met Phe Pro Ile Pro Ser Gln Phe Glu Leu
    1170                1175                1180                
Pro Arg Glu Tyr Arg Asn Arg Phe Leu His Met His Glu Leu Gln Glu
1185                1190                1195               1200 
Trp Arg Ala Tyr Arg Asp Lys Leu Lys Phe Trp Thr His Cys Val Leu
                1205                1210                1215    
Ala Thr Leu Ile Met Phe Thr Ile Phe Ser Phe Phe Ala Glu Gln Leu
            1220                1225                1230        
Ile Ala Leu Lys Arg Lys Ile Phe Pro Arg Arg Arg Ile His Lys Glu
        1235                1240                1245            
Ala Ser Pro Asn Arg Ile Arg Val
    1250                1255    


<210> 2
<211> 1235
<212> PRT
<213> Mus musculus

<400> 2
Met Leu Leu Lys Leu Leu Gln Arg Gln Thr Tyr Thr Cys Leu Ser His
 1               5                  10                  15      
Arg Tyr Gly Leu Tyr Val Cys Phe Val Gly Val Val Val Thr Ile Val
            20                  25                  30          
Ser Ala Phe Gln Phe Gly Glu Val Val Leu Glu Trp Ser Arg Asp Gln
        35                  40                  45              
Tyr His Val Leu Phe Asp Ser Tyr Arg Asp Asn Ile Ala Gly Lys Ser
    50                  55                  60                  
Phe Gln Asn Arg Leu Cys Leu Pro Met Pro Ile Asp Val Val Tyr Thr
65                  70                  75                  80  
Trp Val Asn Gly Thr Asp Leu Glu Leu Leu Lys Glu Leu Gln Gln Val
                85                  90                  95      
Arg Glu His Met Glu Glu Glu Gln Arg Ala Met Arg Glu Thr Leu Gly
            100                 105                 110         
Lys Asn Thr Thr Glu Pro Thr Lys Lys Ser Glu Lys Gln Leu Glu Cys
        115                 120                 125             
Leu Leu Thr His Cys Ile Lys Val Pro Met Leu Val Leu Asp Pro Pro
    130                 135                 140                 
Leu Pro Ala Asn Cys Thr Leu Lys Asp Leu Pro Thr Leu Tyr Pro Ser
145                 150                 155                 160 
Phe His Ala Ala Ser Asp Met Phe Asn Val Ala Lys Pro Lys Asn Pro
                165                 170                 175     
Ser Thr Asn Val Ser Val Val Val Phe Asp Thr Thr Lys Asp Val Glu
            180                 185                 190         
Asp Ala His Ala Gly Pro Phe Lys Gly Gly Ser Lys Gln Met Val Trp
        195                 200                 205             
Arg Ala Tyr Leu Thr Thr Asp Lys Glu Ala Pro Gly Leu Val Leu Met
    210                 215                 220                 
Gln Gly Leu Ala Phe Leu Ser Gly Phe Pro Pro Thr Phe Lys Glu Thr
225                 230                 235                 240 
Ser Gln Leu Lys Thr Lys Leu Pro Glu Lys Leu Ser Ser Lys Ile Lys
                245                 250                 255     
Leu Leu Arg Leu Tyr Ser Glu Ala Ser Val Ala Leu Leu Lys Leu Asn
            260                 265                 270         
Asn Pro Lys Gly Phe Gln Glu Leu Asn Lys Gln Thr Lys Lys Asn Met
        275                 280                 285             
Thr Ile Asp Gly Lys Glu Leu Thr Ile Ser Pro Ala Tyr Leu Leu Trp
    290                 295                 300                 
Asp Leu Ser Ala Ile Ser Gln Ser Lys Gln Asp Glu Asp Val Ser Ala
305                 310                 315                 320 
Ser Arg Phe Glu Asp Asn Glu Glu Leu Arg Tyr Ser Leu Arg Ser Ile
                325                 330                 335     
Glu Arg His Ala Pro Trp Val Arg Asn Ile Phe Ile Val Thr Asn Gly
            340                 345                 350         
Gln Ile Pro Ser Trp Leu Asn Leu Asp Asn Pro Arg Val Thr Ile Val
        355                 360                 365             
Thr His Gln Asp Ile Phe Gln Asn Leu Ser His Leu Pro Thr Phe Ser
    370                 375                 380                 
Ser Pro Ala Ile Glu Ser His Ile His Arg Ile Glu Gly Leu Ser Gln
385                 390                 395                 400 
Lys Phe Ile Tyr Leu Asn Asp Asp Val Met Phe Gly Lys Asp Val Trp
                405                 410                 415     
Pro Asp Asp Phe Tyr Ser His Ser Lys Gly Gln Lys Val Tyr Leu Thr
            420                 425                 430         
Trp Pro Val Pro Asn Cys Ala Glu Gly Cys Pro Gly Ser Trp Ile Lys
        435                 440                 445             
Asp Gly Tyr Cys Asp Lys Ala Cys Asn Asn Ser Ala Cys Asp Trp Asp
    450                 455                 460                 
Gly Gly Asp Cys Ser Gly Asn Thr Ala Gly Asn Arg Phe Val Ala Gly
465                 470                 475                 480 
Gly Gly Gly Thr Gly Asn Ile Gly Ala Gly Gln His Trp Gln Phe Gly
                485                 490                 495     
Gly Gly Ile Asn Thr Ile Ser Tyr Cys Asn Gln Gly Cys Ala Asn Ser
            500                 505                 510         
Trp Leu Ala Asp Lys Phe Cys Asp Gln Ala Cys Asn Val Leu Ser Cys
        515                 520                 525             
Gly Phe Asp Ala Gly Asp Cys Gly Gln Asp His Phe His Glu Leu Tyr
    530                 535                 540                 
Lys Val Thr Leu Leu Pro Asn Gln Thr His Tyr Val Val Pro Lys Gly
545                 550                 555                 560 
Glu Tyr Leu Ser Tyr Phe Ser Phe Ala Asn Ile Ala Arg Arg Gly Val
                565                 570                 575     
Glu Gly Thr Tyr Ser Asp Asn Pro Ile Ile Arg His Ala Ser Ile Ala
            580                 585                 590         
Asn Lys Trp Lys Thr Ile His Leu Ile Met His Ser Gly Met Asn Ala
        595                 600                 605             
Thr Thr Ile Tyr Phe Asn Leu Thr Leu Gln Asn Ala Asn Asp Glu Glu
    610                 615                 620                 
Phe Lys Ile Gln Ile Ala Val Glu Val Asp Thr Arg Glu Ala Pro Lys
625                 630                 635                 640 
Leu Asn Ser Thr Thr Gln Lys Ala Tyr Glu Ser Leu Val Ser Pro Val
                645                 650                 655     
Thr Pro Leu Pro Gln Ala Asp Val Pro Phe Glu Asp Val Pro Lys Glu
            660                 665                 670         
Lys Arg Phe Pro Lys Ile Arg Arg His Asp Val Asn Ala Thr Gly Arg
        675                 680                 685             
Phe Gln Glu Glu Val Lys Ile Pro Arg Val Asn Ile Ser Leu Leu Pro
    690                 695                 700                 
Lys Glu Ala Gln Val Arg Leu Ser Asn Leu Asp Leu Gln Leu Glu Arg
705                 710                 715                 720 
Gly Asp Ile Thr Leu Lys Gly Tyr Asn Leu Ser Lys Ser Ala Leu Leu
                725                 730                 735     
Arg Ser Phe Leu Gly Asn Ser Leu Asp Thr Lys Ile Lys Pro Gln Ala
            740                 745                 750         
Arg Thr Asp Glu Thr Lys Gly Asn Leu Glu Val Pro Gln Glu Asn Pro
        755                 760                 765             
Ser His Arg Arg Pro His Gly Phe Ala Gly Glu His Arg Ser Glu Arg
    770                 775                 780                 
Trp Thr Ala Pro Ala Glu Thr Val Thr Val Lys Gly Arg Asp His Ala
785                 790                 795                 800 
Leu Asn Pro Pro Pro Val Leu Glu Thr Asn Ala Arg Leu Ala Gln Pro
                805                 810                 815     
Thr Leu Gly Val Thr Val Ser Lys Glu Asn Leu Ser Pro Leu Ile Val
            820                 825                 830         
Pro Pro Glu Ser His Leu Pro Lys Glu Glu Glu Ser Asp Arg Ala Glu
        835                 840                 845             
Gly Asn Ala Val Pro Val Lys Glu Leu Val Pro Gly Arg Arg Leu Gln
    850                 855                 860                 
Gln Asn Tyr Pro Gly Phe Leu Pro Trp Glu Lys Lys Lys Tyr Phe Gln
865                 870                 875                 880 
Asp Leu Leu Asp Glu Glu Glu Ser Leu Lys Thr Gln Leu Ala Tyr Phe
                885                 890                 895     
Thr Asp Ser Lys His Thr Gly Arg Gln Leu Lys Asp Thr Phe Ala Asp
            900                 905                 910         
Ser Leu Arg Tyr Val Asn Lys Ile Leu Asn Ser Lys Phe Gly Phe Thr
        915                 920                 925             
Ser Arg Lys Val Pro Ala His Met Pro His Met Ile Asp Arg Ile Val
    930                 935                 940                 
Met Gln Glu Leu Gln Asp Met Phe Pro Glu Glu Phe Asp Lys Thr Ser
945                 950                 955                 960 
Phe His Lys Val Arg His Ser Glu Asp Met Gln Phe Ala Phe Ser Tyr
                965                 970                 975     
Phe Tyr Tyr Leu Met Ser Ala Val Gln Pro Leu Asn Ile Ser Gln Val
            980                 985                 990         
Phe His Glu Val Asp Thr Asp Gln Ser Gly Val Leu Ser Asp Arg Glu
        995                 1000                1005            
Ile Arg Thr Leu Ala Thr Arg Ile His Asp Leu Pro Leu Ser Leu Gln
    1010                1015                1020                
Asp Leu Thr Gly Leu Glu His Met Leu Ile Asn Cys Ser Lys Met Leu
1025                1030                1035               1040 
Pro Ala Asn Ile Thr Gln Leu Asn Asn Ile Pro Pro Thr Gln Glu Ala
                1045                1050                1055    
Tyr Tyr Asp Pro Asn Leu Pro Pro Val Thr Lys Ser Leu Val Thr Asn
            1060                1065                1070        
Cys Lys Pro Val Thr Asp Lys Ile His Lys Ala Tyr Lys Asp Lys Asn
        1075                1080                1085            
Lys Tyr Arg Phe Glu Ile Met Gly Glu Glu Glu Ile Ala Phe Lys Met
    1090                1095                1100                
Ile Arg Thr Asn Val Ser His Val Val Gly Gln Leu Asp Asp Ile Arg
1105                1110                1115               1120 
Lys Asn Pro Arg Lys Phe Val Cys Leu Asn Asp Asn Ile Asp His Asn
                1125                1130                1135    
His Lys Asp Ala Arg Thr Val Lys Ala Val Leu Arg Asp Phe Tyr Glu
            1140                1145                1150        
Ser Met Phe Pro Ile Pro Ser Gln Phe Glu Leu Pro Arg Glu Tyr Arg
        1155                1160                1165            
Asn Arg Phe Leu His Met His Glu Leu Gln Glu Trp Arg Ala Tyr Arg
    1170                1175                1180                
Asp Lys Leu Lys Phe Trp Thr His Cys Val Leu Ala Thr Leu Ile Ile
1185                1190                1195               1200 
Phe Thr Ile Phe Ser Phe Phe Ala Glu Gln Ile Ile Ala Leu Lys Arg
                1205                1210                1215    
Lys Ile Phe Pro Arg Arg Arg Ile His Lys Glu Ala Ser Pro Asp Arg
            1220                1225                1230        
Ile Arg Val
        1235

