                         SEQUENCE LISTING

<110>  uniQure biopharma B.V.
 
<120>  Adeno-associated virus vectors modified to bind High-Density 
       Lipoprotein

<130>  P6102741PCT

<150>  EP 21177449.2
<151>  2021-06-02

<150>  EP 21179856.6
<151>  2021-06-16

<160>  25    

<170>  PatentIn version 3.5

<210>  1
<211>  724
<212>  PRT
<213>  adeno-associated virus serotype 5

<400>  1

Met Ser Phe Val Asp His Pro Pro Asp Trp Leu Glu Glu Val Gly Glu 
1               5                   10                  15      


Gly Leu Arg Glu Phe Leu Gly Leu Glu Ala Gly Pro Pro Lys Pro Lys 
            20                  25                  30          


Pro Asn Gln Gln His Gln Asp Gln Ala Arg Gly Leu Val Leu Pro Gly 
        35                  40                  45              


Tyr Asn Tyr Leu Gly Pro Gly Asn Gly Leu Asp Arg Gly Glu Pro Val 
    50                  55                  60                  


Asn Arg Ala Asp Glu Val Ala Arg Glu His Asp Ile Ser Tyr Asn Glu 
65                  70                  75                  80  


Gln Leu Glu Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala Asp 
                85                  90                  95      


Ala Glu Phe Gln Glu Lys Leu Ala Asp Asp Thr Ser Phe Gly Gly Asn 
            100                 105                 110         


Leu Gly Lys Ala Val Phe Gln Ala Lys Lys Arg Val Leu Glu Pro Phe 
        115                 120                 125             


Gly Leu Val Glu Glu Gly Ala Lys Thr Ala Pro Thr Gly Lys Arg Ile 
    130                 135                 140                 


Asp Asp His Phe Pro Lys Arg Lys Lys Ala Arg Thr Glu Glu Asp Ser 
145                 150                 155                 160 


Lys Pro Ser Thr Ser Ser Asp Ala Glu Ala Gly Pro Ser Gly Ser Gln 
                165                 170                 175     


Gln Leu Gln Ile Pro Ala Gln Pro Ala Ser Ser Leu Gly Ala Asp Thr 
            180                 185                 190         


Met Ser Ala Gly Gly Gly Gly Pro Leu Gly Asp Asn Asn Gln Gly Ala 
        195                 200                 205             


Asp Gly Val Gly Asn Ala Ser Gly Asp Trp His Cys Asp Ser Thr Trp 
    210                 215                 220                 


Met Gly Asp Arg Val Val Thr Lys Ser Thr Arg Thr Trp Val Leu Pro 
225                 230                 235                 240 


Ser Tyr Asn Asn His Gln Tyr Arg Glu Ile Lys Ser Gly Ser Val Asp 
                245                 250                 255     


Gly Ser Asn Ala Asn Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr 
            260                 265                 270         


Phe Asp Phe Asn Arg Phe His Ser His Trp Ser Pro Arg Asp Trp Gln 
        275                 280                 285             


Arg Leu Ile Asn Asn Tyr Trp Gly Phe Arg Pro Arg Ser Leu Arg Val 
    290                 295                 300                 


Lys Ile Phe Asn Ile Gln Val Lys Glu Val Thr Val Gln Asp Ser Thr 
305                 310                 315                 320 


Thr Thr Ile Ala Asn Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp 
                325                 330                 335     


Asp Asp Tyr Gln Leu Pro Tyr Val Val Gly Asn Gly Thr Glu Gly Cys 
            340                 345                 350         


Leu Pro Ala Phe Pro Pro Gln Val Phe Thr Leu Pro Gln Tyr Gly Tyr 
        355                 360                 365             


Ala Thr Leu Asn Arg Asp Asn Thr Glu Asn Pro Thr Glu Arg Ser Ser 
    370                 375                 380                 


Phe Phe Cys Leu Glu Tyr Phe Pro Ser Lys Met Leu Arg Thr Gly Asn 
385                 390                 395                 400 


Asn Phe Glu Phe Thr Tyr Asn Phe Glu Glu Val Pro Phe His Ser Ser 
                405                 410                 415     


Phe Ala Pro Ser Gln Asn Leu Phe Lys Leu Ala Asn Pro Leu Val Asp 
            420                 425                 430         


Gln Tyr Leu Tyr Arg Phe Val Ser Thr Asn Asn Thr Gly Gly Val Gln 
        435                 440                 445             


Phe Asn Lys Asn Leu Ala Gly Arg Tyr Ala Asn Thr Tyr Lys Asn Trp 
    450                 455                 460                 


Phe Pro Gly Pro Met Gly Arg Thr Gln Gly Trp Asn Leu Gly Ser Gly 
465                 470                 475                 480 


Val Asn Arg Ala Ser Val Ser Ala Phe Ala Thr Thr Asn Arg Met Glu 
                485                 490                 495     


Leu Glu Gly Ala Ser Tyr Gln Val Pro Pro Gln Pro Asn Gly Met Thr 
            500                 505                 510         


Asn Asn Leu Gln Gly Ser Asn Thr Tyr Ala Leu Glu Asn Thr Met Ile 
        515                 520                 525             


Phe Asn Ser Gln Pro Ala Asn Pro Gly Thr Thr Ala Thr Tyr Leu Glu 
    530                 535                 540                 


Gly Asn Met Leu Ile Thr Ser Glu Ser Glu Thr Gln Pro Val Asn Arg 
545                 550                 555                 560 


Val Ala Tyr Asn Val Gly Gly Gln Met Ala Thr Asn Asn Gln Ser Ser 
                565                 570                 575     


Thr Thr Ala Pro Ala Thr Gly Thr Tyr Asn Leu Gln Glu Ile Val Pro 
            580                 585                 590         


Gly Ser Val Trp Met Glu Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp 
        595                 600                 605             


Ala Lys Ile Pro Glu Thr Gly Ala His Phe His Pro Ser Pro Ala Met 
    610                 615                 620                 


Gly Gly Phe Gly Leu Lys His Pro Pro Pro Met Met Leu Ile Lys Asn 
625                 630                 635                 640 


Thr Pro Val Pro Gly Asn Ile Thr Ser Phe Ser Asp Val Pro Val Ser 
                645                 650                 655     


Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val Thr Val Glu Met Glu 
            660                 665                 670         


Trp Glu Leu Lys Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln 
        675                 680                 685             


Tyr Thr Asn Asn Tyr Asn Asp Pro Gln Phe Val Asp Phe Ala Pro Asp 
    690                 695                 700                 


Ser Thr Gly Glu Tyr Arg Thr Thr Arg Pro Ile Gly Thr Arg Tyr Leu 
705                 710                 715                 720 


Thr Arg Pro Leu 
                


<210>  2
<211>  588
<212>  PRT
<213>  adeno-associated virus serotype 5

<400>  2

Thr Ala Pro Thr Gly Lys Arg Ile Asp Asp His Phe Pro Lys Arg Lys 
1               5                   10                  15      


Lys Ala Arg Thr Glu Glu Asp Ser Lys Pro Ser Thr Ser Ser Asp Ala 
            20                  25                  30          


Glu Ala Gly Pro Ser Gly Ser Gln Gln Leu Gln Ile Pro Ala Gln Pro 
        35                  40                  45              


Ala Ser Ser Leu Gly Ala Asp Thr Met Ser Ala Gly Gly Gly Gly Pro 
    50                  55                  60                  


Leu Gly Asp Asn Asn Gln Gly Ala Asp Gly Val Gly Asn Ala Ser Gly 
65                  70                  75                  80  


Asp Trp His Cys Asp Ser Thr Trp Met Gly Asp Arg Val Val Thr Lys 
                85                  90                  95      


Ser Thr Arg Thr Trp Val Leu Pro Ser Tyr Asn Asn His Gln Tyr Arg 
            100                 105                 110         


Glu Ile Lys Ser Gly Ser Val Asp Gly Ser Asn Ala Asn Ala Tyr Phe 
        115                 120                 125             


Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg Phe His Ser 
    130                 135                 140                 


His Trp Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn Tyr Trp Gly 
145                 150                 155                 160 


Phe Arg Pro Arg Ser Leu Arg Val Lys Ile Phe Asn Ile Gln Val Lys 
                165                 170                 175     


Glu Val Thr Val Gln Asp Ser Thr Thr Thr Ile Ala Asn Asn Leu Thr 
            180                 185                 190         


Ser Thr Val Gln Val Phe Thr Asp Asp Asp Tyr Gln Leu Pro Tyr Val 
        195                 200                 205             


Val Gly Asn Gly Thr Glu Gly Cys Leu Pro Ala Phe Pro Pro Gln Val 
    210                 215                 220                 


Phe Thr Leu Pro Gln Tyr Gly Tyr Ala Thr Leu Asn Arg Asp Asn Thr 
225                 230                 235                 240 


Glu Asn Pro Thr Glu Arg Ser Ser Phe Phe Cys Leu Glu Tyr Phe Pro 
                245                 250                 255     


Ser Lys Met Leu Arg Thr Gly Asn Asn Phe Glu Phe Thr Tyr Asn Phe 
            260                 265                 270         


Glu Glu Val Pro Phe His Ser Ser Phe Ala Pro Ser Gln Asn Leu Phe 
        275                 280                 285             


Lys Leu Ala Asn Pro Leu Val Asp Gln Tyr Leu Tyr Arg Phe Val Ser 
    290                 295                 300                 


Thr Asn Asn Thr Gly Gly Val Gln Phe Asn Lys Asn Leu Ala Gly Arg 
305                 310                 315                 320 


Tyr Ala Asn Thr Tyr Lys Asn Trp Phe Pro Gly Pro Met Gly Arg Thr 
                325                 330                 335     


Gln Gly Trp Asn Leu Gly Ser Gly Val Asn Arg Ala Ser Val Ser Ala 
            340                 345                 350         


Phe Ala Thr Thr Asn Arg Met Glu Leu Glu Gly Ala Ser Tyr Gln Val 
        355                 360                 365             


Pro Pro Gln Pro Asn Gly Met Thr Asn Asn Leu Gln Gly Ser Asn Thr 
    370                 375                 380                 


Tyr Ala Leu Glu Asn Thr Met Ile Phe Asn Ser Gln Pro Ala Asn Pro 
385                 390                 395                 400 


Gly Thr Thr Ala Thr Tyr Leu Glu Gly Asn Met Leu Ile Thr Ser Glu 
                405                 410                 415     


Ser Glu Thr Gln Pro Val Asn Arg Val Ala Tyr Asn Val Gly Gly Gln 
            420                 425                 430         


Met Ala Thr Asn Asn Gln Ser Ser Thr Thr Ala Pro Ala Thr Gly Thr 
        435                 440                 445             


Tyr Asn Leu Gln Glu Ile Val Pro Gly Ser Val Trp Met Glu Arg Asp 
    450                 455                 460                 


Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro Glu Thr Gly Ala 
465                 470                 475                 480 


His Phe His Pro Ser Pro Ala Met Gly Gly Phe Gly Leu Lys His Pro 
                485                 490                 495     


Pro Pro Met Met Leu Ile Lys Asn Thr Pro Val Pro Gly Asn Ile Thr 
            500                 505                 510         


Ser Phe Ser Asp Val Pro Val Ser Ser Phe Ile Thr Gln Tyr Ser Thr 
        515                 520                 525             


Gly Gln Val Thr Val Glu Met Glu Trp Glu Leu Lys Lys Glu Asn Ser 
    530                 535                 540                 


Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Asn Asn Tyr Asn Asp Pro 
545                 550                 555                 560 


Gln Phe Val Asp Phe Ala Pro Asp Ser Thr Gly Glu Tyr Arg Thr Thr 
                565                 570                 575     


Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Pro Leu 
            580                 585             


<210>  3
<211>  532
<212>  PRT
<213>  adeno-associated virus serotype 5

<400>  3

Met Ser Ala Gly Gly Gly Gly Pro Leu Gly Asp Asn Asn Gln Gly Ala 
1               5                   10                  15      


Asp Gly Val Gly Asn Ala Ser Gly Asp Trp His Cys Asp Ser Thr Trp 
            20                  25                  30          


Met Gly Asp Arg Val Val Thr Lys Ser Thr Arg Thr Trp Val Leu Pro 
        35                  40                  45              


Ser Tyr Asn Asn His Gln Tyr Arg Glu Ile Lys Ser Gly Ser Val Asp 
    50                  55                  60                  


Gly Ser Asn Ala Asn Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr 
65                  70                  75                  80  


Phe Asp Phe Asn Arg Phe His Ser His Trp Ser Pro Arg Asp Trp Gln 
                85                  90                  95      


Arg Leu Ile Asn Asn Tyr Trp Gly Phe Arg Pro Arg Ser Leu Arg Val 
            100                 105                 110         


Lys Ile Phe Asn Ile Gln Val Lys Glu Val Thr Val Gln Asp Ser Thr 
        115                 120                 125             


Thr Thr Ile Ala Asn Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp 
    130                 135                 140                 


Asp Asp Tyr Gln Leu Pro Tyr Val Val Gly Asn Gly Thr Glu Gly Cys 
145                 150                 155                 160 


Leu Pro Ala Phe Pro Pro Gln Val Phe Thr Leu Pro Gln Tyr Gly Tyr 
                165                 170                 175     


Ala Thr Leu Asn Arg Asp Asn Thr Glu Asn Pro Thr Glu Arg Ser Ser 
            180                 185                 190         


Phe Phe Cys Leu Glu Tyr Phe Pro Ser Lys Met Leu Arg Thr Gly Asn 
        195                 200                 205             


Asn Phe Glu Phe Thr Tyr Asn Phe Glu Glu Val Pro Phe His Ser Ser 
    210                 215                 220                 


Phe Ala Pro Ser Gln Asn Leu Phe Lys Leu Ala Asn Pro Leu Val Asp 
225                 230                 235                 240 


Gln Tyr Leu Tyr Arg Phe Val Ser Thr Asn Asn Thr Gly Gly Val Gln 
                245                 250                 255     


Phe Asn Lys Asn Leu Ala Gly Arg Tyr Ala Asn Thr Tyr Lys Asn Trp 
            260                 265                 270         


Phe Pro Gly Pro Met Gly Arg Thr Gln Gly Trp Asn Leu Gly Ser Gly 
        275                 280                 285             


Val Asn Arg Ala Ser Val Ser Ala Phe Ala Thr Thr Asn Arg Met Glu 
    290                 295                 300                 


Leu Glu Gly Ala Ser Tyr Gln Val Pro Pro Gln Pro Asn Gly Met Thr 
305                 310                 315                 320 


Asn Asn Leu Gln Gly Ser Asn Thr Tyr Ala Leu Glu Asn Thr Met Ile 
                325                 330                 335     


Phe Asn Ser Gln Pro Ala Asn Pro Gly Thr Thr Ala Thr Tyr Leu Glu 
            340                 345                 350         


Gly Asn Met Leu Ile Thr Ser Glu Ser Glu Thr Gln Pro Val Asn Arg 
        355                 360                 365             


Val Ala Tyr Asn Val Gly Gly Gln Met Ala Thr Asn Asn Gln Ser Ser 
    370                 375                 380                 


Thr Thr Ala Pro Ala Thr Gly Thr Tyr Asn Leu Gln Glu Ile Val Pro 
385                 390                 395                 400 


Gly Ser Val Trp Met Glu Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp 
                405                 410                 415     


Ala Lys Ile Pro Glu Thr Gly Ala His Phe His Pro Ser Pro Ala Met 
            420                 425                 430         


Gly Gly Phe Gly Leu Lys His Pro Pro Pro Met Met Leu Ile Lys Asn 
        435                 440                 445             


Thr Pro Val Pro Gly Asn Ile Thr Ser Phe Ser Asp Val Pro Val Ser 
    450                 455                 460                 


Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val Thr Val Glu Met Glu 
465                 470                 475                 480 


Trp Glu Leu Lys Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln 
                485                 490                 495     


Tyr Thr Asn Asn Tyr Asn Asp Pro Gln Phe Val Asp Phe Ala Pro Asp 
            500                 505                 510         


Ser Thr Gly Glu Tyr Arg Thr Thr Arg Pro Ile Gly Thr Arg Tyr Leu 
        515                 520                 525             


Thr Arg Pro Leu 
    530         


<210>  4
<211>  18
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Flexible linker

<400>  4

Lys Glu Ser Gly Ser Val Ser Ser Glu Gln Leu Ala Gln Phe Arg Ser 
1               5                   10                  15      


Leu Asp 
        


<210>  5
<211>  14
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Flexible linker

<400>  5

Glu Gly Lys Ser Ser Gly Ser Gly Ser Glu Ser Lys Ser Thr 
1               5                   10                  


<210>  6
<211>  8
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  PON1_P1_ApoA1-epitope

<400>  6

Gly Lys Gln Leu Asn Leu Lys Leu 
1               5               


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

<220>
<223>  PON1 P2 ApoA1 epitope

<400>  7

Thr Leu Ser Glu Lys Ala Lys 
1               5           


<210>  8
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  LCAT s108 ApoA1 epitope

<400>  8

Thr Tyr Ser Val Glu Tyr Leu Asp Ser Ser Lys 
1               5                   10      


<210>  9
<211>  6
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  LCAT K249 ApoA1 epitope

<400>  9

Leu Lys Glu Glu Gln Arg 
1               5       


<210>  10
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  ABCA1 ApoA1 epitope 1

<400>  10

Arg Tyr Val Trp Gly Gly Phe Ala Tyr Leu Gln 
1               5                   10      


<210>  11
<211>  9
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  epitope 1 r587

<400>  11

Phe Glu Asp Met Arg Tyr Val Trp Gly 
1               5                   


<210>  12
<211>  9
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  epitpe 1 q597

<400>  12

Phe Ala Tyr Leu Gln Asp Val Val Glu 
1               5                   


<210>  13
<211>  30
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  ABCA1 epitope 2

<400>  13

Cys Pro Pro Gly Ala Gly Gly Leu Pro Pro Pro Gln Arg Lys Gln Asn 
1               5                   10                  15      


Thr Ala Asp Ile Leu Gln Asp Leu Thr Gly Arg Asn Ile Ser 
            20                  25                  30  


<210>  14
<211>  10
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  C1477 surround epitope

<400>  14

Met Leu Pro Val Cys Pro Pro Gly Ala Gly 
1               5                   10  


<210>  15
<211>  9
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  S1506 surround epitope

<400>  15

Gly Arg Asn Ile Ser Asp Tyr Leu Val 
1               5                   


<210>  16
<211>  9
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  apoB LDL-receptor binding epitope

<400>  16

Arg Leu Thr Arg Lys Arg Gly Leu Lys 
1               5                   


<210>  17
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CMV promoter forward primer

<400>  17
aatgggcggt aggcgtgta                                                    19


<210>  18
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CMV promoter reverse primer

<400>  18
aggcgatctg acggttcact aa                                                22


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

<220>
<223>  CMV promoter probe

<400>  19
tgggaggtct atataagcag                                                   20


<210>  20
<211>  461
<212>  PRT
<213>  Homo sapiens

<400>  20

Met Gln Arg Val Asn Met Ile Met Ala Glu Ser Pro Gly Leu Ile Thr 
1               5                   10                  15      


Ile Cys Leu Leu Gly Tyr Leu Leu Ser Ala Glu Cys Thr Val Phe Leu 
            20                  25                  30          


Asp His Glu Asn Ala Asn Lys Ile Leu Asn Arg Pro Lys Arg Tyr Asn 
        35                  40                  45              


Ser Gly Lys Leu Glu Glu Phe Val Gln Gly Asn Leu Glu Arg Glu Cys 
    50                  55                  60                  


Met Glu Glu Lys Cys Ser Phe Glu Glu Ala Arg Glu Val Phe Glu Asn 
65                  70                  75                  80  


Thr Glu Arg Thr Thr Glu Phe Trp Lys Gln Tyr Val Asp Gly Asp Gln 
                85                  90                  95      


Cys Glu Ser Asn Pro Cys Leu Asn Gly Gly Ser Cys Lys Asp Asp Ile 
            100                 105                 110         


Asn Ser Tyr Glu Cys Trp Cys Pro Phe Gly Phe Glu Gly Lys Asn Cys 
        115                 120                 125             


Glu Leu Asp Val Thr Cys Asn Ile Lys Asn Gly Arg Cys Glu Gln Phe 
    130                 135                 140                 


Cys Lys Asn Ser Ala Asp Asn Lys Val Val Cys Ser Cys Thr Glu Gly 
145                 150                 155                 160 


Tyr Arg Leu Ala Glu Asn Gln Lys Ser Cys Glu Pro Ala Val Pro Phe 
                165                 170                 175     


Pro Cys Gly Arg Val Ser Val Ser Gln Thr Ser Lys Leu Thr Arg Ala 
            180                 185                 190         


Glu Ala Val Phe Pro Asp Val Asp Tyr Val Asn Ser Thr Glu Ala Glu 
        195                 200                 205             


Thr Ile Leu Asp Asn Ile Thr Gln Ser Thr Gln Ser Phe Asn Asp Phe 
    210                 215                 220                 


Thr Arg Val Val Gly Gly Glu Asp Ala Lys Pro Gly Gln Phe Pro Trp 
225                 230                 235                 240 


Gln Val Val Leu Asn Gly Lys Val Asp Ala Phe Cys Gly Gly Ser Ile 
                245                 250                 255     


Val Asn Glu Lys Trp Ile Val Thr Ala Ala His Cys Val Glu Thr Gly 
            260                 265                 270         


Val Lys Ile Thr Val Val Ala Gly Glu His Asn Ile Glu Glu Thr Glu 
        275                 280                 285             


His Thr Glu Gln Lys Arg Asn Val Ile Arg Ile Ile Pro His His Asn 
    290                 295                 300                 


Tyr Asn Ala Ala Ile Asn Lys Tyr Asn His Asp Ile Ala Leu Leu Glu 
305                 310                 315                 320 


Leu Asp Glu Pro Leu Val Leu Asn Ser Tyr Val Thr Pro Ile Cys Ile 
                325                 330                 335     


Ala Asp Lys Glu Tyr Thr Asn Ile Phe Leu Lys Phe Gly Ser Gly Tyr 
            340                 345                 350         


Val Ser Gly Trp Gly Arg Val Phe His Lys Gly Arg Ser Ala Leu Val 
        355                 360                 365             


Leu Gln Tyr Leu Arg Val Pro Leu Val Asp Arg Ala Thr Cys Leu Leu 
    370                 375                 380                 


Ser Thr Lys Phe Thr Ile Tyr Asn Asn Met Phe Cys Ala Gly Phe His 
385                 390                 395                 400 


Glu Gly Gly Arg Asp Ser Cys Gln Gly Asp Ser Gly Gly Pro His Val 
                405                 410                 415     


Thr Glu Val Glu Gly Thr Ser Phe Leu Thr Gly Ile Ile Ser Trp Gly 
            420                 425                 430         


Glu Glu Cys Ala Met Lys Gly Lys Tyr Gly Ile Tyr Thr Lys Val Ser 
        435                 440                 445             


Arg Tyr Val Asn Trp Ile Lys Glu Lys Thr Lys Leu Thr 
    450                 455                 460     


<210>  21
<211>  2414
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV vector construct comprising human FIX_Padua coding sequence 
       operably linked to an LP1 promoter and SV40 polyA-site and 
       flanked by wt AAV2 ITRs

<400>  21
tgggccactc cctctctgcg cgctcgctcg ctcactgagg ccgcccgggc aaagcccggg       60

cgtcgggcga cctttggtcg cccggcctca gtgagcgagc gagcgcgcag agagggagtg      120

gccaactcca tcactagggg ttcctggagg ggtggagtcg tgacccctaa aatgggcaaa      180

cattgcaagc agcaaacagc aaacacacag ccctccctgc ctgctgacct tggagctggg      240

gcagaggtca gagacctctc tgggcccatg ccacctccaa catccactcg accccttgga      300

atttcggtgg agaggagcag aggttgtcct ggcgtggttt aggtagtgtg agaggggaat      360

gactcctttc ggtaagtgca gtggaagctg tacactgccc aggcaaagcg tccgggcagc      420

gtaggcgggc gactcagatc ccagccagtg gacttagccc ctgtttgctc ctccgataac      480

tggggtgacc ttggttaata ttcaccagca gcctcccccg ttgcccctct ggatccactg      540

cttaaatacg gacgaggaca gggccctgtc tcctcagctt caggcaccac cactgacctg      600

ggacagtgaa tccggactct aaggtaaata taaaattttt aagtgtataa tgtgttaaac      660

tactgattct aattgtttct ctcttttaga ttccaacctt tggaactgaa ttctagacca      720

ccatgcagag ggtgaacatg atcatggctg agagccctgg cctgatcacc atctgcctgc      780

tgggctacct gctgtctgct gagtgcactg tgttcctgga ccatgagaat gccaacaaga      840

tcctgaacag gcccaagaga tacaactctg gcaagctgga ggagtttgtg cagggcaacc      900

tggagaggga gtgcatggag gagaagtgca gctttgagga ggccagggag gtgtttgaga      960

acactgagag gaccactgag ttctggaagc agtatgtgga tggggaccag tgtgagagca     1020

acccctgcct gaatgggggc agctgcaagg atgacatcaa cagctatgag tgctggtgcc     1080

cctttggctt tgagggcaag aactgtgagc tggatgtgac ctgcaacatc aagaatggca     1140

gatgtgagca gttctgcaag aactctgctg acaacaaggt ggtgtgcagc tgcactgagg     1200

gctacaggct ggctgagaac cagaagagct gtgagcctgc tgtgccattc ccatgtggca     1260

gagtgtctgt gagccagacc agcaagctga ccagggctga ggctgtgttc cctgatgtgg     1320

actatgtgaa cagcactgag gctgaaacca tcctggacaa catcacccag agcacccaga     1380

gcttcaatga cttcaccagg gtggtggggg gggaggatgc caagcctggc cagttcccct     1440

ggcaagtggt gctgaatggc aaggtggatg ccttctgtgg gggcagcatt gtgaatgaga     1500

agtggattgt gactgctgcc cactgtgtgg agactggggt gaagatcact gtggtggctg     1560

gggagcacaa cattgaggag actgagcaca ctgagcagaa gaggaatgtg atcaggatca     1620

tcccccacca caactacaat gctgccatca acaagtacaa ccatgacatt gccctgctgg     1680

agctggatga gcccctggtg ctgaacagct atgtgacccc catctgcatt gctgacaagg     1740

agtacaccaa catcttcctg aagtttggct ctggctatgt gtctggctgg ggcagggtgt     1800

tccacaaggg caggtctgcc ctggtgctgc agtacctgag ggtgcccctg gtggacaggg     1860

ccacctgcct gctgagcacc aagttcacca tctacaacaa catgttctgt gctggcttcc     1920

atgagggggg cagggacagc tgccaggggg actctggggg cccccatgtg actgaggtgg     1980

agggcaccag cttcctgact ggcatcatca gctgggggga ggagtgtgcc atgaagggca     2040

agtatggcat ctacaccaaa gtctccagat atgtgaactg gatcaaggag aagaccaagc     2100

tgacctgact cgatgcttta tttgtgaaat ttgtgatgct attgctttat ttgtaaccat     2160

tataagctgc aataaacaag ttaacaacaa caattgcatt cattttatgt ttcaggttca     2220

gggggaggtg tgggaggttt tttaaactag gtcacgactc cacccctcca ggaaccccta     2280

gtgatggagt tggccactcc ctctctgcgc gctcgctcgc tcactgaggc cgggcgacca     2340

aaggtcgccc gacgcccggg ctttgcccgg gcggcctcag tgagcgagcg agcgcgcaga     2400

gagggagtgg ccca                                                       2414


<210>  22
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEAP forward primer

<400>  22
gtacccagat gactacagcc aag                                               23


<210>  23
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SEAP reverse primer

<400>  23
ggtggatctc gtatttcatg tct                                               23


<210>  24
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV forward primer

<400>  24
aatgggcggt aggcgtgta                                                    19


<210>  25
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV reverse primer

<400>  25
aggcgatctg acggttcact aa                                                22


