                         SEQUENCE LISTING

<110>  University of Massachusetts
 
<120>  TRANSGENIC EXPRESSION OF DNASE I IN VIVO DELIVERED BY AN 
       ADENO-ASSOCIATED VIRUS VECTOR

<130>  U0120.70071WO00

<150>  62/198,100
<151>  2015-07-28

<160>  10    

<170>  PatentIn version 3.5

<210>  1
<211>  736
<212>  PRT
<213>  Adeno-associated virus 9

<400>  1

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


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


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


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


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


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


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


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ala Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Ala Gln Ala Gln 
            580                 585                 590         


Thr Gly Trp Val Gln Asn Gln Gly Ile Leu Pro Gly Met Val Trp Gln 
        595                 600                 605             


Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His 
    610                 615                 620                 


Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Met 
625                 630                 635                 640 


Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala 
                645                 650                 655     


Asp Pro Pro Thr Ala Phe Asn Lys Asp Lys Leu Asn Ser Phe Ile Thr 
            660                 665                 670         


Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln 
        675                 680                 685             


Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Ser Asn 
    690                 695                 700                 


Tyr Tyr Lys Ser Asn Asn Val Glu Phe Ala Val Asn Thr Glu Gly Val 
705                 710                 715                 720 


Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
                725                 730                 735     


<210>  2
<211>  855
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic polynucleotide

<400>  2
atgcggtaca cagggctaat gggaacactg ctcaccttgg tcaacctgct gcagctggct       60

gggactctga gaattgcagc cttcaacatt cggacttttg gggagactaa gatgtccaat      120

gctaccctct ctgtatactt tgtgaaaatc ctgagtcgct atgacatcgc tgttatccaa      180

gaggtcagag actcccacct ggttgctgtt gggaagctcc tggatgaact caatcgggac      240

aaacctgaca cctaccgcta tgtagtcagt gagccgctgg gccgcaaaag ctacaaggaa      300

cagtaccttt ttgtgtacag gcctgaccag gtgtctattc tggacagcta tcaatatgat      360

gatggctgtg aaccctgtgg aaatgacacc ttcagcagag agccagccat tgttaagttc      420

ttttccccat acactgaggt ccaagaattt gcgatcgtgc ccttgcatgc agccccaaca      480

gaagctgtga gtgagatcga cgccctctac gatgtttacc tagatgtctg gcaaaagtgg      540

ggcctggagg acatcatgtt catgggagac ttcaatgctg gctgcagcta cgtcacttcc      600

tcccagtggt cctccattcg ccttcggaca agccccatct tccagtggct gatccctgac      660

agtgcggaca ccacagtcac atcaacacac tgtgcttatg acaggattgt ggttgctgga      720

gctctgctcc aggctgctgt tgttcccaac tcggctgttc cttttgattt ccaagcagaa      780

tacggacttt ccaaccagct ggctgaagcc atcagtgacc attacccagt ggaggtgaca      840

ctcagaaaaa tctga                                                       855


<210>  3
<211>  284
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic polypeptide

<400>  3

Met Arg Tyr Thr Gly Leu Met Gly Thr Leu Leu Thr Leu Val Asn Leu 
1               5                   10                  15      


Leu Gln Leu Ala Gly Thr Leu Arg Ile Ala Ala Phe Asn Ile Arg Thr 
            20                  25                  30          


Phe Gly Glu Thr Lys Met Ser Asn Ala Thr Leu Ser Val Tyr Phe Val 
        35                  40                  45              


Lys Ile Leu Ser Arg Tyr Asp Ile Ala Val Ile Gln Glu Val Arg Asp 
    50                  55                  60                  


Ser His Leu Val Ala Val Gly Lys Leu Leu Asp Glu Leu Asn Arg Asp 
65                  70                  75                  80  


Lys Pro Asp Thr Tyr Arg Tyr Val Val Ser Glu Pro Leu Gly Arg Lys 
                85                  90                  95      


Ser Tyr Lys Glu Gln Tyr Leu Phe Val Tyr Arg Pro Asp Gln Val Ser 
            100                 105                 110         


Ile Leu Asp Ser Tyr Gln Tyr Asp Asp Gly Cys Glu Pro Cys Gly Asn 
        115                 120                 125             


Asp Thr Phe Ser Arg Glu Pro Ala Ile Val Lys Phe Phe Ser Pro Tyr 
    130                 135                 140                 


Thr Glu Val Gln Glu Phe Ala Ile Val Pro Leu His Ala Ala Pro Thr 
145                 150                 155                 160 


Glu Ala Val Ser Glu Ile Asp Ala Leu Tyr Asp Val Tyr Leu Asp Val 
                165                 170                 175     


Trp Gln Lys Trp Gly Leu Glu Asp Ile Met Phe Met Gly Asp Phe Asn 
            180                 185                 190         


Ala Gly Cys Ser Tyr Val Thr Ser Ser Gln Trp Ser Ser Ile Arg Leu 
        195                 200                 205             


Arg Thr Ser Pro Ile Phe Gln Trp Leu Ile Pro Asp Ser Ala Asp Thr 
    210                 215                 220                 


Thr Val Thr Ser Thr His Cys Ala Tyr Asp Arg Ile Val Val Ala Gly 
225                 230                 235                 240 


Ala Leu Leu Gln Ala Ala Val Val Pro Asn Ser Ala Val Pro Phe Asp 
                245                 250                 255     


Phe Gln Ala Glu Tyr Gly Leu Ser Asn Gln Leu Ala Glu Ala Ile Ser 
            260                 265                 270         


Asp His Tyr Pro Val Glu Val Thr Leu Arg Lys Ile 
        275                 280                 


<210>  4
<211>  554
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic polynucleotide

<400>  4
tacggtaaat ggcccgcctg gctgaccgcc caacgacccc gcccattgac gtcaataatg       60

acgtatgttc ccatagtaac gccaataggg actttccatt gacgtcaatg ggtggagtat      120

ttacggtaaa ctgcccactt ggcagtacat caagtgtatc atatgccaag tacgccccct      180

attgacgtca atgacggtaa atggcccgcc tggcattatg cccagtacat gaccttatgg      240

gactttccta cttggcagta catctactcg aggccacgtt ctgcttcact ctccccatct      300

cccccccctc cccaccccca attttgtatt tatttatttt ttaattattt tgtgcagcga      360

tgggggcggg gggggggggg gggggggcgc gcgccaggcg gggcggggcg gggcgagggg      420

cggggcgggg cgaggcggag aggtgcggcg gcagccaatc agagcggcgc gctccgaaag      480

tttcctttta tggcgaggcg gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg      540

gcgggagcgg gatc                                                        554


<210>  5
<211>  2236
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic polynucleotide

<400>  5
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtgtagcc atgctctagg      120

aagatcaatt cggtacaatt cacgcgtcga cattgattat tgactctggt cgttacataa      180

cttacggtaa atggcccgcc tggctgaccg cccaacgacc ccgcccattg acgtcaataa      240

tgacgtatgt tcccatagta acgccaatag ggactttcca ttgacgtcaa tgggtggagt      300

atttacggta aactgcccac ttggcagtac atcaagtgta tcatatgcca agtacgcccc      360

ctattgacgt caatgacggt aaatggcccg cctggcatta tgcccagtac atgaccttat      420

gggactttcc tacttggcag tacatctact cgaggccacg ttctgcttca ctctccccat      480

ctcccccccc tccccacccc caattttgta tttatttatt ttttaattat tttgtgcagc      540

gatgggggcg gggggggggg gggggggggc gcgcgccagg cggggcgggg cggggcgagg      600

ggcggggcgg ggcgaggcgg agaggtgcgg cggcagccaa tcagagcggc gcgctccgaa      660

agtttccttt tatggcgagg cggcggcggc ggcggcccta taaaaagcga agcgcgcggc      720

gggcgggagc gggatcagcc accgcggtgg cggccctaga gtcgatcgag gaactgaaaa      780

accagaaagt taactggtaa gtttagtctt tttgtctttt atttcaggtc ccggatccgg      840

tggtggtgca aatcaaagaa ctgctcctca gtggatgttg cctttacttc taggcctgta      900

cggaagtgtt acttctgctc taaaagctgc ggaattgtac ccgcggccga tccaccggtc      960

gccaccatgc ggtacacagg gctaatggga acactgctca ccttggtcaa cctgctgcag     1020

ctggctggga ctctgagaat tgcagccttc aacattcgga cttttgggga gactaagatg     1080

tccaatgcta ccctctctgt atactttgtg aaaatcctga gtcgctatga catcgctgtt     1140

atccaagagg tcagagactc ccacctggtt gctgttggga agctcctgga tgaactcaat     1200

cgggacaaac ctgacaccta ccgctatgta gtcagtgagc cgctgggccg caaaagctac     1260

aaggaacagt acctttttgt gtacaggcct gaccaggtgt ctattctgga cagctatcaa     1320

tatgatgatg gctgtgaacc ctgtggaaat gacaccttca gcagagagcc agccattgtt     1380

aagttctttt ccccatacac tgaggtccaa gaatttgcga tcgtgccctt gcatgcagcc     1440

ccaacagaag ctgtgagtga gatcgacgcc ctctacgatg tttacctaga tgtctggcaa     1500

aagtggggcc tggaggacat catgttcatg ggagacttca atgctggctg cagctacgtc     1560

acttcctccc agtggtcctc cattcgcctt cggacaagcc ccatcttcca gtggctgatc     1620

cctgacagtg cggacaccac agtcacatca acacactgtg cttatgacag gattgtggtt     1680

gctggagctc tgctccaggc tgctgttgtt cccaactcgg ctgttccttt tgatttccaa     1740

gcagaatacg gactttccaa ccagctggct gaagccatca gtgaccatta cccagtggag     1800

gtgacactca gaaaaatctg aagcggccat caagcttatc gataccgtcg actagagctc     1860

gctgatcagc ctcgactgtg ccttctagtt gccagccatc tgttgtttgc ccctcccccg     1920

tgccttcctt gaccctggaa ggtgccactc ccactgtcct ttcctaataa aatgaggaaa     1980

ttgcatcgca ttgtctgagt aggtgtcatt ctattctggg gggtggggtg gggcaggaca     2040

gcaaggggga ggattgggaa gacaattagg tagataagta gcatggcggg ttaatcatta     2100

actacaagga acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca     2160

ctgaggccgg gcgaccaaag gtcgcccgac gcccgggctt tgcccgggcg gcctcagtga     2220

gcgagcgagc gcgcag                                                     2236


<210>  6
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic polynucleotide

<400>  6
tcgctatgac atcgctgtta t                                                 21


<210>  7
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic polynucleotide

<400>  7
gtcaggtttg tcccgattga g                                                 21


<210>  8
<211>  22
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic polynucleotide

<400>  8
tggcaaagtg gagattgttg cc                                                22


<210>  9
<211>  21
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic polynucleotide

<400>  9
aagatggtga tgggcttccc g                                                 21


<210>  10
<211>  282
<212>  PRT
<213>  Homo sapiens

<400>  10

Met Arg Gly Met Lys Leu Leu Gly Ala Leu Leu Ala Leu Ala Ala Leu 
1               5                   10                  15      


Leu Gln Gly Ala Val Ser Leu Lys Ile Ala Ala Phe Asn Ile Gln Thr 
            20                  25                  30          


Phe Gly Glu Thr Lys Met Ser Asn Ala Thr Leu Val Ser Tyr Ile Val 
        35                  40                  45              


Gln Ile Leu Ser Arg Tyr Asp Ile Ala Leu Val Gln Glu Val Arg Asp 
    50                  55                  60                  


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


Ala Pro Asp Thr Tyr His Tyr Val Val Ser Glu Pro Leu Gly Arg Asn 
                85                  90                  95      


Ser Tyr Lys Glu Arg Tyr Leu Phe Val Tyr Arg Pro Asp Gln Val Ser 
            100                 105                 110         


Ala Val Asp Ser Tyr Tyr Tyr Asp Asp Gly Cys Glu Pro Cys Gly Asn 
        115                 120                 125             


Asp Thr Phe Asn Arg Glu Pro Ala Ile Val Arg Phe Phe Ser Arg Phe 
    130                 135                 140                 


Thr Glu Val Arg Glu Phe Ala Ile Val Pro Leu His Ala Ala Pro Gly 
145                 150                 155                 160 


Asp Ala Val Ala Glu Ile Asp Ala Leu Tyr Asp Val Tyr Leu Asp Val 
                165                 170                 175     


Gln Glu Lys Trp Gly Leu Glu Asp Val Met Leu Met Gly Asp Phe Asn 
            180                 185                 190         


Ala Gly Cys Ser Tyr Val Arg Pro Ser Gln Trp Ser Ser Ile Arg Leu 
        195                 200                 205             


Trp Thr Ser Pro Thr Phe Gln Trp Leu Ile Pro Asp Ser Ala Asp Thr 
    210                 215                 220                 


Thr Ala Thr Pro Thr His Cys Ala Tyr Asp Arg Ile Val Val Ala Gly 
225                 230                 235                 240 


Met Leu Leu Arg Gly Ala Val Val Pro Asp Ser Ala Leu Pro Phe Asn 
                245                 250                 255     


Phe Gln Ala Ala Tyr Gly Leu Ser Asp Gln Leu Ala Gln Ala Ile Ser 
            260                 265                 270         


Asp His Tyr Pro Val Glu Val Met Leu Lys 
        275                 280         


