                               SEQUENCE LISTING

<110> SANGAMO THERAPEUTICS, INC.
 
<120> IMPROVED CLINICAL PARAMETERS BY EXPRESSION OF FACTOR VIII

<130> 1147465

<140>
<141>

<150> 62/869,445
<151> 2019-07-01

<150> 62/826,887
<151> 2019-03-29

<150> 62/714,553
<151> 2018-08-03

<160> 14    

<170> PatentIn version 3.5

<210> 1
<211> 1457
<212> PRT
<213> Homo sapiens

<400> 1
Met Gln Ile Glu Leu Ser Thr Cys Phe Phe Leu Cys Leu Leu Arg Phe 
1               5                   10                  15      


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


Trp Asp Tyr Met Gln Ser Asp Leu Gly Glu Leu Pro Val Asp Ala Arg 
        35                  40                  45              


Phe Pro Pro Arg Val Pro Lys Ser Phe Pro Phe Asn Thr Ser Val Val 
    50                  55                  60                  


Tyr Lys Lys Thr Leu Phe Val Glu Phe Thr Asp His Leu Phe Asn Ile 
65                  70                  75                  80  


Ala Lys Pro Arg Pro Pro Trp Met Gly Leu Leu Gly Pro Thr Ile Gln 
                85                  90                  95      


Ala Glu Val Tyr Asp Thr Val Val Ile Thr Leu Lys Asn Met Ala Ser 
            100                 105                 110         


His Pro Val Ser Leu His Ala Val Gly Val Ser Tyr Trp Lys Ala Ser 
        115                 120                 125             


Glu Gly Ala Glu Tyr Asp Asp Gln Thr Ser Gln Arg Glu Lys Glu Asp 
    130                 135                 140                 


Asp Lys Val Phe Pro Gly Gly Ser His Thr Tyr Val Trp Gln Val Leu 
145                 150                 155                 160 


Lys Glu Asn Gly Pro Met Ala Ser Asp Pro Leu Cys Leu Thr Tyr Ser 
                165                 170                 175     


Tyr Leu Ser His Val Asp Leu Val Lys Asp Leu Asn Ser Gly Leu Ile 
            180                 185                 190         


Gly Ala Leu Leu Val Cys Arg Glu Gly Ser Leu Ala Lys Glu Lys Thr 
        195                 200                 205             


Gln Thr Leu His Lys Phe Ile Leu Leu Phe Ala Val Phe Asp Glu Gly 
    210                 215                 220                 


Lys Ser Trp His Ser Glu Thr Lys Asn Ser Leu Met Gln Asp Arg Asp 
225                 230                 235                 240 


Ala Ala Ser Ala Arg Ala Trp Pro Lys Met His Thr Val Asn Gly Tyr 
                245                 250                 255     


Val Asn Arg Ser Leu Pro Gly Leu Ile Gly Cys His Arg Lys Ser Val 
            260                 265                 270         


Tyr Trp His Val Ile Gly Met Gly Thr Thr Pro Glu Val His Ser Ile 
        275                 280                 285             


Phe Leu Glu Gly His Thr Phe Leu Val Arg Asn His Arg Gln Ala Ser 
    290                 295                 300                 


Leu Glu Ile Ser Pro Ile Thr Phe Leu Thr Ala Gln Thr Leu Leu Met 
305                 310                 315                 320 


Asp Leu Gly Gln Phe Leu Leu Phe Cys His Ile Ser Ser His Gln His 
                325                 330                 335     


Asp Gly Met Glu Ala Tyr Val Lys Val Asp Ser Cys Pro Glu Glu Pro 
            340                 345                 350         


Gln Leu Arg Met Lys Asn Asn Glu Glu Ala Glu Asp Tyr Asp Asp Asp 
        355                 360                 365             


Leu Thr Asp Ser Glu Met Asp Val Val Arg Phe Asp Asp Asp Asn Ser 
    370                 375                 380                 


Pro Ser Phe Ile Gln Ile Arg Ser Val Ala Lys Lys His Pro Lys Thr 
385                 390                 395                 400 


Trp Val His Tyr Ile Ala Ala Glu Glu Glu Asp Trp Asp Tyr Ala Pro 
                405                 410                 415     


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


Asn Gly Pro Gln Arg Ile Gly Arg Lys Tyr Lys Lys Val Arg Phe Met 
        435                 440                 445             


Ala Tyr Thr Asp Glu Thr Phe Lys Thr Arg Glu Ala Ile Gln His Glu 
    450                 455                 460                 


Ser Gly Ile Leu Gly Pro Leu Leu Tyr Gly Glu Val Gly Asp Thr Leu 
465                 470                 475                 480 


Leu Ile Ile Phe Lys Asn Gln Ala Ser Arg Pro Tyr Asn Ile Tyr Pro 
                485                 490                 495     


His Gly Ile Thr Asp Val Arg Pro Leu Tyr Ser Arg Arg Leu Pro Lys 
            500                 505                 510         


Gly Val Lys His Leu Lys Asp Phe Pro Ile Leu Pro Gly Glu Ile Phe 
        515                 520                 525             


Lys Tyr Lys Trp Thr Val Thr Val Glu Asp Gly Pro Thr Lys Ser Asp 
    530                 535                 540                 


Pro Arg Cys Leu Thr Arg Tyr Tyr Ser Ser Phe Val Asn Met Glu Arg 
545                 550                 555                 560 


Asp Leu Ala Ser Gly Leu Ile Gly Pro Leu Leu Ile Cys Tyr Lys Glu 
                565                 570                 575     


Ser Val Asp Gln Arg Gly Asn Gln Ile Met Ser Asp Lys Arg Asn Val 
            580                 585                 590         


Ile Leu Phe Ser Val Phe Asp Glu Asn Arg Ser Trp Tyr Leu Thr Glu 
        595                 600                 605             


Asn Ile Gln Arg Phe Leu Pro Asn Pro Ala Gly Val Gln Leu Glu Asp 
    610                 615                 620                 


Pro Glu Phe Gln Ala Ser Asn Ile Met His Ser Ile Asn Gly Tyr Val 
625                 630                 635                 640 


Phe Asp Ser Leu Gln Leu Ser Val Cys Leu His Glu Val Ala Tyr Trp 
                645                 650                 655     


Tyr Ile Leu Ser Ile Gly Ala Gln Thr Asp Phe Leu Ser Val Phe Phe 
            660                 665                 670         


Ser Gly Tyr Thr Phe Lys His Lys Met Val Tyr Glu Asp Thr Leu Thr 
        675                 680                 685             


Leu Phe Pro Phe Ser Gly Glu Thr Val Phe Met Ser Met Glu Asn Pro 
    690                 695                 700                 


Gly Leu Trp Ile Leu Gly Cys His Asn Ser Asp Phe Arg Asn Arg Gly 
705                 710                 715                 720 


Met Thr Ala Leu Leu Lys Val Ser Ser Cys Asp Lys Asn Thr Gly Asp 
                725                 730                 735     


Tyr Tyr Glu Asp Ser Tyr Glu Asp Ile Ser Ala Tyr Leu Leu Ser Lys 
            740                 745                 750         


Asn Asn Ala Ile Glu Pro Arg Ser Phe Ser Gln Asn Pro Pro Val Leu 
        755                 760                 765             


Lys Arg His Gln Arg Glu Ile Thr Arg Thr Thr Leu Gln Ser Asp Gln 
    770                 775                 780                 


Glu Glu Ile Asp Tyr Asp Asp Thr Ile Ser Val Glu Met Lys Lys Glu 
785                 790                 795                 800 


Asp Phe Asp Ile Tyr Asp Glu Asp Glu Asn Gln Ser Pro Arg Ser Phe 
                805                 810                 815     


Gln Lys Lys Thr Arg His Tyr Phe Ile Ala Ala Val Glu Arg Leu Trp 
            820                 825                 830         


Asp Tyr Gly Met Ser Ser Ser Pro His Val Leu Arg Asn Arg Ala Gln 
        835                 840                 845             


Ser Gly Ser Val Pro Gln Phe Lys Lys Val Val Phe Gln Glu Phe Thr 
    850                 855                 860                 


Asp Gly Ser Phe Thr Gln Pro Leu Tyr Arg Gly Glu Leu Asn Glu His 
865                 870                 875                 880 


Leu Gly Leu Leu Gly Pro Tyr Ile Arg Ala Glu Val Glu Asp Asn Ile 
                885                 890                 895     


Met Val Thr Phe Arg Asn Gln Ala Ser Arg Pro Tyr Ser Phe Tyr Ser 
            900                 905                 910         


Ser Leu Ile Ser Tyr Glu Glu Asp Gln Arg Gln Gly Ala Glu Pro Arg 
        915                 920                 925             


Lys Asn Phe Val Lys Pro Asn Glu Thr Lys Thr Tyr Phe Trp Lys Val 
    930                 935                 940                 


Gln His His Met Ala Pro Thr Lys Asp Glu Phe Asp Cys Lys Ala Trp 
945                 950                 955                 960 


Ala Tyr Phe Ser Asp Val Asp Leu Glu Lys Asp Val His Ser Gly Leu 
                965                 970                 975     


Ile Gly Pro Leu Leu Val Cys His Thr Asn Thr Leu Asn Pro Ala His 
            980                 985                 990         


Gly Arg Gln Val Thr Val Gln Glu  Phe Ala Leu Phe Phe  Thr Ile Phe 
        995                 1000                 1005             


Asp Glu  Thr Lys Ser Trp Tyr  Phe Thr Glu Asn Met  Glu Arg Asn 
    1010                 1015                 1020             


Cys Arg  Ala Pro Cys Asn Ile  Gln Met Glu Asp Pro  Thr Phe Lys 
    1025                 1030                 1035             


Glu Asn  Tyr Arg Phe His Ala  Ile Asn Gly Tyr Ile  Met Asp Thr 
    1040                 1045                 1050             


Leu Pro  Gly Leu Val Met Ala  Gln Asp Gln Arg Ile  Arg Trp Tyr 
    1055                 1060                 1065             


Leu Leu  Ser Met Gly Ser Asn  Glu Asn Ile His Ser  Ile His Phe 
    1070                 1075                 1080             


Ser Gly  His Val Phe Thr Val  Arg Lys Lys Glu Glu  Tyr Lys Met 
    1085                 1090                 1095             


Ala Leu  Tyr Asn Leu Tyr Pro  Gly Val Phe Glu Thr  Val Glu Met 
    1100                 1105                 1110             


Leu Pro  Ser Lys Ala Gly Ile  Trp Arg Val Glu Cys  Leu Ile Gly 
    1115                 1120                 1125             


Glu His  Leu His Ala Gly Met  Ser Thr Leu Phe Leu  Val Tyr Ser 
    1130                 1135                 1140             


Asn Lys  Cys Gln Thr Pro Leu  Gly Met Ala Ser Gly  His Ile Arg 
    1145                 1150                 1155             


Asp Phe  Gln Ile Thr Ala Ser  Gly Gln Tyr Gly Gln  Trp Ala Pro 
    1160                 1165                 1170             


Lys Leu  Ala Arg Leu His Tyr  Ser Gly Ser Ile Asn  Ala Trp Ser 
    1175                 1180                 1185             


Thr Lys  Glu Pro Phe Ser Trp  Ile Lys Val Asp Leu  Leu Ala Pro 
    1190                 1195                 1200             


Met Ile  Ile His Gly Ile Lys  Thr Gln Gly Ala Arg  Gln Lys Phe 
    1205                 1210                 1215             


Ser Ser  Leu Tyr Ile Ser Gln  Phe Ile Ile Met Tyr  Ser Leu Asp 
    1220                 1225                 1230             


Gly Lys  Lys Trp Gln Thr Tyr  Arg Gly Asn Ser Thr  Gly Thr Leu 
    1235                 1240                 1245             


Met Val  Phe Phe Gly Asn Val  Asp Ser Ser Gly Ile  Lys His Asn 
    1250                 1255                 1260             


Ile Phe  Asn Pro Pro Ile Ile  Ala Arg Tyr Ile Arg  Leu His Pro 
    1265                 1270                 1275             


Thr His  Tyr Ser Ile Arg Ser  Thr Leu Arg Met Glu  Leu Met Gly 
    1280                 1285                 1290             


Cys Asp  Leu Asn Ser Cys Ser  Met Pro Leu Gly Met  Glu Ser Lys 
    1295                 1300                 1305             


Ala Ile  Ser Asp Ala Gln Ile  Thr Ala Ser Ser Tyr  Phe Thr Asn 
    1310                 1315                 1320             


Met Phe  Ala Thr Trp Ser Pro  Ser Lys Ala Arg Leu  His Leu Gln 
    1325                 1330                 1335             


Gly Arg  Ser Asn Ala Trp Arg  Pro Gln Val Asn Asn  Pro Lys Glu 
    1340                 1345                 1350             


Trp Leu  Gln Val Asp Phe Gln  Lys Thr Met Lys Val  Thr Gly Val 
    1355                 1360                 1365             


Thr Thr  Gln Gly Val Lys Ser  Leu Leu Thr Ser Met  Tyr Val Lys 
    1370                 1375                 1380             


Glu Phe  Leu Ile Ser Ser Ser  Gln Asp Gly His Gln  Trp Thr Leu 
    1385                 1390                 1395             


Phe Phe  Gln Asn Gly Lys Val  Lys Val Phe Gln Gly  Asn Gln Asp 
    1400                 1405                 1410             


Ser Phe  Thr Pro Val Val Asn  Ser Leu Asp Pro Pro  Leu Leu Thr 
    1415                 1420                 1425             


Arg Tyr  Leu Arg Ile His Pro  Gln Ser Trp Val His  Gln Ile Ala 
    1430                 1435                 1440             


Leu Arg  Met Glu Val Leu Gly  Cys Glu Ala Gln Asp  Leu Tyr 
    1445                 1450                 1455         


<210> 2
<211> 84
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      SERPIN1 enhancer sequence

<400> 2
gggggaggct gctggtgaat attaaccaag atcaccccag ttaccggagg agcaaacagg       60

gactaagttc acacgcgtgg tacc                                              84


<210> 3
<211> 223
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      TTRm promoter sequence

<400> 3
gtctgtctgc acatttcgta gagcgagtgt tccgatactc taatctccct aggcaaggtt       60

catatttgtg taggttactt attctccttt tgttgactaa gtcaataatc agaatcagca      120

ggtttggagt cagcttggca gggatcagca gcctgggttg gaaggagggg gtataaaagc      180

cccttcacca ggagaagccg tcacacagat ccacaagctc ctg                        223


<210> 4
<211> 4374
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      Coding sequence for FVIII

<400> 4
atgcagatcg agctctccac ctgcttcttt ctgtgcctgt tgagattctg cttcagcgcc       60

accaggagat actacctggg ggctgtggag ctgagctggg actacatgca gtctgacctg      120

ggggagctgc ctgtggatgc caggttcccc cccagagtgc ccaagagctt ccccttcaac      180

acctctgtgg tgtacaagaa gaccctgttt gtggagttca ctgaccacct gttcaacatt      240

gccaagccca ggcccccctg gatgggcctg ctgggcccca ccatccaggc tgaggtgtat      300

gacactgtgg tgatcaccct gaagaacatg gccagccacc ctgtgagcct gcatgctgtg      360

ggggtgagct actggaaggc ctctgagggg gctgagtatg atgaccagac cagccagagg      420

gagaaggagg atgacaaggt gttccctggg ggcagccaca cctatgtgtg gcaggtgctg      480

aaggagaatg gccccatggc ctctgacccc ctgtgcctga cctacagcta cctgagccat      540

gtggacctgg tgaaggacct gaactctggc ctgattgggg ccctgctggt gtgcagggag      600

ggcagcctgg ccaaggagaa gacccagacc ctgcacaagt tcatcctgct gtttgctgtg      660

tttgatgagg gcaagagctg gcactctgaa accaagaaca gcctgatgca ggacagggat      720

gctgcctctg ccagggcctg gcccaagatg cacactgtga atggctatgt gaacaggagc      780

ctgcctggcc tgattggctg ccacaggaag tctgtgtact ggcatgtgat tggcatgggc      840

accacccctg aggtgcacag catcttcctg gagggccaca ccttcctggt caggaaccac      900

aggcaggcca gcctggagat cagccccatc accttcctga ctgcccagac cctgctgatg      960

gacctgggcc agttcctgct gttctgccac atcagcagcc accagcatga tggcatggag     1020

gcctatgtga aggtggacag ctgccctgag gagccccagc tgaggatgaa gaacaatgag     1080

gaggctgagg actatgatga tgacctgact gactctgaga tggatgtggt gaggtttgat     1140

gatgacaaca gccccagctt catccagatc aggtctgtgg ccaagaagca ccccaagacc     1200

tgggtgcact acattgctgc tgaggaggag gactgggact atgcccccct ggtgctggcc     1260

cctgatgaca ggagctacaa gagccagtac ctgaacaatg gcccccagag gattggcagg     1320

aagtacaaga aggtcaggtt catggcctac actgatgaaa ccttcaagac cagggaggcc     1380

atccagcatg agtctggcat cctgggcccc ctgctgtatg gggaggtggg ggacaccctg     1440

ctgatcatct tcaagaacca ggccagcagg ccctacaaca tctaccccca tggcatcact     1500

gatgtgaggc ccctgtacag caggaggctg cccaaggggg tgaagcacct gaaggacttc     1560

cccatcctgc ctggggagat cttcaagtac aagtggactg tgactgtgga ggatggcccc     1620

accaagtctg accccaggtg cctgaccaga tactacagca gctttgtgaa catggagagg     1680

gacctggcct ctggcctgat tggccccctg ctgatctgct acaaggagtc tgtggaccag     1740

aggggcaacc agatcatgtc tgacaagagg aatgtgatcc tgttctctgt gtttgatgag     1800

aacaggagct ggtacctgac tgagaacatc cagaggttcc tgcccaaccc tgctggggtg     1860

cagctggagg accctgagtt ccaggccagc aacatcatgc acagcatcaa tggctatgtg     1920

tttgacagcc tgcagctgtc tgtgtgcctg catgaggtgg cctactggta catcctgagc     1980

attggggccc agactgactt cctgtctgtg ttcttctctg gctacacctt caagcacaag     2040

atggtgtatg aggacaccct gaccctgttc cccttctctg gggagactgt gttcatgagc     2100

atggagaacc ctggcctgtg gattctgggc tgccacaact ctgacttcag gaacaggggc     2160

atgactgccc tgctgaaagt ctccagctgt gacaagaaca ctggggacta ctatgaggac     2220

agctatgagg acatctctgc ctacctgctg agcaagaaca atgccattga gcccaggagc     2280

ttcagccaga atccacccgt ccttaagcgc catcagcgcg agatcaccag gaccaccctg     2340

cagtctgacc aggaggagat tgactatgat gacaccatct ctgtggagat gaagaaggag     2400

gactttgaca tctacgacga ggacgagaac cagagcccca ggagcttcca gaagaagacc     2460

aggcactact tcattgctgc tgtggagagg ctgtgggact atggcatgag cagcagcccc     2520

catgtgctga ggaacagggc ccagtctggc tctgtgcccc agttcaagaa ggtggtgttc     2580

caggagttca ctgatggcag cttcacccag cccctgtaca gaggggagct gaatgagcac     2640

ctgggcctgc tgggccccta catcagggct gaggtggagg acaacatcat ggtgaccttc     2700

aggaaccagg ccagcaggcc ctacagcttc tacagcagcc tgatcagcta tgaggaggac     2760

cagaggcagg gggctgagcc caggaagaac tttgtgaagc ccaatgaaac caagacctac     2820

ttctggaagg tgcagcacca catggccccc accaaggatg agtttgactg caaggcctgg     2880

gcctacttct ctgatgtgga cctggagaag gatgtgcact ctggcctgat tggccccctg     2940

ctggtgtgcc acaccaacac cctgaaccct gcccatggca ggcaggtgac tgtgcaggag     3000

tttgccctgt tcttcaccat ctttgatgaa accaagagct ggtacttcac tgagaacatg     3060

gagaggaact gcagggcccc ctgcaacatc cagatggagg accccacctt caaggagaac     3120

tacaggttcc atgccatcaa tggctacatc atggacaccc tgcctggcct ggtgatggcc     3180

caggaccaga ggatcaggtg gtacctgctg agcatgggca gcaatgagaa catccacagc     3240

atccacttct ctggccatgt gttcactgtg aggaagaagg aggagtacaa gatggccctg     3300

tacaacctgt accctggggt gtttgagact gtggagatgc tgcccagcaa ggctggcatc     3360

tggagggtgg agtgcctgat tggggagcac ctgcatgctg gcatgagcac cctgttcctg     3420

gtgtacagca acaagtgcca gacccccctg ggcatggcct ctggccacat cagggacttc     3480

cagatcactg cctctggcca gtatggccag tgggccccca agctggccag gctgcactac     3540

tctggcagca tcaatgcctg gagcaccaag gagcccttca gctggatcaa ggtggacctg     3600

ctggccccca tgatcatcca tggcatcaag acccaggggg ccaggcagaa gttcagcagc     3660

ctgtacatca gccagttcat catcatgtac agcctggatg gcaagaagtg gcagacctac     3720

aggggcaaca gcactggcac cctgatggtg ttctttggca atgtggacag ctctggcatc     3780

aagcacaaca tcttcaaccc ccccatcatt gccagataca tcaggctgca ccccacccac     3840

tacagcatca ggagcaccct gaggatggag ctgatgggct gtgacctgaa cagctgcagc     3900

atgcccctgg gcatggagag caaggccatc tctgatgccc agatcactgc cagcagctac     3960

ttcaccaaca tgtttgccac ctggagcccc agcaaggcca ggctgcatct gcagggcagg     4020

agcaatgcct ggaggcccca ggtcaacaac cccaaggagt ggctgcaggt ggacttccag     4080

aagaccatga aggtgactgg ggtgaccacc cagggggtga agagcctgct gaccagcatg     4140

tatgtgaagg agttcctgat cagcagcagc caggatggcc accagtggac cctgttcttc     4200

cagaatggca aggtgaaggt gttccagggc aaccaggaca gcttcacccc tgtggtgaac     4260

agcctggacc cccccctgct gaccagatac ctgaggattc acccccagag ctgggtgcac     4320

cagattgccc tgaggatgga ggtgctgggc tgtgaggccc aggacctgta ctga           4374


<210> 5
<211> 4894
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      Expression cassette sequence

<400> 5
gcggcctaag cttggaacca ttgccacctt cagggggagg ctgctggtga atattaacca       60

agatcacccc agttaccgga ggagcaaaca gggactaagt tcacacgcgt ggtaccgtct      120

gtctgcacat ttcgtagagc gagtgttccg atactctaat ctccctaggc aaggttcata      180

tttgtgtagg ttacttattc tccttttgtt gactaagtca ataatcagaa tcagcaggtt      240

tggagtcagc ttggcaggga tcagcagcct gggttggaag gagggggtat aaaagcccct      300

tcaccaggag aagccgtcac acagatccac aagctcctga agaggtaagg gtttaagtta      360

tcgttagttc gtgcaccatt aatgtttaat tacctggagc acctgcctga aatcattttt      420

ttttcaggtt ggctagtatg cagatcgagc tctccacctg cttctttctg tgcctgttga      480

gattctgctt cagcgccacc aggagatact acctgggggc tgtggagctg agctgggact      540

acatgcagtc tgacctgggg gagctgcctg tggatgccag gttccccccc agagtgccca      600

agagcttccc cttcaacacc tctgtggtgt acaagaagac cctgtttgtg gagttcactg      660

accacctgtt caacattgcc aagcccaggc ccccctggat gggcctgctg ggccccacca      720

tccaggctga ggtgtatgac actgtggtga tcaccctgaa gaacatggcc agccaccctg      780

tgagcctgca tgctgtgggg gtgagctact ggaaggcctc tgagggggct gagtatgatg      840

accagaccag ccagagggag aaggaggatg acaaggtgtt ccctgggggc agccacacct      900

atgtgtggca ggtgctgaag gagaatggcc ccatggcctc tgaccccctg tgcctgacct      960

acagctacct gagccatgtg gacctggtga aggacctgaa ctctggcctg attggggccc     1020

tgctggtgtg cagggagggc agcctggcca aggagaagac ccagaccctg cacaagttca     1080

tcctgctgtt tgctgtgttt gatgagggca agagctggca ctctgaaacc aagaacagcc     1140

tgatgcagga cagggatgct gcctctgcca gggcctggcc caagatgcac actgtgaatg     1200

gctatgtgaa caggagcctg cctggcctga ttggctgcca caggaagtct gtgtactggc     1260

atgtgattgg catgggcacc acccctgagg tgcacagcat cttcctggag ggccacacct     1320

tcctggtcag gaaccacagg caggccagcc tggagatcag ccccatcacc ttcctgactg     1380

cccagaccct gctgatggac ctgggccagt tcctgctgtt ctgccacatc agcagccacc     1440

agcatgatgg catggaggcc tatgtgaagg tggacagctg ccctgaggag ccccagctga     1500

ggatgaagaa caatgaggag gctgaggact atgatgatga cctgactgac tctgagatgg     1560

atgtggtgag gtttgatgat gacaacagcc ccagcttcat ccagatcagg tctgtggcca     1620

agaagcaccc caagacctgg gtgcactaca ttgctgctga ggaggaggac tgggactatg     1680

cccccctggt gctggcccct gatgacagga gctacaagag ccagtacctg aacaatggcc     1740

cccagaggat tggcaggaag tacaagaagg tcaggttcat ggcctacact gatgaaacct     1800

tcaagaccag ggaggccatc cagcatgagt ctggcatcct gggccccctg ctgtatgggg     1860

aggtggggga caccctgctg atcatcttca agaaccaggc cagcaggccc tacaacatct     1920

acccccatgg catcactgat gtgaggcccc tgtacagcag gaggctgccc aagggggtga     1980

agcacctgaa ggacttcccc atcctgcctg gggagatctt caagtacaag tggactgtga     2040

ctgtggagga tggccccacc aagtctgacc ccaggtgcct gaccagatac tacagcagct     2100

ttgtgaacat ggagagggac ctggcctctg gcctgattgg ccccctgctg atctgctaca     2160

aggagtctgt ggaccagagg ggcaaccaga tcatgtctga caagaggaat gtgatcctgt     2220

tctctgtgtt tgatgagaac aggagctggt acctgactga gaacatccag aggttcctgc     2280

ccaaccctgc tggggtgcag ctggaggacc ctgagttcca ggccagcaac atcatgcaca     2340

gcatcaatgg ctatgtgttt gacagcctgc agctgtctgt gtgcctgcat gaggtggcct     2400

actggtacat cctgagcatt ggggcccaga ctgacttcct gtctgtgttc ttctctggct     2460

acaccttcaa gcacaagatg gtgtatgagg acaccctgac cctgttcccc ttctctgggg     2520

agactgtgtt catgagcatg gagaaccctg gcctgtggat tctgggctgc cacaactctg     2580

acttcaggaa caggggcatg actgccctgc tgaaagtctc cagctgtgac aagaacactg     2640

gggactacta tgaggacagc tatgaggaca tctctgccta cctgctgagc aagaacaatg     2700

ccattgagcc caggagcttc agccagaatc cacccgtcct taagcgccat cagcgcgaga     2760

tcaccaggac caccctgcag tctgaccagg aggagattga ctatgatgac accatctctg     2820

tggagatgaa gaaggaggac tttgacatct acgacgagga cgagaaccag agccccagga     2880

gcttccagaa gaagaccagg cactacttca ttgctgctgt ggagaggctg tgggactatg     2940

gcatgagcag cagcccccat gtgctgagga acagggccca gtctggctct gtgccccagt     3000

tcaagaaggt ggtgttccag gagttcactg atggcagctt cacccagccc ctgtacagag     3060

gggagctgaa tgagcacctg ggcctgctgg gcccctacat cagggctgag gtggaggaca     3120

acatcatggt gaccttcagg aaccaggcca gcaggcccta cagcttctac agcagcctga     3180

tcagctatga ggaggaccag aggcaggggg ctgagcccag gaagaacttt gtgaagccca     3240

atgaaaccaa gacctacttc tggaaggtgc agcaccacat ggcccccacc aaggatgagt     3300

ttgactgcaa ggcctgggcc tacttctctg atgtggacct ggagaaggat gtgcactctg     3360

gcctgattgg ccccctgctg gtgtgccaca ccaacaccct gaaccctgcc catggcaggc     3420

aggtgactgt gcaggagttt gccctgttct tcaccatctt tgatgaaacc aagagctggt     3480

acttcactga gaacatggag aggaactgca gggccccctg caacatccag atggaggacc     3540

ccaccttcaa ggagaactac aggttccatg ccatcaatgg ctacatcatg gacaccctgc     3600

ctggcctggt gatggcccag gaccagagga tcaggtggta cctgctgagc atgggcagca     3660

atgagaacat ccacagcatc cacttctctg gccatgtgtt cactgtgagg aagaaggagg     3720

agtacaagat ggccctgtac aacctgtacc ctggggtgtt tgagactgtg gagatgctgc     3780

ccagcaaggc tggcatctgg agggtggagt gcctgattgg ggagcacctg catgctggca     3840

tgagcaccct gttcctggtg tacagcaaca agtgccagac ccccctgggc atggcctctg     3900

gccacatcag ggacttccag atcactgcct ctggccagta tggccagtgg gcccccaagc     3960

tggccaggct gcactactct ggcagcatca atgcctggag caccaaggag cccttcagct     4020

ggatcaaggt ggacctgctg gcccccatga tcatccatgg catcaagacc cagggggcca     4080

ggcagaagtt cagcagcctg tacatcagcc agttcatcat catgtacagc ctggatggca     4140

agaagtggca gacctacagg ggcaacagca ctggcaccct gatggtgttc tttggcaatg     4200

tggacagctc tggcatcaag cacaacatct tcaacccccc catcattgcc agatacatca     4260

ggctgcaccc cacccactac agcatcagga gcaccctgag gatggagctg atgggctgtg     4320

acctgaacag ctgcagcatg cccctgggca tggagagcaa ggccatctct gatgcccaga     4380

tcactgccag cagctacttc accaacatgt ttgccacctg gagccccagc aaggccaggc     4440

tgcatctgca gggcaggagc aatgcctgga ggccccaggt caacaacccc aaggagtggc     4500

tgcaggtgga cttccagaag accatgaagg tgactggggt gaccacccag ggggtgaaga     4560

gcctgctgac cagcatgtat gtgaaggagt tcctgatcag cagcagccag gatggccacc     4620

agtggaccct gttcttccag aatggcaagg tgaaggtgtt ccagggcaac caggacagct     4680

tcacccctgt ggtgaacagc ctggaccccc ccctgctgac cagatacctg aggattcacc     4740

cccagagctg ggtgcaccag attgccctga ggatggaggt gctgggctgt gaggcccagg     4800

acctgtactg aggatccaat aaaatatctt tattttcatt acatctgtgt gttggttttt     4860

tgtgtgtttt cctgtaacga tcgggctcga gcgc                                 4894


<210> 6
<211> 23
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      primer

<400> 6
ggagatgaag aaggaggact ttg                                               23


<210> 7
<211> 25
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      probe

<400> 7
acatctacga cgaggacgag aacca                                             25


<210> 8
<211> 21
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      primer

<400> 8
tccacagcag caatgaagta g                                                 21


<210> 9
<211> 18
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      primer

<400> 9
cctgggccag ttcctgct                                                     18


<210> 10
<211> 23
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      probe

<400> 10
ttctgccaca tcagcagcca cca                                               23


<210> 11
<211> 18
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      primer

<400> 11
ggcctccatg ccatcatg                                                     18


<210> 12
<211> 130
<212> DNA
<213> Adeno-associated virus

<400> 12
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct                                                             130


<210> 13
<211> 108
<212> DNA
<213> Adeno-associated virus

<400> 13
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgcccgggc tttgcccggg cggcctcagt gagcgagcga gcgcgcag                   108


<210> 14
<211> 19
<212> PRT
<213> Homo sapiens

<400> 14
Met Gln Ile Glu Leu Ser Thr Cys Phe Phe Leu Cys Leu Leu Arg Phe 
1               5                   10                  15      


Cys Phe Ser 
            



