                         SEQUENCE LISTING

<110>  VIVET THERAPEUTICS
 
<120>  CODON-OPTIMIZED ABCB11 TRANSGENE FOR THE TREATMENT OF PROGRESSIVE
        FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 (PFIC2)

<130>  BCT190365QT

<150>  EP18306458
<151>  2018-11-07

<160>  14    

<170>  PatentIn version 3.5

<210>  1
<211>  3966
<212>  DNA
<213>  artificial sequence

<220>
<223>  Codon-optimized sequence encoding BSEP (co-BSEP)

<400>  1
atgagcgact ccgtgattct gagatcaatc aaaaaattcg gcgaagaaaa tgacgggttc       60

gagagcgata aatcctataa taatgacaag aagtctaggc tgcaggacga gaagaagggc      120

gatggcgtgc gggtgggctt ctttcagctg ttccggttca gcagcagcac cgacatctgg      180

ctgatgtttg tgggcagcct gtgcgccttc ctgcacggca tcgcacagcc aggcgtgctg      240

ctgatctttg gcaccatgac agacgtgttc atcgactacg atgtggagct gcaggagctg      300

cagatccctg gcaaagcctg cgtgaacaat accatcgtgt ggacaaacag ctccctgaac      360

cagaatatga ccaacggcac acgctgtggc ctgctgaata tcgagtctga gatgatcaag      420

tttgccagct actatgcagg aatcgcagtg gccgtgctga tcaccggcta catccagatt      480

tgcttctggg tcatcgcagc agcaaggcag atccagaaga tgagaaagtt ctattttcgg      540

agaatcatgc ggatggagat cggctggttt gactgtaact ctgtgggcga gctgaataca      600

agattcagcg acgacatcaa caagatcaat gacgccatcg ccgatcagat ggccctgttt      660

atccagcgga tgaccagcac aatctgtggc ttcctgctgg gcttctttag aggctggaag      720

ctgaccctgg tcatcatcag cgtgtcccca ctgatcggaa tcggagcagc aacaatcggc      780

ctgtctgtga gcaagttcac cgactacgag ctgaaagcct acgccaaggc aggagtggtg      840

gcagatgaag tgatcagcag catgaggacc gtggcagcct ttggcggaga gaagagggag      900

gtggagcggt acgagaagaa cctggtgttc gcccagcggt ggggcatcag aaagggcatc      960

gtgatgggct tctttacagg cttcgtgtgg tgcctgatct tcctgtgcta cgccctggcc     1020

ttttggtatg gctccaccct ggtgctggac gagggagagt atacccctgg cacactggtg     1080

cagattttcc tgagcgtgat cgtgggcgcc ctgaacctgg gaaatgcatc cccatgcctg     1140

gaagccttcg ccacaggaag ggcagcagcc acctccatct tcgagacaat cgaccgcaag     1200

cctatcatcg actgtatgtc tgaggatggc tacaagctgg acaggatcaa gggcgagatc     1260

gagtttcaca atgtgacctt ccactatccc agccgccctg aggtgaagat cctgaacgat     1320

ctgaatatgg tcatcaagcc aggagagatg accgccctgg tgggaccctc tggagcaggc     1380

aagagcaccg ccctgcagct gatccagcgg ttttacgacc cttgcgaggg aatggtgacc     1440

gtggacggac acgacatcag gtccctgaac atccagtggc tgcgcgatca gatcggcatc     1500

gtggagcagg agccagtgct gttctctacc acaatcgccg agaatatcag atacggccgc     1560

gaggatgcca caatggagga catcgtgcag gccgccaagg aggccaacgc ctataacttc     1620

atcatggatc tgccccagca gttcgacacc ctggtgggag agggaggagg acagatgtcc     1680

ggaggccaga agcagagagt ggccatcgcc agagccctga tccgcaaccc taagatcctg     1740

ctgctggata tggccacaag cgccctggac aatgagtccg aggctatggt gcaggaggtg     1800

ctgagcaaga tccagcacgg ccacaccatc atctctgtgg cacacaggct gagcacagtg     1860

agagcagccg acaccatcat cggctttgag cacggcacag cagtggagag gggcacccac     1920

gaggagctgc tggagaggaa gggcgtgtac ttcaccctgg tgacactgca gtcccagggc     1980

aaccaggccc tgaatgagga ggacatcaag gatgccacag aggacgatat gctggcccgg     2040

accttcagca gaggctccta tcaggattct ctgagggcca gcatcaggca gcggagcaag     2100

tctcagctga gctacctggt gcacgagcca cctctggcag tggtggacca caagtccacc     2160

tatgaggagg atcgcaagga caaggacatc ccagtgcagg aggaggtgga gcctgcacca     2220

gtgaggcgca tcctgaagtt ttccgcccca gagtggccct acatgctggt gggatctgtg     2280

ggagcagcag tgaacggcac cgtgacacca ctgtatgcct tcctgttttc ccagatcctg     2340

ggcaccttct ctatccccga caaggaggag cagcggtccc agatcaatgg cgtgtgcctg     2400

ctgtttgtgg ctatgggctg cgtgagcctg tttacacagt tcctgcaggg ctacgccttc     2460

gccaagagcg gcgagctgct gaccaagcgg ctgagaaagt tcggctttag agccatgctg     2520

ggccaggaca tcgcctggtt tgacgatctg cggaacagcc caggcgccct gaccacaaga     2580

ctggccacag atgcatctca ggtgcaggga gcagcaggca gccagatcgg catgatcgtg     2640

aactccttca ccaatgtgac agtggccatg atcatcgcct tcagcttttc ctggaagctg     2700

agcctggtca tcctgtgctt cttccccttt ctggccctga gcggagcaac ccagacaagg     2760

atgctgaccg gcttcgcctc cagagacaag caggccctgg agatggtggg ccagatcaca     2820

aacgaggccc tgagcaatat caggaccgtg gcaggaatcg gcaaggagcg gcggttcatc     2880

gaggccctgg agacagagct ggagaagcct ttcaagaccg ccatccagaa ggccaacatc     2940

tacggcttct gctttgcctt cgcccagtgt atcatgttca tcgccaactc tgccagctac     3000

cgctatggcg gctacctgat cagcaatgag ggcctgcact tcagctacgt gttcagagtg     3060

atcagcgccg tggtgctgtc tgccacagcc ctgggaaggg ccttctccta caccccatct     3120

tatgccaagg ccaagatcag cgccgccagg ttctttcagc tgctggaccg ccagccaccc     3180

atcagcgtgt acaacacagc cggcgagaag tgggataatt tccagggcaa gatcgacttt     3240

gtggattgca agttcaccta tcctagcaga ccagactccc aggtgctgaa tggcctgtcc     3300

gtgtctatca gcccaggcca gacactggcc tttgtgggct cctctggctg tggcaagtcc     3360

acctctatcc agctgctgga gcggttctat gaccccgatc agggcaaagt gatgatcgac     3420

ggccacgata gcaagaaggt gaacgtgcag tttctgagat ccaatatcgg catcgtgtct     3480

caggagcctg tgctgttcgc ctgctccatc atggataaca tcaagtacgg cgacaataca     3540

aaggagatcc caatggagag agtgatcgca gcagcaaagc aggcacagct gcacgatttc     3600

gtgatgtccc tgcccgagaa gtatgagaca aacgtgggct ctcagggcag ccagctgtcc     3660

aggggcgaga agcagaggat cgcaatcgcc agggccatcg tgcgcgatcc caagatcctg     3720

ctgctggacg aggccaccag cgccctggat acagagtccg agaagaccgt gcaggtggcc     3780

ctggacaagg cccgggaggg aagaacatgt atcgtgatcg cccacagact gagcaccatc     3840

cagaatgccg acatcatcgc cgtgatggcc cagggcgtgg tcatcgagaa gggcacccac     3900

gaggaactga tggcacagaa aggggcttac tacaaactgg tcacaacagg ctcacctatc     3960

tcatag                                                                3966


<210>  2
<211>  3966
<212>  DNA
<213>  artificial sequence

<220>
<223>  Codon-optimized sequence encoding BSEP #2 (co-BSEP-2)

<400>  2
atgtctgatt ctgtgatcct gagatccatc aagaaatttg gggaagagaa tgatggcttt       60

gagtctgaca agagctacaa caatgacaag aaaagcaggc tgcaggatga gaaaaagggt      120

gatggtgtca gagtgggctt cttccagctg ttcagattca gcagcagcac agacatctgg      180

ctgatgtttg tgggcagcct gtgtgccttc ctgcatggaa ttgctcagcc tggggtgctg      240

ctgatctttg gcaccatgac agatgtgttc attgactatg atgtggaact gcaagagctg      300

cagatccctg gcaaggcttg tgtgaacaac accattgtgt ggaccaacag cagcctgaac      360

cagaacatga ccaatggcac cagatgtggc ctgctgaaca tagagtctga gatgatcaag      420

tttgccagct actatgctgg cattgctgtg gcagtgctga tcacaggcta catccagatc      480

tgcttttggg tcatagctgc tgccagacag atccagaaga tgaggaagtt ctactttaga      540

aggatcatga ggatggaaat tggatggttt gactgcaact ctgtgggaga gctgaacacc      600

agattctctg atgacatcaa caagatcaat gatgccattg ctgaccagat ggccctgttc      660

atccagagga tgaccagcac catctgtggc tttctgctgg gctttttcag aggctggaag      720

ctgaccctgg ttatcatctc tgtgtcccca ctgattggca ttggagctgc caccattggc      780

ctgtctgtgt ccaagttcac agactatgag ctgaaagcct atgccaaggc tggtgttgtg      840

gctgatgaag tgatcagctc catgagaaca gtggctgcct ttggtggtga aaagagggaa      900

gttgagagat atgagaagaa cctggtgttt gcccagagat ggggcatcag aaagggcatt      960

gtgatgggat tcttcacagg ctttgtgtgg tgcctgatct tcctgtgcta tgccctggcc     1020

ttttggtatg gcagcaccct ggttcttgat gaaggggagt acacccctgg aactctggtg     1080

cagatctttc tgtctgtgat tgtgggagcc ctgaacctgg gcaatgcctc tccatgtctg     1140

gaagcctttg ccacaggcag agctgctgct accagcatct ttgagacaat tgacagaaag     1200

cccatcattg actgcatgtc tgaggatggc tacaagctgg acaggatcaa aggggagatt     1260

gagttccaca acgtgacctt tcactacccc agcagacctg aagtgaagat cctgaatgac     1320

ctgaacatgg tcatcaagcc tggggagatg acagcccttg tgggacctag tggtgctggc     1380

aaatctacag ccctgcagct gatccagaga ttctatgacc cctgtgaagg catggtcaca     1440

gtggatggcc atgacatcag atctctgaac atccagtggc tgagggacca gattggaatt     1500

gtggaacaag agcctgtgct gttcagcacc accattgcag agaacatcag atatggcagg     1560

gaagatgcca caatggaaga tattgtgcag gctgccaaag aggccaacgc ctacaacttc     1620

atcatggacc tgcctcagca gtttgacacc cttgttggag agggtggtgg ccaaatgagt     1680

ggtggacaga aacagagagt ggccattgct agagccctga tcagaaaccc caagatcctg     1740

ctgctggaca tggctacatc tgccctggac aatgagtctg aggctatggt gcaagaggtg     1800

ctgagcaaga tccagcatgg ccacaccatc attagtgtgg cccacagact gagcacagtc     1860

agggctgctg acacaatcat tggatttgag catggcacag cagtggaaag gggcacccat     1920

gaggaactgc tggaaagaaa aggggtctac ttcaccctgg tcaccctgca gtctcagggc     1980

aatcaggccc tgaatgaaga ggacatcaag gatgccactg aggatgacat gctggccaga     2040

accttcagca gaggcagcta ccaggatagc ctgagagcca gcatcagaca gagaagcaag     2100

agccagctga gctacctggt gcatgaacct ccactggctg tggtggacca caagtccacc     2160

tatgaggaag ataggaagga caaggacatc cctgtgcaag aagaggtgga acctgctcct     2220

gtcagaagaa tcctgaagtt ttctgcccct gagtggccct acatgcttgt gggttctgtt     2280

ggggctgctg tgaatggcac agtgacccct ctgtatgcct ttctgttctc ccagatcctg     2340

ggcaccttta gcatccctga caaagaggaa cagaggtccc agatcaatgg tgtctgcctg     2400

ctctttgtgg ctatgggctg tgtgtccctg tttacccagt tcctgcaggg atatgccttt     2460

gctaagagtg gggagctgct cacaaagagg ctgagaaagt ttggcttcag agccatgctt     2520

ggccaggaca ttgcttggtt tgatgacctg agaaacagcc ctggggctct gaccacaaga     2580

ctggctacag atgctagcca ggtgcagggt gcagcaggca gccaaattgg catgattgtg     2640

aacagcttca ccaatgtgac agtggccatg atcattgcct tcagcttcag ctggaaactg     2700

agccttgtga tcctctgctt cttccccttt ctggccctgt ctggggctac ccagacaaga     2760

atgctgactg gctttgcctc cagagacaag caggccctgg aaatggttgg acagatcacc     2820

aatgaggccc tgtccaacat caggacagtg gcaggcattg gcaaagagag aagattcatt     2880

gaggcccttg agacagagct tgagaagccc ttcaagacag ccatccagaa agctaacatc     2940

tatgggttct gctttgcttt tgcccagtgc atcatgttca ttgccaactc agccagctac     3000

agatatggtg gctacctgat ctctaatgag ggcctgcact tcagctatgt gttcagagtg     3060

atctctgctg tggtgctgtc tgccactgct ctgggcagag cctttagcta cacccctagc     3120

tatgccaaag ccaagatctc tgcagccaga ttctttcagc tgctggatag acagcctcct     3180

atcagtgtgt acaacacagc tggggagaag tgggacaact tccagggcaa gattgacttt     3240

gtggattgca agttcaccta tcctagcaga ccagactctc aggtgctgaa tggactgagt     3300

gtgtctatca gccctggcca gacactggcc tttgtgggaa gctctggatg tggcaagagc     3360

accagcatcc agctgcttga gaggttctat gatccagacc agggcaaagt gatgattgat     3420

gggcatgaca gcaagaaagt gaatgtgcag ttcctgaggt ccaacattgg gattgtgtcc     3480

caagaacctg ttctgtttgc ctgcagcatc atggataaca ttaagtatgg ggacaacacc     3540

aaagaaatcc ctatggaaag agtgattgct gcagccaagc aggcacagct gcatgatttt     3600

gtgatgagcc tgcctgagaa gtatgagaca aatgtgggct cccagggcag ccagctgtct     3660

agaggggaaa aacagagaat tgccatagcc agggccatag tcagagatcc taagattctg     3720

ctcctggatg aggccacctc tgctctggat acagagtctg aaaagacagt ccaggtggca     3780

ctggacaagg ccagagaggg cagaacctgt attgtgattg cccataggct gtccacaatc     3840

caaaatgctg acatcattgc agtgatggcc caaggggttg tgattgagaa gggaacacat     3900

gaagaactca tggcccaaaa aggggcctat tataagctgg tcaccactgg cagccccatc     3960

agctag                                                                3966


<210>  3
<211>  4734
<212>  DNA
<213>  artificial sequence

<220>
<223>  recombinant AAV vector comprising codon-optimized sequence 
       encoding BSEP

<400>  3
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgcg aattccatgg taccaggcat caagacacgt      180

gcgccacccc ctccaccttg gacacaggac gctgtggttt ctgagccagg tacaatgact      240

cctttcggta agtgcagtgg aagctgtaca ctgcccaggc aaagcgtccg ggcagcgtag      300

gcgggcgact cagatcccag ccagtggact tagcccctgt ttgctcctcc gataactggg      360

gtgaccttgg ttaatattca ccagcagcct cccccgttgc ccctctggat ccactgctta      420

aatacggacg aggacagggc cctgtctcct cagcttcagg caccaccact gacctgggac      480

agtgaaggcc tgtcgacgga tccgagctcg ccgccaccat gagcgactcc gtgattctga      540

gatcaatcaa aaaattcggc gaagaaaatg acgggttcga gagcgataaa tcctataata      600

atgacaagaa gtctaggctg caggacgaga agaagggcga tggcgtgcgg gtgggcttct      660

ttcagctgtt ccggttcagc agcagcaccg acatctggct gatgtttgtg ggcagcctgt      720

gcgccttcct gcacggcatc gcacagccag gcgtgctgct gatctttggc accatgacag      780

acgtgttcat cgactacgat gtggagctgc aggagctgca gatccctggc aaagcctgcg      840

tgaacaatac catcgtgtgg acaaacagct ccctgaacca gaatatgacc aacggcacac      900

gctgtggcct gctgaatatc gagtctgaga tgatcaagtt tgccagctac tatgcaggaa      960

tcgcagtggc cgtgctgatc accggctaca tccagatttg cttctgggtc atcgcagcag     1020

caaggcagat ccagaagatg agaaagttct attttcggag aatcatgcgg atggagatcg     1080

gctggtttga ctgtaactct gtgggcgagc tgaatacaag attcagcgac gacatcaaca     1140

agatcaatga cgccatcgcc gatcagatgg ccctgtttat ccagcggatg accagcacaa     1200

tctgtggctt cctgctgggc ttctttagag gctggaagct gaccctggtc atcatcagcg     1260

tgtccccact gatcggaatc ggagcagcaa caatcggcct gtctgtgagc aagttcaccg     1320

actacgagct gaaagcctac gccaaggcag gagtggtggc agatgaagtg atcagcagca     1380

tgaggaccgt ggcagccttt ggcggagaga agagggaggt ggagcggtac gagaagaacc     1440

tggtgttcgc ccagcggtgg ggcatcagaa agggcatcgt gatgggcttc tttacaggct     1500

tcgtgtggtg cctgatcttc ctgtgctacg ccctggcctt ttggtatggc tccaccctgg     1560

tgctggacga gggagagtat acccctggca cactggtgca gattttcctg agcgtgatcg     1620

tgggcgccct gaacctggga aatgcatccc catgcctgga agccttcgcc acaggaaggg     1680

cagcagccac ctccatcttc gagacaatcg accgcaagcc tatcatcgac tgtatgtctg     1740

aggatggcta caagctggac aggatcaagg gcgagatcga gtttcacaat gtgaccttcc     1800

actatcccag ccgccctgag gtgaagatcc tgaacgatct gaatatggtc atcaagccag     1860

gagagatgac cgccctggtg ggaccctctg gagcaggcaa gagcaccgcc ctgcagctga     1920

tccagcggtt ttacgaccct tgcgagggaa tggtgaccgt ggacggacac gacatcaggt     1980

ccctgaacat ccagtggctg cgcgatcaga tcggcatcgt ggagcaggag ccagtgctgt     2040

tctctaccac aatcgccgag aatatcagat acggccgcga ggatgccaca atggaggaca     2100

tcgtgcaggc cgccaaggag gccaacgcct ataacttcat catggatctg ccccagcagt     2160

tcgacaccct ggtgggagag ggaggaggac agatgtccgg aggccagaag cagagagtgg     2220

ccatcgccag agccctgatc cgcaacccta agatcctgct gctggatatg gccacaagcg     2280

ccctggacaa tgagtccgag gctatggtgc aggaggtgct gagcaagatc cagcacggcc     2340

acaccatcat ctctgtggca cacaggctga gcacagtgag agcagccgac accatcatcg     2400

gctttgagca cggcacagca gtggagaggg gcacccacga ggagctgctg gagaggaagg     2460

gcgtgtactt caccctggtg acactgcagt cccagggcaa ccaggccctg aatgaggagg     2520

acatcaagga tgccacagag gacgatatgc tggcccggac cttcagcaga ggctcctatc     2580

aggattctct gagggccagc atcaggcagc ggagcaagtc tcagctgagc tacctggtgc     2640

acgagccacc tctggcagtg gtggaccaca agtccaccta tgaggaggat cgcaaggaca     2700

aggacatccc agtgcaggag gaggtggagc ctgcaccagt gaggcgcatc ctgaagtttt     2760

ccgccccaga gtggccctac atgctggtgg gatctgtggg agcagcagtg aacggcaccg     2820

tgacaccact gtatgccttc ctgttttccc agatcctggg caccttctct atccccgaca     2880

aggaggagca gcggtcccag atcaatggcg tgtgcctgct gtttgtggct atgggctgcg     2940

tgagcctgtt tacacagttc ctgcagggct acgccttcgc caagagcggc gagctgctga     3000

ccaagcggct gagaaagttc ggctttagag ccatgctggg ccaggacatc gcctggtttg     3060

acgatctgcg gaacagccca ggcgccctga ccacaagact ggccacagat gcatctcagg     3120

tgcagggagc agcaggcagc cagatcggca tgatcgtgaa ctccttcacc aatgtgacag     3180

tggccatgat catcgccttc agcttttcct ggaagctgag cctggtcatc ctgtgcttct     3240

tcccctttct ggccctgagc ggagcaaccc agacaaggat gctgaccggc ttcgcctcca     3300

gagacaagca ggccctggag atggtgggcc agatcacaaa cgaggccctg agcaatatca     3360

ggaccgtggc aggaatcggc aaggagcggc ggttcatcga ggccctggag acagagctgg     3420

agaagccttt caagaccgcc atccagaagg ccaacatcta cggcttctgc tttgccttcg     3480

cccagtgtat catgttcatc gccaactctg ccagctaccg ctatggcggc tacctgatca     3540

gcaatgaggg cctgcacttc agctacgtgt tcagagtgat cagcgccgtg gtgctgtctg     3600

ccacagccct gggaagggcc ttctcctaca ccccatctta tgccaaggcc aagatcagcg     3660

ccgccaggtt ctttcagctg ctggaccgcc agccacccat cagcgtgtac aacacagccg     3720

gcgagaagtg ggataatttc cagggcaaga tcgactttgt ggattgcaag ttcacctatc     3780

ctagcagacc agactcccag gtgctgaatg gcctgtccgt gtctatcagc ccaggccaga     3840

cactggcctt tgtgggctcc tctggctgtg gcaagtccac ctctatccag ctgctggagc     3900

ggttctatga ccccgatcag ggcaaagtga tgatcgacgg ccacgatagc aagaaggtga     3960

acgtgcagtt tctgagatcc aatatcggca tcgtgtctca ggagcctgtg ctgttcgcct     4020

gctccatcat ggataacatc aagtacggcg acaatacaaa ggagatccca atggagagag     4080

tgatcgcagc agcaaagcag gcacagctgc acgatttcgt gatgtccctg cccgagaagt     4140

atgagacaaa cgtgggctct cagggcagcc agctgtccag gggcgagaag cagaggatcg     4200

caatcgccag ggccatcgtg cgcgatccca agatcctgct gctggacgag gccaccagcg     4260

ccctggatac agagtccgag aagaccgtgc aggtggccct ggacaaggcc cgggagggaa     4320

gaacatgtat cgtgatcgcc cacagactga gcaccatcca gaatgccgac atcatcgccg     4380

tgatggccca gggcgtggtc atcgagaagg gcacccacga ggaactgatg gcacagaaag     4440

gggcttacta caaactggtc acaacaggct cacctatctc atagggatcc atatgatatc     4500

aataaagacc tcttattttc attcatcagg tgtggttggt ttttttgtgt gggggctcga     4560

gatctgagga acccctagtg atggaggcgg ccgcaggaac ccctagtgat ggagttggcc     4620

actccctctc tgcgcgctcg ctcgctcact gaggccgggc gaccaaaggt cgcccgacgc     4680

ccgggctttg cccgggcggc ctcagtgagc gagcgagcgc gcagctgcct gcag           4734


<210>  4
<211>  4734
<212>  DNA
<213>  artificial sequence

<220>
<223>  recombinant AAV vector comprising codon-optimized sequence 
       encoding BSEP #2

<400>  4
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag cccgggcgtc       60

gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag ggagtggcca      120

actccatcac taggggttcc tgcggccgcg aattccatgg taccaggcat caagacacgt      180

gcgccacccc ctccaccttg gacacaggac gctgtggttt ctgagccagg tacaatgact      240

cctttcggta agtgcagtgg aagctgtaca ctgcccaggc aaagcgtccg ggcagcgtag      300

gcgggcgact cagatcccag ccagtggact tagcccctgt ttgctcctcc gataactggg      360

gtgaccttgg ttaatattca ccagcagcct cccccgttgc ccctctggat ccactgctta      420

aatacggacg aggacagggc cctgtctcct cagcttcagg caccaccact gacctgggac      480

agtgaaggcc tgtcgacgga tccgagctcg ccgccaccat gtctgattct gtgatcctga      540

gatccatcaa gaaatttggg gaagagaatg atggctttga gtctgacaag agctacaaca      600

atgacaagaa aagcaggctg caggatgaga aaaagggtga tggtgtcaga gtgggcttct      660

tccagctgtt cagattcagc agcagcacag acatctggct gatgtttgtg ggcagcctgt      720

gtgccttcct gcatggaatt gctcagcctg gggtgctgct gatctttggc accatgacag      780

atgtgttcat tgactatgat gtggaactgc aagagctgca gatccctggc aaggcttgtg      840

tgaacaacac cattgtgtgg accaacagca gcctgaacca gaacatgacc aatggcacca      900

gatgtggcct gctgaacata gagtctgaga tgatcaagtt tgccagctac tatgctggca      960

ttgctgtggc agtgctgatc acaggctaca tccagatctg cttttgggtc atagctgctg     1020

ccagacagat ccagaagatg aggaagttct actttagaag gatcatgagg atggaaattg     1080

gatggtttga ctgcaactct gtgggagagc tgaacaccag attctctgat gacatcaaca     1140

agatcaatga tgccattgct gaccagatgg ccctgttcat ccagaggatg accagcacca     1200

tctgtggctt tctgctgggc tttttcagag gctggaagct gaccctggtt atcatctctg     1260

tgtccccact gattggcatt ggagctgcca ccattggcct gtctgtgtcc aagttcacag     1320

actatgagct gaaagcctat gccaaggctg gtgttgtggc tgatgaagtg atcagctcca     1380

tgagaacagt ggctgccttt ggtggtgaaa agagggaagt tgagagatat gagaagaacc     1440

tggtgtttgc ccagagatgg ggcatcagaa agggcattgt gatgggattc ttcacaggct     1500

ttgtgtggtg cctgatcttc ctgtgctatg ccctggcctt ttggtatggc agcaccctgg     1560

ttcttgatga aggggagtac acccctggaa ctctggtgca gatctttctg tctgtgattg     1620

tgggagccct gaacctgggc aatgcctctc catgtctgga agcctttgcc acaggcagag     1680

ctgctgctac cagcatcttt gagacaattg acagaaagcc catcattgac tgcatgtctg     1740

aggatggcta caagctggac aggatcaaag gggagattga gttccacaac gtgacctttc     1800

actaccccag cagacctgaa gtgaagatcc tgaatgacct gaacatggtc atcaagcctg     1860

gggagatgac agcccttgtg ggacctagtg gtgctggcaa atctacagcc ctgcagctga     1920

tccagagatt ctatgacccc tgtgaaggca tggtcacagt ggatggccat gacatcagat     1980

ctctgaacat ccagtggctg agggaccaga ttggaattgt ggaacaagag cctgtgctgt     2040

tcagcaccac cattgcagag aacatcagat atggcaggga agatgccaca atggaagata     2100

ttgtgcaggc tgccaaagag gccaacgcct acaacttcat catggacctg cctcagcagt     2160

ttgacaccct tgttggagag ggtggtggcc aaatgagtgg tggacagaaa cagagagtgg     2220

ccattgctag agccctgatc agaaacccca agatcctgct gctggacatg gctacatctg     2280

ccctggacaa tgagtctgag gctatggtgc aagaggtgct gagcaagatc cagcatggcc     2340

acaccatcat tagtgtggcc cacagactga gcacagtcag ggctgctgac acaatcattg     2400

gatttgagca tggcacagca gtggaaaggg gcacccatga ggaactgctg gaaagaaaag     2460

gggtctactt caccctggtc accctgcagt ctcagggcaa tcaggccctg aatgaagagg     2520

acatcaagga tgccactgag gatgacatgc tggccagaac cttcagcaga ggcagctacc     2580

aggatagcct gagagccagc atcagacaga gaagcaagag ccagctgagc tacctggtgc     2640

atgaacctcc actggctgtg gtggaccaca agtccaccta tgaggaagat aggaaggaca     2700

aggacatccc tgtgcaagaa gaggtggaac ctgctcctgt cagaagaatc ctgaagtttt     2760

ctgcccctga gtggccctac atgcttgtgg gttctgttgg ggctgctgtg aatggcacag     2820

tgacccctct gtatgccttt ctgttctccc agatcctggg cacctttagc atccctgaca     2880

aagaggaaca gaggtcccag atcaatggtg tctgcctgct ctttgtggct atgggctgtg     2940

tgtccctgtt tacccagttc ctgcagggat atgcctttgc taagagtggg gagctgctca     3000

caaagaggct gagaaagttt ggcttcagag ccatgcttgg ccaggacatt gcttggtttg     3060

atgacctgag aaacagccct ggggctctga ccacaagact ggctacagat gctagccagg     3120

tgcagggtgc agcaggcagc caaattggca tgattgtgaa cagcttcacc aatgtgacag     3180

tggccatgat cattgccttc agcttcagct ggaaactgag ccttgtgatc ctctgcttct     3240

tcccctttct ggccctgtct ggggctaccc agacaagaat gctgactggc tttgcctcca     3300

gagacaagca ggccctggaa atggttggac agatcaccaa tgaggccctg tccaacatca     3360

ggacagtggc aggcattggc aaagagagaa gattcattga ggcccttgag acagagcttg     3420

agaagccctt caagacagcc atccagaaag ctaacatcta tgggttctgc tttgcttttg     3480

cccagtgcat catgttcatt gccaactcag ccagctacag atatggtggc tacctgatct     3540

ctaatgaggg cctgcacttc agctatgtgt tcagagtgat ctctgctgtg gtgctgtctg     3600

ccactgctct gggcagagcc tttagctaca cccctagcta tgccaaagcc aagatctctg     3660

cagccagatt ctttcagctg ctggatagac agcctcctat cagtgtgtac aacacagctg     3720

gggagaagtg ggacaacttc cagggcaaga ttgactttgt ggattgcaag ttcacctatc     3780

ctagcagacc agactctcag gtgctgaatg gactgagtgt gtctatcagc cctggccaga     3840

cactggcctt tgtgggaagc tctggatgtg gcaagagcac cagcatccag ctgcttgaga     3900

ggttctatga tccagaccag ggcaaagtga tgattgatgg gcatgacagc aagaaagtga     3960

atgtgcagtt cctgaggtcc aacattggga ttgtgtccca agaacctgtt ctgtttgcct     4020

gcagcatcat ggataacatt aagtatgggg acaacaccaa agaaatccct atggaaagag     4080

tgattgctgc agccaagcag gcacagctgc atgattttgt gatgagcctg cctgagaagt     4140

atgagacaaa tgtgggctcc cagggcagcc agctgtctag aggggaaaaa cagagaattg     4200

ccatagccag ggccatagtc agagatccta agattctgct cctggatgag gccacctctg     4260

ctctggatac agagtctgaa aagacagtcc aggtggcact ggacaaggcc agagagggca     4320

gaacctgtat tgtgattgcc cataggctgt ccacaatcca aaatgctgac atcattgcag     4380

tgatggccca aggggttgtg attgagaagg gaacacatga agaactcatg gcccaaaaag     4440

gggcctatta taagctggtc accactggca gccccatcag ctagggatcc atatgatatc     4500

aataaagacc tcttattttc attcatcagg tgtggttggt ttttttgtgt gggggctcga     4560

gatctgagga acccctagtg atggaggcgg ccgcaggaac ccctagtgat ggagttggcc     4620

actccctctc tgcgcgctcg ctcgctcact gaggccgggc gaccaaaggt cgcccgacgc     4680

ccgggctttg cccgggcggc ctcagtgagc gagcgagcgc gcagctgcct gcag           4734


<210>  5
<211>  255
<212>  DNA
<213>  artificial sequence

<220>
<223>  Alpha 1 antitrypsin promoter

<400>  5
cgccaccccc tccaccttgg acacaggacg ctgtggtttc tgagccaggt acaatgactc       60

ctttcggtaa gtgcagtgga agctgtacac tgcccaggca aagcgtccgg gcagcgtagg      120

cgggcgactc agatcccagc cagtggactt agcccctgtt tgctcctccg ataactgggg      180

tgaccttggt taatattcac cagcagcctc ccccgttgcc cctctggatc cactgcttaa      240

atacggacga ggaca                                                       255


<210>  6
<211>  145
<212>  DNA
<213>  artificial sequence

<220>
<223>  Human minimal bile salt export pump (ABCB11) gene promoter

<400>  6
ttcccaagca cactctgtgt ttggggttat tgctctgagt atgtttctcg tatgtcactg       60

aactgtgctt gggctgccct tagggacatt gatccttagg caaatagata atgttcttga      120

aaaagtttga attctgttca gtgct                                            145


<210>  7
<211>  145
<212>  DNA
<213>  artificial sequence

<220>
<223>  Mouse minimal Bile Salt export pump (ABCB11) gene promoter

<400>  7
ggttcctgct ttgagtatgt tcgacctttc ctctcatgtc actgaactgt gctagatctg       60

gactttaggc cattgaccta taagcaaata gatagtgttc ttaaaaaagc ctgatttctg      120

ttcaatgctt tattaccatg aaaac                                            145


<210>  8
<211>  56
<212>  DNA
<213>  artificial sequence

<220>
<223>  Synthetic poly A sequence

<400>  8
aataaagacc tcttattttc attcatcagg tgtggttggt ttttttgtgt gggggc           56


<210>  9
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  Forward alpha-1-anti-trypsin promoter primer

<400>  9
ttgctcctcc gataactggg                                                   20


<210>  10
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  Reverse alpha-1-anti-trypsin promoter primer

<400>  10
ccctgtcctc gtccgtattt                                                   20


<210>  11
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  Forward wild type BSEP primer

<400>  11
tcatccgaaa tcccaagatt                                                   20


<210>  12
<211>  19
<212>  DNA
<213>  artificial sequence

<220>
<223>  Reverse   wild type BSEP primer

<400>  12
caagcgatga gcaactgaa                                                    19


<210>  13
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  Forward codon-optimized BSEP primer

<400>  13
taatttccag ggcaagatcg                                                   20


<210>  14
<211>  20
<212>  DNA
<213>  artificial sequence

<220>
<223>  Reverse codon-optimized BSEP primer

<400>  14
agcagctgga tagaggtgga                                                   20


