                         SEQUENCE LISTING

<110>  Wake Forest University Health Sciences
       Lu, Baisong
       Atala, Anthony
 
<120>  Gene Therapy for Dent Disease

<130>  205286-7010WO1(00087)

<150>  US 63/193,212
<151>  2021-05-26

<160>  20    

<170>  PatentIn version 3.5

<210>  1
<211>  11359
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hCLCN5 vector

<400>  1
gacggatcgg gagatctccc gatcccctat ggtcgactct cagtacaatc tgctctgatg       60

ccgcatagtt aagccagtat ctgctccctg cttgtgtgtt ggaggtcgct gagtagtgcg      120

cgagcaaaat ttaagctaca acaaggcaag gcttgaccga caattgcatg aagaatctgc      180

ttagggttag gcgttttgcg ctgcttcgcg atgtacgggc cagatatacg cgttgacatt      240

gattattgac tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata      300

tggagttccg cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc      360

cccgcccatt gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc      420

attgacgtca atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt      480

atcatatgcc aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt      540

atgcccagta catgacctta tgggactttc ctacttggca gtacatctac gtattagtca      600

tcgctattac catggtgatg cggttttggc agtacatcaa tgggcgtgga tagcggtttg      660

actcacgggg atttccaagt ctccacccca ttgacgtcaa tgggagtttg ttttggcacc      720

aaaatcaacg ggactttcca aaatgtcgta acaactccgc cccattgacg caaatgggcg      780

gtaggcgtgt acggtgggag gtctatataa gcagcgcgtt ttgcctgtac tgggtctctc      840

tggttagacc agatctgagc ctgggagctc tctggctaac tagggaaccc actgcttaag      900

cctcaataaa gcttgccttg agtgcttcaa gtagtgtgtg cccgtctgtt gtgtgactct      960

ggtaactaga gatccctcag acccttttag tcagtgtgga aaatctctag cagtggcgcc     1020

cgaacaggga cttgaaagcg aaagggaaac cagaggagct ctctcgacgc aggactcggc     1080

ttgctgaagc gcgcacggca agaggcgagg ggcggcgact ggtgagtacg ccaaaaattt     1140

tgactagcgg aggctagaag gagagagatg ggtgcgagag cgtcagtatt aagcggggga     1200

gaattagatc gcgatgggaa aaaattcggt taaggccagg gggaaagaaa aaatataaat     1260

taaaacatat agtatgggca agcagggagc tagaacgatt cgcagttaat cctggcctgt     1320

tagaaacatc agaaggctgt agacaaatac tgggacagct acaaccatcc cttcagacag     1380

gatcagaaga acttagatca ttatataata cagtagcaac cctctattgt gtgcatcaaa     1440

ggatagagat aaaagacacc aaggaagctt tagacaagat agaggaagag caaaacaaaa     1500

gtaagaccac cgcacagcaa gcggccggcc gctgatcttc agacctggag gaggagatat     1560

gagggacaat tggagaagtg aattatataa atataaagta gtaaaaattg aaccattagg     1620

agtagcaccc accaaggcaa agagaagagt ggtgcagaga gaaaaaagag cagtgggaat     1680

aggagctttg ttccttgggt tcttgggagc agcaggaagc actatgggcg cagcgtcaat     1740

gacgctgacg gtacaggcca gacaattatt gtctggtata gtgcagcagc agaacaattt     1800

gctgagggct attgaggcgc aacagcatct gttgcaactc acagtctggg gcatcaagca     1860

gctccaggca agaatcctgg ctgtggaaag atacctaaag gatcaacagc tcctggggat     1920

ttggggttgc tctggaaaac tcatttgcac cactgctgtg ccttggaatg ctagttggag     1980

taataaatct ctggaacaga tttggaatca cacgacctgg atggagtggg acagagaaat     2040

taacaattac acaagcttaa tacactcctt aattgaagaa tcgcaaaacc agcaagaaaa     2100

gaatgaacaa gaattattgg aattagataa atgggcaagt ttgtggaatt ggtttaacat     2160

aacaaattgg ctgtggtata taaaattatt cataatgata gtaggaggct tggtaggttt     2220

aagaatagtt tttgctgtac tttctatagt gaatagagtt aggcagggat attcaccatt     2280

atcgtttcag acccacctcc caaccccgag gggacccgac aggcccgaag gaatagaaga     2340

agaaggtgga gagagagaca gagacagatc cattcgatta gtgaacggat ctacaaatgg     2400

cagtattcat ccacaatttt aaaagaaaag gggggattgg ggggtacagt gcaggggaaa     2460

gaatagtaga cataatagca acagacatac aaactaaaga attacaaaaa caaattacaa     2520

aaattcaaaa ttttcgggtt tattacaggg acagcagaaa ttcactttga attaattcaa     2580

gcttcgtgag gctccggtgc ccgtcagtgg gcagagcgca catcgcccac agtccccgag     2640

aagttggggg gaggggtcgg caattgaacc ggtgcctaga gaaggtggcg cggggtaaac     2700

tgggaaagtg atgtcgtgta ctggctccgc ctttttcccg agggtggggg agaaccgtat     2760

ataagtgcag tagtcgccgt gaacgttctt tttcgcaacg ggtttgccgc cagaacacag     2820

gtaagtgccg tgtgtggttc ccgcgggcct ggcctcttta cgggttatgg cccttgcgtg     2880

ccttgaatta cttccacctg gctccagtac gtgattcttg atcccgagct ggagccaggg     2940

gcgggccttg cgctttagga gccccttcgc ctcgtgcttg agttgaggcc tggcctgggc     3000

gctggggccg ccgcgtgcga atctggtggc accttcgcgc ctgtctcgct gctttcgata     3060

agtctctagc catttaaaat ttttgatgac ctgctgcgac gctttttttc tggcaagata     3120

gtcttgtaaa tgcgggccag gatctgcaca ctggtatttc ggtttttggg gccgcgggcg     3180

gcgacggggc ccgtgcgtcc cagcgcacat gttcggcgag gcggggcctg cgagcgcggc     3240

caccgagaat cggacggggg tagtctcaag ctggccggcc tgctctggtg cctggcctcg     3300

cgccgccgtg tatcgccccg ccctgggcgg caaggctggc ccggtcggca ccagttgcgt     3360

gagcggaaag atggccgctt cccggccctg ctccaggggg ctcaaaatgg aggacgcggc     3420

gctcgggaga gcgggcgggt gagtcaccca cacaaaggaa aggggccttt ccgtcctcag     3480

ccgtcgcttc atgtgactcc acggagtacc gggcgccgtc caggcacctc gattagttct     3540

ggagcttttg gagtacgtcg tctttaggtt ggggggaggg gttttatgcg atggagtttc     3600

cccacactga gtgggtggag actgaagtta ggccagcttg gcacttgatg taattctcct     3660

tggaatttgc cctttttgag tttggatctt ggttcattct caagcctcag acagtggttc     3720

aaagtttttt tcttccattt caggtgtcgt gaacacgcta ccggtctcga gccaccatgg     3780

atttcctgga ggaaccaata ccaggtgtag gaacatatga cgatttcaat actatagact     3840

gggtgcgaga gaaatcacgc gatcgagaca gacaccggga gatcacgaat aagtctaagg     3900

aatctacctg ggccctcatt cacagtgtgt cagacgcttt tagcggatgg ctgcttatgc     3960

ttctgattgg acttcttagt ggtagtttgg cgggcctgat agacattagc gcgcactgga     4020

tgactgatct taaagaaggc atatgcacgg ggggattttg gttcaaccac gaacattgct     4080

gctggaactc cgagcatgtg acattcgagg agagggacaa gtgccccgag tggaatagtt     4140

ggagccaact gataatttct acagatgagg gggcttttgc ctatatagtt aattatttca     4200

tgtatgtttt gtgggccctc ctcttcgcct tcctcgcggt atccctcgtt aaggtctttg     4260

ccccatatgc ctgtggctct ggtattccag aaataaaaac tatcctttct ggatttataa     4320

tcaggggata tctgggcaag tggacgttgg tcattaagac aatcaccctt gtccttgctg     4380

tatcttcagg gttgtccttg ggcaaagagg gtcctctcgt tcacgtagct tgctgctgtg     4440

ggaacatcct ttgccattgt ttcaataaat ataggaagaa cgaagcaaag cgccgagaag     4500

ttctgagcgc agcagcggcc gcaggtgtca gtgttgcctt cggggctcct ataggagggg     4560

tactgtttag tctcgaagaa gtgtcatatt actttcctct caagacactg tggaggtcct     4620

tttttgcagc cctggtcgcg gcttttactc tgcgctctat taatcctttt ggaaacagca     4680

gacttgtgct gttctacgtc gaattccaca ccccgtggca tttgtttgaa ctcgtaccct     4740

ttattttgct ggggattttc ggtggattgt ggggtgctct gttcatacgc actaacattg     4800

cgtggtgccg gaagaggaag actactcagt tgggcaaata cccagttatt gaggtcctcg     4860

tcgttacagc tatcacagca attcttgcgt tccccaacga gtacacacgg atgtctacat     4920

ccgaactgat tagcgaactg ttcaatgatt gtgggctctt ggactcctca aaactgtgcg     4980

attatgaaaa tcgatttaat acatcaaagg gcggagaact tcccgatcgg ccggctggag     5040

tgggagtata ctccgctatg tggcagctgg cgttgacgct catactcaaa atcgtcatta     5100

ccatattcac ttttggaatg aagattccct caggtctctt tatccctagt atggcagttg     5160

gtgcgattgc gggacggctc ctgggcgttg gcatggagca gctggcttat taccatcagg     5220

agtggaccgt attcaatagc tggtgctctc agggcgctga ttgcatcaca ccaggcctgt     5280

atgccatggt aggcgctgct gcttgtcttg gaggggtgac taggatgacg gtttctctcg     5340

tcgtgataat gttcgagctt actgggggtc ttgagtacat tgtgcccctg atggcggcgg     5400

caatgacatc caaatgggtg gcggatgcgt tgggtaggga agggatatac gatgcacata     5460

ttcgccttaa tggctaccca tttttggagg ctaaggaaga atttgcacat aaaactctcg     5520

ccatggatgt tatgaaaccg agacgaaacg acccattgct tacagtactt acacaggatt     5580

ccatgaccgt tgaggacgtg gaaacaataa tatctgaaac aacttatagt ggctttcccg     5640

tcgtcgtatc ccgagaatca caaaggttgg taggattcgt gctgcgacgc gacctgatca     5700

tatccataga aaacgcacgc aagaagcaag acggggtagt gtccacgtct ataatttatt     5760

tcaccgagca tagccctccc ttgcctccat atactccgcc tacactgaaa cttcgaaaca     5820

tcctcgattt gtctcctttt acagtaaccg accttactcc aatggaaatc gtagtagaca     5880

tatttagaaa gcttggattg aggcaatgcc tggttaccca caacggtcgg ttgctcggga     5940

taataacgaa gaaggacgta ctcaaacata tagcacaaat ggcaaaccag gacccggatt     6000

caatcttgtt caactagtct agagttaaca tcgagggatc aagcttatcg ataatcaacc     6060

tctggattac aaaatttgtg aaagattgac tggtattctt aactatgttg ctccttttac     6120

gctatgtgga tacgctgctt taatgccttt gtatcatgct attgcttccc gtatggcttt     6180

cattttctcc tccttgtata aatcctggtt gctgtctctt tatgaggagt tgtggcccgt     6240

tgtcaggcaa cgtggcgtgg tgtgcactgt gtttgctgac gcaaccccca ctggttgggg     6300

cattgccacc acctgtcagc tcctttccgg gactttcgct ttccccctcc ctattgccac     6360

ggcggaactc atcgccgcct gccttgcccg ctgctggaca ggggctcggc tgttgggcac     6420

tgacaattcc gtggtgttgt cggggaagct gacgtccttt ccatggctgc tcgcctgtgt     6480

tgccacctgg attctgcgcg ggacgtcctt ctgctacgtc ccttcggccc tcaatccagc     6540

ggaccttcct tcccgcggcc tgctgccggc tctgcggcct cttccgcgtc ttcgccttcg     6600

ccctcagacg agtcggatct ccctttgggc cgcctccccg catcgatacc gtcgagacct     6660

ggaaaaacat ggagcaatca caagtagcaa cacagcagct accaatgctg cttgtgcctg     6720

gctagaagca caagaggagg aggaggtggg ttttccagtc acacctcagg tacctttaag     6780

accaatgact tacaaggcag ctgtagatct tagccacttt ttaaaagaaa aggggggact     6840

ggaagggcta attcactccc aacgaagaca agatatcctt gatctgtgga tctaccacac     6900

acaaggctac ttccctgatt ggcagaacta cacaccaggg ccagggatca gatatccact     6960

gacctttgga tggtgctaca agctagtacc agttgagcaa gagaaggtag aagaagccaa     7020

tgaaggagag aacacccgct tgttacaccc tgtgagcctg catgggatgg atgacccgga     7080

gagagaagta ttagagtgga ggtttgacag ccgcctagca tttcatcaca tggcccgaga     7140

gctgcatccg gactgtactg ggtctctctg gttagaccag atctgagcct gggagctctc     7200

tggctaacta gggaacccac tgcttaagcc tcaataaagc ttgccttgag tgcttcaagt     7260

agtgtgtgcc cgtctgttgt gtgactctgg taactagaga tccctcagac ccttttagtc     7320

agtgtggaaa atctctagca gggcccgttt aaacccgctg atcagcctcg actgtgcctt     7380

ctagttgcca gccatctgtt gtttgcccct cccccgtgcc ttccttgacc ctggaaggtg     7440

ccactcccac tgtcctttcc taataaaatg aggaaattgc atcgcattgt ctgagtaggt     7500

gtcattctat tctggggggt ggggtggggc aggacagcaa gggggaggat tgggaagaca     7560

atagcaggca tgctggggat gcggtgggct ctatggcttc tgaggcggaa agaaccagct     7620

ggggctctag ggggtatccc cacgcgccct gtagcggcgc attaagcgcg gcgggtgtgg     7680

tggttacgcg cagcgtgacc gctacacttg ccagcgccct agcgcccgct cctttcgctt     7740

tcttcccttc ctttctcgcc acgttcgccg gctttccccg tcaagctcta aatcggggca     7800

tccctttagg gttccgattt agtgctttac ggcacctcga ccccaaaaaa cttgattagg     7860

gtgatggttc acgtagtggg ccatcgccct gatagacggt ttttcgccct ttgacgttgg     7920

agtccacgtt ctttaatagt ggactcttgt tccaaactgg aacaacactc aaccctatct     7980

cggtctattc ttttgattta taagggattt tggggatttc ggcctattgg ttaaaaaatg     8040

agctgattta acaaaaattt aacgcgaatt aattctgtgg aatgtgtgtc agttagggtg     8100

tggaaagtcc ccaggctccc caggcaggca gaagtatgca aagcatgcat ctcaattagt     8160

cagcaaccag gtgtggaaag tccccaggct ccccagcagg cagaagtatg caaagcatgc     8220

atctcaatta gtcagcaacc atagtcccgc ccctaactcc gcccatcccg cccctaactc     8280

cgcccagttc cgcccattct ccgccccatg gctgactaat tttttttatt tatgcagagg     8340

ccgaggccgc ctctgcctct gagctattcc agaagtagtg aggaggcttt tttggaggcc     8400

taggcttttg caaaaagctc ccgggagctt gtatatccat tttcggatct gatcagcacg     8460

tgttgacaat taatcatcgg catagtatat cggcatagta taatacgaca aggtgaggaa     8520

ctaaaccatg gccaagttga ccagtgccgt tccggtgctc accgcgcgcg acgtcgccgg     8580

agcggtcgag ttctggaccg accggctcgg gttctcccgg gacttcgtgg aggacgactt     8640

cgccggtgtg gtccgggacg acgtgaccct gttcatcagc gcggtccagg accaggtggt     8700

gccggacaac accctggcct gggtgtgggt gcgcggcctg gacgagctgt acgccgagtg     8760

gtcggaggtc gtgtccacga acttccggga cgcctccggg ccggccatga ccgagatcgg     8820

cgagcagccg tgggggcggg agttcgccct gcgcgacccg gccggcaact gcgtgcactt     8880

cgtggccgag gagcaggact gacacgtgct acgagatttc gattccaccg ccgccttcta     8940

tgaaaggttg ggcttcggaa tcgttttccg ggacgccggc tggatgatcc tccagcgcgg     9000

ggatctcatg ctggagttct tcgcccaccc caacttgttt attgcagctt ataatggtta     9060

caaataaagc aatagcatca caaatttcac aaataaagca tttttttcac tgcattctag     9120

ttgtggtttg tccaaactca tcaatgtatc ttatcatgtc tgtataccgt cgacctctag     9180

ctagagcttg gcgtaatcat ggtcatagct gtttcctgtg tgaaattgtt atccgctcac     9240

aattccacac aacatacgag ccggaagcat aaagtgtaaa gcctggggtg cctaatgagt     9300

gagctaactc acattaattg cgttgcgctc actgcccgct ttccagtcgg gaaacctgtc     9360

gtgccagctg cattaatgaa tcggccaacg cgcggggaga ggcggtttgc gtattgggcg     9420

ctcttccgct tcctcgctca ctgactcgct gcgctcggtc gttcggctgc ggcgagcggt     9480

atcagctcac tcaaaggcgg taatacggtt atccacagaa tcaggggata acgcaggaaa     9540

gaacatgtga gcaaaaggcc agcaaaaggc caggaaccgt aaaaaggccg cgttgctggc     9600

gtttttccat aggctccgcc cccctgacga gcatcacaaa aatcgacgct caagtcagag     9660

gtggcgaaac ccgacaggac tataaagata ccaggcgttt ccccctggaa gctccctcgt     9720

gcgctctcct gttccgaccc tgccgcttac cggatacctg tccgcctttc tcccttcggg     9780

aagcgtggcg ctttctcaat gctcacgctg taggtatctc agttcggtgt aggtcgttcg     9840

ctccaagctg ggctgtgtgc acgaaccccc cgttcagccc gaccgctgcg ccttatccgg     9900

taactatcgt cttgagtcca acccggtaag acacgactta tcgccactgg cagcagccac     9960

tggtaacagg attagcagag cgaggtatgt aggcggtgct acagagttct tgaagtggtg    10020

gcctaactac ggctacacta gaaggacagt atttggtatc tgcgctctgc tgaagccagt    10080

taccttcgga aaaagagttg gtagctcttg atccggcaaa caaaccaccg ctggtagcgg    10140

tggttttttt gtttgcaagc agcagattac gcgcagaaaa aaaggatctc aagaagatcc    10200

tttgatcttt tctacggggt ctgacgctca gtggaacgaa aactcacgtt aagggatttt    10260

ggtcatgaga ttatcaaaaa ggatcttcac ctagatcctt ttaaattaaa aatgaagttt    10320

taaatcaatc taaagtatat atgagtaaac ttggtctgac agttaccaat gcttaatcag    10380

tgaggcacct atctcagcga tctgtctatt tcgttcatcc atagttgcct gactccccgt    10440

cgtgtagata actacgatac gggagggctt accatctggc cccagtgctg caatgatacc    10500

gcgagaccca cgctcaccgg ctccagattt atcagcaata aaccagccag ccggaagggc    10560

cgagcgcaga agtggtcctg caactttatc cgcctccatc cagtctatta attgttgccg    10620

ggaagctaga gtaagtagtt cgccagttaa tagtttgcgc aacgttgttg ccattgctac    10680

aggcatcgtg gtgtcacgct cgtcgtttgg tatggcttca ttcagctccg gttcccaacg    10740

atcaaggcga gttacatgat cccccatgtt gtgcaaaaaa gcggttagct ccttcggtcc    10800

tccgatcgtt gtcagaagta agttggccgc agtgttatca ctcatggtta tggcagcact    10860

gcataattct cttactgtca tgccatccgt aagatgcttt tctgtgactg gtgagtactc    10920

aaccaagtca ttctgagaat agtgtatgcg gcgaccgagt tgctcttgcc cggcgtcaat    10980

acgggataat accgcgccac atagcagaac tttaaaagtg ctcatcattg gaaaacgttc    11040

ttcggggcga aaactctcaa ggatcttacc gctgttgaga tccagttcga tgtaacccac    11100

tcgtgcaccc aactgatctt cagcatcttt tactttcacc agcgtttctg ggtgagcaaa    11160

aacaggaagg caaaatgccg caaaaaaggg aataagggcg acacggaaat gttgaatact    11220

catactcttc ctttttcaat attattgaag catttatcag ggttattgtc tcatgagcgg    11280

atacatattt gaatgtattt agaaaaataa acaaataggg gttccgcgca catttccccg    11340

aaaagtgcca cctgacgtc                                                 11359


<210>  2
<211>  2241
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hCLCN5 cDNA

<400>  2
atggatttcc tggaggaacc aataccaggt gtaggaacat atgacgattt caatactata       60

gactgggtgc gagagaaatc acgcgatcga gacagacacc gggagatcac gaataagtct      120

aaggaatcta cctgggccct cattcacagt gtgtcagacg cttttagcgg atggctgctt      180

atgcttctga ttggacttct tagtggtagt ttggcgggcc tgatagacat tagcgcgcac      240

tggatgactg atcttaaaga aggcatatgc acggggggat tttggttcaa ccacgaacat      300

tgctgctgga actccgagca tgtgacattc gaggagaggg acaagtgccc cgagtggaat      360

agttggagcc aactgataat ttctacagat gagggggctt ttgcctatat agttaattat      420

ttcatgtatg ttttgtgggc cctcctcttc gccttcctcg cggtatccct cgttaaggtc      480

tttgccccat atgcctgtgg ctctggtatt ccagaaataa aaactatcct ttctggattt      540

ataatcaggg gatatctggg caagtggacg ttggtcatta agacaatcac ccttgtcctt      600

gctgtatctt cagggttgtc cttgggcaaa gagggtcctc tcgttcacgt agcttgctgc      660

tgtgggaaca tcctttgcca ttgtttcaat aaatatagga agaacgaagc aaagcgccga      720

gaagttctga gcgcagcagc ggccgcaggt gtcagtgttg ccttcggggc tcctatagga      780

ggggtactgt ttagtctcga agaagtgtca tattactttc ctctcaagac actgtggagg      840

tccttttttg cagccctggt cgcggctttt actctgcgct ctattaatcc ttttggaaac      900

agcagacttg tgctgttcta cgtcgaattc cacaccccgt ggcatttgtt tgaactcgta      960

ccctttattt tgctggggat tttcggtgga ttgtggggtg ctctgttcat acgcactaac     1020

attgcgtggt gccggaagag gaagactact cagttgggca aatacccagt tattgaggtc     1080

ctcgtcgtta cagctatcac agcaattctt gcgttcccca acgagtacac acggatgtct     1140

acatccgaac tgattagcga actgttcaat gattgtgggc tcttggactc ctcaaaactg     1200

tgcgattatg aaaatcgatt taatacatca aagggcggag aacttcccga tcggccggct     1260

ggagtgggag tatactccgc tatgtggcag ctggcgttga cgctcatact caaaatcgtc     1320

attaccatat tcacttttgg aatgaagatt ccctcaggtc tctttatccc tagtatggca     1380

gttggtgcga ttgcgggacg gctcctgggc gttggcatgg agcagctggc ttattaccat     1440

caggagtgga ccgtattcaa tagctggtgc tctcagggcg ctgattgcat cacaccaggc     1500

ctgtatgcca tggtaggcgc tgctgcttgt cttggagggg tgactaggat gacggtttct     1560

ctcgtcgtga taatgttcga gcttactggg ggtcttgagt acattgtgcc cctgatggcg     1620

gcggcaatga catccaaatg ggtggcggat gcgttgggta gggaagggat atacgatgca     1680

catattcgcc ttaatggcta cccatttttg gaggctaagg aagaatttgc acataaaact     1740

ctcgccatgg atgttatgaa accgagacga aacgacccat tgcttacagt acttacacag     1800

gattccatga ccgttgagga cgtggaaaca ataatatctg aaacaactta tagtggcttt     1860

cccgtcgtcg tatcccgaga atcacaaagg ttggtaggat tcgtgctgcg acgcgacctg     1920

atcatatcca tagaaaacgc acgcaagaag caagacgggg tagtgtccac gtctataatt     1980

tatttcaccg agcatagccc tcccttgcct ccatatactc cgcctacact gaaacttcga     2040

aacatcctcg atttgtctcc ttttacagta accgacctta ctccaatgga aatcgtagta     2100

gacatattta gaaagcttgg attgaggcaa tgcctggtta cccacaacgg tcggttgctc     2160

gggataataa cgaagaagga cgtactcaaa catatagcac aaatggcaaa ccaggacccg     2220

gattcaatct tgttcaacta g                                               2241


<210>  3
<211>  746
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  hCLCN5 protein

<400>  3

Met Asp Phe Leu Glu Glu Pro Ile Pro Gly Val Gly Thr Tyr Asp Asp 
1               5                   10                  15      


Phe Asn Thr Ile Asp Trp Val Arg Glu Lys Ser Arg Asp Arg Asp Arg 
            20                  25                  30          


His Arg Glu Ile Thr Asn Lys Ser Lys Glu Ser Thr Trp Ala Leu Ile 
        35                  40                  45              


His Ser Val Ser Asp Ala Phe Ser Gly Trp Leu Leu Met Leu Leu Ile 
    50                  55                  60                  


Gly Leu Leu Ser Gly Ser Leu Ala Gly Leu Ile Asp Ile Ser Ala His 
65                  70                  75                  80  


Trp Met Thr Asp Leu Lys Glu Gly Ile Cys Thr Gly Gly Phe Trp Phe 
                85                  90                  95      


Asn His Glu His Cys Cys Trp Asn Ser Glu His Val Thr Phe Glu Glu 
            100                 105                 110         


Arg Asp Lys Cys Pro Glu Trp Asn Ser Trp Ser Gln Leu Ile Ile Ser 
        115                 120                 125             


Thr Asp Glu Gly Ala Phe Ala Tyr Ile Val Asn Tyr Phe Met Tyr Val 
    130                 135                 140                 


Leu Trp Ala Leu Leu Phe Ala Phe Leu Ala Val Ser Leu Val Lys Val 
145                 150                 155                 160 


Phe Ala Pro Tyr Ala Cys Gly Ser Gly Ile Pro Glu Ile Lys Thr Ile 
                165                 170                 175     


Leu Ser Gly Phe Ile Ile Arg Gly Tyr Leu Gly Lys Trp Thr Leu Val 
            180                 185                 190         


Ile Lys Thr Ile Thr Leu Val Leu Ala Val Ser Ser Gly Leu Ser Leu 
        195                 200                 205             


Gly Lys Glu Gly Pro Leu Val His Val Ala Cys Cys Cys Gly Asn Ile 
    210                 215                 220                 


Leu Cys His Cys Phe Asn Lys Tyr Arg Lys Asn Glu Ala Lys Arg Arg 
225                 230                 235                 240 


Glu Val Leu Ser Ala Ala Ala Ala Ala Gly Val Ser Val Ala Phe Gly 
                245                 250                 255     


Ala Pro Ile Gly Gly Val Leu Phe Ser Leu Glu Glu Val Ser Tyr Tyr 
            260                 265                 270         


Phe Pro Leu Lys Thr Leu Trp Arg Ser Phe Phe Ala Ala Leu Val Ala 
        275                 280                 285             


Ala Phe Thr Leu Arg Ser Ile Asn Pro Phe Gly Asn Ser Arg Leu Val 
    290                 295                 300                 


Leu Phe Tyr Val Glu Phe His Thr Pro Trp His Leu Phe Glu Leu Val 
305                 310                 315                 320 


Pro Phe Ile Leu Leu Gly Ile Phe Gly Gly Leu Trp Gly Ala Leu Phe 
                325                 330                 335     


Ile Arg Thr Asn Ile Ala Trp Cys Arg Lys Arg Lys Thr Thr Gln Leu 
            340                 345                 350         


Gly Lys Tyr Pro Val Ile Glu Val Leu Val Val Thr Ala Ile Thr Ala 
        355                 360                 365             


Ile Leu Ala Phe Pro Asn Glu Tyr Thr Arg Met Ser Thr Ser Glu Leu 
    370                 375                 380                 


Ile Ser Glu Leu Phe Asn Asp Cys Gly Leu Leu Asp Ser Ser Lys Leu 
385                 390                 395                 400 


Cys Asp Tyr Glu Asn Arg Phe Asn Thr Ser Lys Gly Gly Glu Leu Pro 
                405                 410                 415     


Asp Arg Pro Ala Gly Val Gly Val Tyr Ser Ala Met Trp Gln Leu Ala 
            420                 425                 430         


Leu Thr Leu Ile Leu Lys Ile Val Ile Thr Ile Phe Thr Phe Gly Met 
        435                 440                 445             


Lys Ile Pro Ser Gly Leu Phe Ile Pro Ser Met Ala Val Gly Ala Ile 
    450                 455                 460                 


Ala Gly Arg Leu Leu Gly Val Gly Met Glu Gln Leu Ala Tyr Tyr His 
465                 470                 475                 480 


Gln Glu Trp Thr Val Phe Asn Ser Trp Cys Ser Gln Gly Ala Asp Cys 
                485                 490                 495     


Ile Thr Pro Gly Leu Tyr Ala Met Val Gly Ala Ala Ala Cys Leu Gly 
            500                 505                 510         


Gly Val Thr Arg Met Thr Val Ser Leu Val Val Ile Met Phe Glu Leu 
        515                 520                 525             


Thr Gly Gly Leu Glu Tyr Ile Val Pro Leu Met Ala Ala Ala Met Thr 
    530                 535                 540                 


Ser Lys Trp Val Ala Asp Ala Leu Gly Arg Glu Gly Ile Tyr Asp Ala 
545                 550                 555                 560 


His Ile Arg Leu Asn Gly Tyr Pro Phe Leu Glu Ala Lys Glu Glu Phe 
                565                 570                 575     


Ala His Lys Thr Leu Ala Met Asp Val Met Lys Pro Arg Arg Asn Asp 
            580                 585                 590         


Pro Leu Leu Thr Val Leu Thr Gln Asp Ser Met Thr Val Glu Asp Val 
        595                 600                 605             


Glu Thr Ile Ile Ser Glu Thr Thr Tyr Ser Gly Phe Pro Val Val Val 
    610                 615                 620                 


Ser Arg Glu Ser Gln Arg Leu Val Gly Phe Val Leu Arg Arg Asp Leu 
625                 630                 635                 640 


Ile Ile Ser Ile Glu Asn Ala Arg Lys Lys Gln Asp Gly Val Val Ser 
                645                 650                 655     


Thr Ser Ile Ile Tyr Phe Thr Glu His Ser Pro Pro Leu Pro Pro Tyr 
            660                 665                 670         


Thr Pro Pro Thr Leu Lys Leu Arg Asn Ile Leu Asp Leu Ser Pro Phe 
        675                 680                 685             


Thr Val Thr Asp Leu Thr Pro Met Glu Ile Val Val Asp Ile Phe Arg 
    690                 695                 700                 


Lys Leu Gly Leu Arg Gln Cys Leu Val Thr His Asn Gly Arg Leu Leu 
705                 710                 715                 720 


Gly Ile Ile Thr Lys Lys Asp Val Leu Lys His Ile Ala Gln Met Ala 
                725                 730                 735     


Asn Gln Asp Pro Asp Ser Ile Leu Phe Asn 
            740                 745     


<210>  4
<211>  1163
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  EF1alpha promoter

<400>  4
ggctccggtg cccgtcagtg ggcagagcgc acatcgccca cagtccccga gaagttgggg       60

ggaggggtcg gcaattgaac cggtgcctag agaaggtggc gcggggtaaa ctgggaaagt      120

gatgtcgtgt actggctccg cctttttccc gagggtgggg gagaaccgta tataagtgca      180

gtagtcgccg tgaacgttct ttttcgcaac gggtttgccg ccagaacaca ggtaagtgcc      240

gtgtgtggtt cccgcgggcc tggcctcttt acgggttatg gcccttgcgt gccttgaatt      300

acttccacct ggctccagta cgtgattctt gatcccgagc tggagccagg ggcgggcctt      360

gcgctttagg agccccttcg cctcgtgctt gagttgaggc ctggcctggg cgctggggcc      420

gccgcgtgcg aatctggtgg caccttcgcg cctgtctcgc tgctttcgat aagtctctag      480

ccatttaaaa tttttgatga cctgctgcga cgcttttttt ctggcaagat agtcttgtaa      540

atgcgggcca ggatctgcac actggtattt cggtttttgg ggccgcgggc ggcgacgggg      600

cccgtgcgtc ccagcgcaca tgttcggcga ggcggggcct gcgagcgcgg ccaccgagaa      660

tcggacgggg gtagtctcaa gctggccggc ctgctctggt gcctggcctc gcgccgccgt      720

gtatcgcccc gccctgggcg gcaaggctgg cccggtcggc accagttgcg tgagcggaaa      780

gatggccgct tcccggccct gctccagggg gctcaaaatg gaggacgcgg cgctcgggag      840

agcgggcggg tgagtcaccc acacaaagga aaggggcctt tccgtcctca gccgtcgctt      900

catgtgactc cacggagtac cgggcgccgt ccaggcacct cgattagttc tggagctttt      960

ggagtacgtc gtctttaggt tggggggagg ggttttatgc gatggagttt ccccacactg     1020

agtgggtgga gactgaagtt aggccagctt ggcacttgat gtaattctcc ttggaatttg     1080

ccctttttga gtttggatct tggttcattc tcaagcctca gacagtggtt caaagttttt     1140

ttcttccatt tcaggtgtcg tga                                             1163


<210>  5
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hCLCN5-F(cDNA) primer

<400>  5
tctcgccatg gatgttatga                                                   20


<210>  6
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hCLCN5-R(cDNA) primer

<400>  6
tcttgcgtgc gttttctatg                                                   20


<210>  7
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mCLCN5-EF primer

<400>  7
ccctggtgta gggacctatg                                                   20


<210>  8
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mCLCN5-ER primer

<400>  8
cagaattcca gcaacagtgc                                                   20


<210>  9
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CLCN5-KF2 primer

<400>  9
aagggacagt catggtctgg                                                   20


<210>  10
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CLCN5-KR2 primer

<400>  10
caatggcctg ttgtgcatac                                                   20


<210>  11
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CLCN5-W2 primer

<400>  11
ctgggtttca tgcatttgtg                                                   20


<210>  12
<211>  17
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Psi-F primer

<400>  12
tctcgacgca ggactcg                                                      17


<210>  13
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Psi-R primer

<400>  13
tactgacgct ctcgcacc                                                     18


<210>  14
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  mCLCN5 intron 2 gRNA

<400>  14
ucuggguuga ucaucuaaac                                                   20


<210>  15
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  mCLCN5 intron 5 gRNA

<400>  15
agggggccga auucuugcaa                                                   20


<210>  16
<211>  20
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  mCLCN5 exon 12 gRNA

<400>  16
gcaaugcuaa cuaguagacg                                                   20


<210>  17
<211>  48
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CLCN5 Intron 2 to Exon 12 Deletion

<400>  17
ggctagcctg gaaggaactc tctgggttgt tagaaatctg ttagtagc                    48


<210>  18
<211>  32
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Endogenous hCLCN5 partial sequence

<400>  18
atggacttct tggaggagcc aatccctggt gt                                     32


<210>  19
<211>  11
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  hCLCN5 partial sequence

<400>  19

Met Asp Phe Leu Glu Glu Pro Ile Pro Gly Val 
1               5                   10      


<210>  20
<211>  32
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimized hCLCN5 partial sequence

<400>  20
atggatttcc tggaggaacc aataccaggt gt                                     32


