                         SEQUENCE LISTING

<110>  Yale University
       Lek, Monkol
       Lek, Angela
       Woodman, Keryn
       Ma, Kaiyue
 
<120>  Compositions and Methods for Upregulating Isoforms of Dystrophin 
       as Therapy for Duchenne Muscular Dystrophy (DMD)

<130>  047162-7245WO1(01236)

<150>  62/913,935
<151>  2019-10-11

<150>  63/064,077
<151>  2020-08-11

<160>  13    

<170>  PatentIn version 3.5

<210>  1
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  1
gagtgagtga tcccaacact ga                                                22


<210>  2
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  2
gtgaaatatc cgggggcctc ta                                                22


<210>  3
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  3
gcacttgctt gtgcgcaggt cc                                                22


<210>  4
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  4
gctttgcatc tgtacagaag a                                                 21


<210>  5
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  5
gtcgactgac gtatcagata gt                                                22


<210>  6
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  6
gatcatgcga aaggggagct gt                                                22


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

<220>
<223>  gRNA

<400>  7
ggcatgccca cctaacctaa c                                                 21


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

<220>
<223>  gRNA

<400>  8
gcacctcact attcacggca ac                                                22


<210>  9
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  9
gaagaaacca ttgctgtgag ag                                                22


<210>  10
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  gRNA

<400>  10
gaactgtgtc gtctgcttta ta                                                22


<210>  11
<211>  7434
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Vector Double VP64 C7 guide

<400>  11
cctggtatct ttatagtcct gtcgggtttc gccacctctg acttgagcgt cgatttttgt       60

gatgctcgtc aggggggcgg agcctatgga aaaacgccag caacgcggcc tttttacggt      120

tcctggcctt ttgctggcct tttgctcaca tgtcctgcag gcagctgcgc gctcgctcgc      180

tcactgaggc cgcccgggcg tcgggcgacc tttggtcgcc cggcctcagt gagcgagcga      240

gcgcgcagag agggagtggc caactccatc actaggggtt cctgcggcct ctagactcga      300

gtgcccatgt aaggaggcaa ggcctgggga cacccgagat gcctggttat aattaaccca      360

gacatgtggc tgcccccccc cccccaacac ctgctgcctc taaaaataac cctgcatgcc      420

atgttcccgg cgaagggcca gctgtccccc gccagctaga ctcagcactt agtttaggaa      480

ccagtgagca agtcagccct tggggcagcc catacaaggc catggggctg ggcaagctgc      540

acgcctgggt ccggggtggg cacggtgccc gggcaacgag ctgaaagctc atctgctctc      600

aggggcccct ccctggggac agcccctcct ggctagtcac accctgtagg ctcctctata      660

taacccaggg gcacaggggc tgccctcatt ctaccaccac ctccacagca cagacagaca      720

ctcaggagcc agccagcacc ggtgccacca tgggtccgcg ggctgacgca ttggacgatt      780

ttgatctgga tatgctggga agtgacgccc tcgatgattt tgaccttgac atgcttggtt      840

cggatgccct tgatgacttt gacctcgaca tgctcggcag tgacgccctt gatgatttcg      900

acctggacat ggttaaccca aagaagaagc ggaaggtcgg tatccacgga gtcccagcag      960

ccaagcggaa ctacatcctg ggcctggcca tcggcatcac cagcgtgggc tacggcatca     1020

tcgactacga gacacgggac gtgatcgatg ccggcgtgcg gctgttcaaa gaggccaacg     1080

tggaaaacaa cgagggcagg cggagcaaga gaggcgccag aaggctgaag cggcggaggc     1140

ggcatagaat ccagagagtg aagaagctgc tgttcgacta caacctgctg accgaccaca     1200

gcgagctgag cggcatcaac ccctacgagg ccagagtgaa gggcctgagc cagaagctga     1260

gcgaggaaga gttctctgcc gccctgctgc acctggccaa gagaagaggc gtgcacaacg     1320

tgaacgaggt ggaagaggac accggcaacg agctgtccac caaagagcag atcagccgga     1380

acagcaaggc cctggaagag aaatacgtgg ccgaactgca gctggaacgg ctgaagaaag     1440

acggcgaagt gcggggcagc atcaacagat tcaagaccag cgactacgtg aaagaagcca     1500

aacagctgct gaaggtgcag aaggcctacc accagctgga ccagagcttc atcgacacct     1560

acatcgacct gctggaaacc cggcggacct actatgaggg acctggcgag ggcagcccct     1620

tcggctggaa ggacatcaaa gaatggtacg agatgctgat gggccactgc acctacttcc     1680

ccgaggaact gcggagcgtg aagtacgcct acaacgccga cctgtacaac gccctgaacg     1740

acctgaacaa tctcgtgatc accagggacg agaacgagaa gctggaatat tacgagaagt     1800

tccagatcat cgagaacgtg ttcaagcaga agaagaagcc caccctgaag cagatcgcca     1860

aagaaatcct cgtgaacgaa gaggatatta agggctacag agtgaccagc accggcaagc     1920

ccgagttcac caacctgaag gtgtaccacg acatcaagga cattaccgcc cggaaagaga     1980

ttattgagaa cgccgagctg ctggatcaga ttgccaagat cctgaccatc taccagagca     2040

gcgaggacat ccaggaagaa ctgaccaatc tgaactccga gctgacccag gaagagatcg     2100

agcagatctc taatctgaag ggctataccg gcacccacaa cctgagcctg aaggccatca     2160

acctgatcct ggacgagctg tggcacacca acgacaacca gatcgctatc ttcaaccggc     2220

tgaagctggt gcccaagaag gtggacctgt cccagcagaa agagatcccc accaccctgg     2280

tggacgactt catcctgagc cccgtcgtga agagaagctt catccagagc atcaaagtga     2340

tcaacgccat catcaagaag tacggcctgc ccaacgacat cattatcgag ctggcccgcg     2400

agaagaactc caaggacgcc cagaaaatga tcaacgagat gcagaagcgg aaccggcaga     2460

ccaacgagcg gatcgaggaa atcatccgga ccaccggcaa agagaacgcc aagtacctga     2520

tcgagaagat caagctgcac gacatgcagg aaggcaagtg cctgtacagc ctggaagcca     2580

tccctctgga agatctgctg aacaacccct tcaactatga ggtggaccac atcatcccca     2640

gaagcgtgtc cttcgacaac agcttcaaca acaaggtgct cgtgaagcag gaagaagcca     2700

gcaagaaggg caaccggacc ccattccagt acctgagcag cagcgacagc aagatcagct     2760

acgaaacctt caagaagcac atcctgaatc tggccaaggg caagggcaga atcagcaaga     2820

ccaagaaaga gtatctgctg gaagaacggg acatcaacag gttctccgtg cagaaagact     2880

tcatcaaccg gaacctggtg gataccagat acgccaccag aggcctgatg aacctgctgc     2940

ggagctactt cagagtgaac aacctggacg tgaaagtgaa gtccatcaat ggcggcttca     3000

ccagctttct gcggcggaag tggaagttta agaaagagcg gaacaagggg tacaagcacc     3060

acgccgagga cgccctgatc attgccaacg ccgatttcat cttcaaagag tggaagaaac     3120

tggacaaggc caaaaaagtg atggaaaacc agatgttcga ggaaaagcag gccgagagca     3180

tgcccgagat cgaaaccgag caggagtaca aagagatctt catcaccccc caccagatca     3240

agcacattaa ggacttcaag gactacaagt acagccaccg ggtggacaag aagcctaata     3300

gagagctgat taacgacacc ctgtactcca cccggaagga cgacaagggc aacaccctga     3360

tcgtgaacaa tctgaacggc ctgtacgaca aggacaatga caagctgaaa aagctgatca     3420

acaagagccc cgaaaagctg ctgatgtacc accacgaccc ccagacctac cagaaactga     3480

agctgattat ggaacagtac ggcgacgaga agaatcccct gtacaagtac tacgaggaaa     3540

ccgggaacta cctgaccaag tactccaaaa aggacaacgg ccccgtgatc aagaagatta     3600

agtattacgg caacaaactg aacgcccatc tggacatcac cgacgactac cccaacagca     3660

gaaacaaggt cgtgaagctg tccctgaagc cctacagatt cgacgtgtac ctggacaatg     3720

gcgtgtacaa gttcgtgacc gtgaagaatc tggatgtgat caaaaaagaa aactactacg     3780

aagtgaatag caagtgctat gaggaagcta agaagctgaa gaagatcagc aaccaggccg     3840

agtttatcgc ctccttctac aacaacgatc tgatcaagat caacggcgag ctgtatagag     3900

tgatcggcgt gaacaacgac ctgctgaacc ggatcgaagt gaacatgatc gacatcacct     3960

accgcgagta cctggaaaac atgaacgaca agaggccccc caggatcatt aagacaatcg     4020

cctccaagac ccagagcatt aagaagtaca gcacagacat tctgggcaac ctgtatgaag     4080

tgaaatctaa gaagcaccct cagatcatca aaaagggcaa aaggccggcg gccacgaaaa     4140

aggccggcca ggcaaaaaag aaaaagggat ccgacgcgct ggacgatttc gatctcgaca     4200

tgctgggttc tgatgccctc gatgactttg acctggatat gttgggaagc gacgcattgg     4260

atgactttga tctggacatg ctcggctccg atgctctgga cgatttcgat ctcgatatgt     4320

tataagaatt catcgatacc gtcgagatct aacttgttta ttgcagctta taatggttac     4380

aaataaagca atagcatcac aaatttcaca aataaagcat ttttttcact gcattctagt     4440

tgtggtttgt ccaaactcat caatgtatct tatcatgtct ggaggtaccg agggcctatt     4500

tcccatgatt ccttcatatt tgcatatacg atacaaggct gttagagaga taattggaat     4560

taatttgact gtaaacacaa agatattagt acaaaatacg tgacgtagaa agtaataatt     4620

tcttgggtag tttgcagttt taaaattatg ttttaaaatg gactatcata tgcttaccgt     4680

aacttgaaag tatttcgatt tcttggcttt atatatcttg tggaaaggac gaaacaccga     4740

tcatgcgaaa ggggagctgt gttttagtac tctggaaaca gaatctacta aaacaaggca     4800

aaatgccgtg tttatctcgt caacttgttg gcgagatttt tgcggccgca ggaaccccta     4860

gtgatggagt tggccactcc ctctctgcgc gctcgctcgc tcactgaggc cgggcgacca     4920

aaggtcgccc gacgcccggg ctttgcccgg gcggcctcag tgagcgagcg agcgcgcagc     4980

tgcctgcagg ggcgcctgat gcggtatttt ctccttacgc atctgtgcgg tatttcacac     5040

cgcatacgtc aaagcaacca tagtacgcgc cctgtagcgg cgcattaagc gcggcgggtg     5100

tggtggttac gcgcagcgtg accgctacac ttgccagcgc cttagcgccc gctcctttcg     5160

ctttcttccc ttcctttctc gccacgttcg ccggctttcc ccgtcaagct ctaaatcggg     5220

ggctcccttt agggttccga tttagtgctt tacggcacct cgaccccaaa aaacttgatt     5280

tgggtgatgg ttcacgtagt gggccatcgc cctgatagac ggtttttcgc cctttgacgt     5340

tggagtccac gttctttaat agtggactct tgttccaaac tggaacaaca ctcaactcta     5400

tctcgggcta ttcttttgat ttataaggga ttttgccgat ttcggtctat tggttaaaaa     5460

atgagctgat ttaacaaaaa tttaacgcga attttaacaa aatattaacg tttacaattt     5520

tatggtgcac tctcagtaca atctgctctg atgccgcata gttaagccag ccccgacacc     5580

cgccaacacc cgctgacgcg ccctgacggg cttgtctgct cccggcatcc gcttacagac     5640

aagctgtgac cgtctccggg agctgcatgt gtcagaggtt ttcaccgtca tcaccgaaac     5700

gcgcgagacg aaagggcctc gtgatacgcc tatttttata ggttaatgtc atgataataa     5760

tggtttctta gacgtcaggt ggcacttttc ggggaaatgt gcgcggaacc cctatttgtt     5820

tatttttcta aatacattca aatatgtatc cgctcatgag acaataaccc tgataaatgc     5880

ttcaataata ttgaaaaagg aagagtatga gtattcaaca tttccgtgtc gcccttattc     5940

ccttttttgc ggcattttgc cttcctgttt ttgctcaccc agaaacgctg gtgaaagtaa     6000

aagatgctga agatcagttg ggtgcacgag tgggttacat cgaactggat ctcaacagcg     6060

gtaagatcct tgagagtttt cgccccgaag aacgttttcc aatgatgagc acttttaaag     6120

ttctgctatg tggcgcggta ttatcccgta ttgacgccgg gcaagagcaa ctcggtcgcc     6180

gcatacacta ttctcagaat gacttggttg agtactcacc agtcacagaa aagcatctta     6240

cggatggcat gacagtaaga gaattatgca gtgctgccat aaccatgagt gataacactg     6300

cggccaactt acttctgaca acgatcggag gaccgaagga gctaaccgct tttttgcaca     6360

acatggggga tcatgtaact cgccttgatc gttgggaacc ggagctgaat gaagccatac     6420

caaacgacga gcgtgacacc acgatgcctg tagcaatggc aacaacgttg cgcaaactat     6480

taactggcga actacttact ctagcttccc ggcaacaatt aatagactgg atggaggcgg     6540

ataaagttgc aggaccactt ctgcgctcgg cccttccggc tggctggttt attgctgata     6600

aatctggagc cggtgagcgt ggaagccgcg gtatcattgc agcactgggg ccagatggta     6660

agccctcccg tatcgtagtt atctacacga cggggagtca ggcaactatg gatgaacgaa     6720

atagacagat cgctgagata ggtgcctcac tgattaagca ttggtaactg tcagaccaag     6780

tttactcata tatactttag attgatttaa aacttcattt ttaatttaaa aggatctagg     6840

tgaagatcct ttttgataat ctcatgacca aaatccctta acgtgagttt tcgttccact     6900

gagcgtcaga ccccgtagaa aagatcaaag gatcttcttg agatcctttt tttctgcgcg     6960

taatctgctg cttgcaaaca aaaaaaccac cgctaccagc ggtggtttgt ttgccggatc     7020

aagagctacc aactcttttt ccgaaggtaa ctggcttcag cagagcgcag ataccaaata     7080

ctgttcttct agtgtagccg tagttaggcc accacttcaa gaactctgta gcaccgccta     7140

catacctcgc tctgctaatc ctgttaccag tggctgctgc cagtggcgat aagtcgtgtc     7200

ttaccgggtt ggactcaaga cgatagttac cggataaggc gcagcggtcg ggctgaacgg     7260

ggggttcgtg cacacagccc agcttggagc gaacgaccta caccgaactg agatacctac     7320

agcgtgagct atgagaaagc gccacgcttc ccgaagggag aaaggcggac aggtatccgg     7380

taagcggcag ggtcggaaca ggagagcgca cgagggagct tccaggggga aacg           7434


<210>  12
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Dp427c Nterm

<400>  12

Met Leu Trp Trp Glu Glu Val Glu Asp Cys Tyr 
1               5                   10      


<210>  13
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Dp427p Nterm

<400>  13

Met Ser Glu Val Ser Ser Asp 
1               5           


