                         SEQUENCE LISTING

<110>  Genevant Sciences GmbH
 
<120>  ORNITHINE TRANSCARBAMYLASE (OTC) CONSTRUCTS AND METHODS OF USING 
       THE SAME

<130>  4170.021PC01

<150>  US 62/924,567
<151>  2019-10-22


<160>  9     

<170>  PatentIn version 3.5

<210>  1
<211>  1065
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  ORF human  OTC mRNA codon optimized for human expression

<400>  1
augcuguuua accugaggau ucugcugaac aacgcugcuu uucggaacgg ccacaacuuu       60

auggugcgga acuuucggug cggacagcca cugcagaaca aagugcagcu gaaggggagg      120

gaccugcuga cccugaaaaa uuucacagga gaggaaauca aguacaugcu guggcugucu      180

gccgaucuga aguuccggau caagcagaag ggcgaauauc ugccacugcu gcagggcaaa      240

agucugggga ugaucuucga aaagaggagu acucggacca gacugucaac agagacugga      300

uucgcucugc ugggaggaca cccaugcuuu cugaccacac aggacauuca ucugggcgug      360

aacgagucac ugaccgacac agcucgaguc cugagcucca uggcagaugc cgugcuggca      420

cgggucuaca aacagagcga ccuggauacc cuggcuaagg aagcaagcau ccccaucauu      480

aaugggcugu ccgaccugua ucacccuauc cagauucugg ccgauuaccu gacccugcag      540

gagcauuauu cuagucugaa aggccugaca cugagcugga uuggggacgg aaacaauauc      600

cugcacucca uuaugauguc ugccgcuaag uuuggaaugc aucugcaggc agccacacca      660

aaaggcuacg aacccgaugc cagugugacu aagcuggccg aacaguaugc uaaagagaac      720

ggcacuaagc ugcugcugac caaugacccu cuggaggcug cacacggagg caacguccug      780

aucacugaua ccuggauuuc caugggccag gaggaagaga agaaaaagcg ccugcaggca      840

uuccaggggu accaggugac aaugaaaacu gccaaggucg ccgcuucuga uuggacuuuu      900

cugcauuguc ugccccgaaa accugaagag guggacgaug aggucuucua uucaccuagg      960

agccuggugu uuccagaagc cgagaaucgc aaguggacaa ucauggcugu gauggugucc     1020

cugcugacug auuauucccc ccagcugcag aaaccuaagu ucuga                     1065


<210>  2
<211>  47
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  5' UTR

<400>  2
aggaaauaag agagaaaaga agaguaagaa gaaauauaag agccacc                     47


<210>  3
<211>  114
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  3' UTR

<400>  3
gcggccgcuu aauuaagcug ccuucugcgg ggcuugccuu cuggccaugc ccuucuucuc       60

ucccuugcac cuguaccucu uggucuuuga auaaagccug aguaggaagu cuag            114


<210>  4
<211>  1226
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  5' UTR, ORF human  OTC mRNA codon optimized for human expression,
       3' UTR

<400>  4
aggaaauaag agagaaaaga agaguaagaa gaaauauaag agccaccaug cuguuuaacc       60

ugaggauucu gcugaacaac gcugcuuuuc ggaacggcca caacuuuaug gugcggaacu      120

uucggugcgg acagccacug cagaacaaag ugcagcugaa ggggagggac cugcugaccc      180

ugaaaaauuu cacaggagag gaaaucaagu acaugcugug gcugucugcc gaucugaagu      240

uccggaucaa gcagaagggc gaauaucugc cacugcugca gggcaaaagu cuggggauga      300

ucuucgaaaa gaggaguacu cggaccagac ugucaacaga gacuggauuc gcucugcugg      360

gaggacaccc augcuuucug accacacagg acauucaucu gggcgugaac gagucacuga      420

ccgacacagc ucgaguccug agcuccaugg cagaugccgu gcuggcacgg gucuacaaac      480

agagcgaccu ggauacccug gcuaaggaag caagcauccc caucauuaau gggcuguccg      540

accuguauca cccuauccag auucuggccg auuaccugac ccugcaggag cauuauucua      600

gucugaaagg ccugacacug agcuggauug gggacggaaa caauauccug cacuccauua      660

ugaugucugc cgcuaaguuu ggaaugcauc ugcaggcagc cacaccaaaa ggcuacgaac      720

ccgaugccag ugugacuaag cuggccgaac aguaugcuaa agagaacggc acuaagcugc      780

ugcugaccaa ugacccucug gaggcugcac acggaggcaa cguccugauc acugauaccu      840

ggauuuccau gggccaggag gaagagaaga aaaagcgccu gcaggcauuc cagggguacc      900

aggugacaau gaaaacugcc aaggucgccg cuucugauug gacuuuucug cauugucugc      960

cccgaaaacc ugaagaggug gacgaugagg ucuucuauuc accuaggagc cugguguuuc     1020

cagaagccga gaaucgcaag uggacaauca uggcugugau ggugucccug cugacugauu     1080

auucccccca gcugcagaaa ccuaaguucu gagcggccgc uuaauuaagc ugccuucugc     1140

ggggcuugcc uucuggccau gcccuucuuc ucucccuugc accuguaccu cuuggucuuu     1200

gaauaaagcc ugaguaggaa gucuag                                          1226


<210>  5
<211>  1311
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  5' UTR, ORF human  OTC mRNA codon optimized for human 
       expression, 3' UTR, and polyA (80)

<400>  5
aggaaauaag agagaaaaga agaguaagaa gaaauauaag agccaccaug cuguuuaacc       60

ugaggauucu gcugaacaac gcugcuuuuc ggaacggcca caacuuuaug gugcggaacu      120

uucggugcgg acagccacug cagaacaaag ugcagcugaa ggggagggac cugcugaccc      180

ugaaaaauuu cacaggagag gaaaucaagu acaugcugug gcugucugcc gaucugaagu      240

uccggaucaa gcagaagggc gaauaucugc cacugcugca gggcaaaagu cuggggauga      300

ucuucgaaaa gaggaguacu cggaccagac ugucaacaga gacuggauuc gcucugcugg      360

gaggacaccc augcuuucug accacacagg acauucaucu gggcgugaac gagucacuga      420

ccgacacagc ucgaguccug agcuccaugg cagaugccgu gcuggcacgg gucuacaaac      480

agagcgaccu ggauacccug gcuaaggaag caagcauccc caucauuaau gggcuguccg      540

accuguauca cccuauccag auucuggccg auuaccugac ccugcaggag cauuauucua      600

gucugaaagg ccugacacug agcuggauug gggacggaaa caauauccug cacuccauua      660

ugaugucugc cgcuaaguuu ggaaugcauc ugcaggcagc cacaccaaaa ggcuacgaac      720

ccgaugccag ugugacuaag cuggccgaac aguaugcuaa agagaacggc acuaagcugc      780

ugcugaccaa ugacccucug gaggcugcac acggaggcaa cguccugauc acugauaccu      840

ggauuuccau gggccaggag gaagagaaga aaaagcgccu gcaggcauuc cagggguacc      900

aggugacaau gaaaacugcc aaggucgccg cuucugauug gacuuuucug cauugucugc      960

cccgaaaacc ugaagaggug gacgaugagg ucuucuauuc accuaggagc cugguguuuc     1020

cagaagccga gaaucgcaag uggacaauca uggcugugau ggugucccug cugacugauu     1080

auucccccca gcugcagaaa ccuaaguucu gagcggccgc uuaauuaagc ugccuucugc     1140

ggggcuugcc uucuggccau gcccuucuuc ucucccuugc accuguaccu cuuggucuuu     1200

gaauaaagcc ugaguaggaa gucuagaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1260

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaagc u              1311


<210>  6
<211>  1666
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  5' UTR, ORF human  OTC mRNA codon optimized for human 
       expression, 3' UTR, and polyA

<400>  6
aggaaauaag agagaaaaga agaguaagaa gaaauauaag agccaccaug cuguuuaacc       60

ugaggauucu gcugaacaac gcugcuuuuc ggaacggcca caacuuuaug gugcggaacu      120

uucggugcgg acagccacug cagaacaaag ugcagcugaa ggggagggac cugcugaccc      180

ugaaaaauuu cacaggagag gaaaucaagu acaugcugug gcugucugcc gaucugaagu      240

uccggaucaa gcagaagggc gaauaucugc cacugcugca gggcaaaagu cuggggauga      300

ucuucgaaaa gaggaguacu cggaccagac ugucaacaga gacuggauuc gcucugcugg      360

gaggacaccc augcuuucug accacacagg acauucaucu gggcgugaac gagucacuga      420

ccgacacagc ucgaguccug agcuccaugg cagaugccgu gcuggcacgg gucuacaaac      480

agagcgaccu ggauacccug gcuaaggaag caagcauccc caucauuaau gggcuguccg      540

accuguauca cccuauccag auucuggccg auuaccugac ccugcaggag cauuauucua      600

gucugaaagg ccugacacug agcuggauug gggacggaaa caauauccug cacuccauua      660

ugaugucugc cgcuaaguuu ggaaugcauc ugcaggcagc cacaccaaaa ggcuacgaac      720

ccgaugccag ugugacuaag cuggccgaac aguaugcuaa agagaacggc acuaagcugc      780

ugcugaccaa ugacccucug gaggcugcac acggaggcaa cguccugauc acugauaccu      840

ggauuuccau gggccaggag gaagagaaga aaaagcgccu gcaggcauuc cagggguacc      900

aggugacaau gaaaacugcc aaggucgccg cuucugauug gacuuuucug cauugucugc      960

cccgaaaacc ugaagaggug gacgaugagg ucuucuauuc accuaggagc cugguguuuc     1020

cagaagccga gaaucgcaag uggacaauca uggcugugau ggugucccug cugacugauu     1080

auucccccca gcugcagaaa ccuaaguucu gagcggccgc uuaauuaagc ugccuucugc     1140

ggggcuugcc uucuggccau gcccuucuuc ucucccuugc accuguaccu cuuggucuuu     1200

gaauaaagcc ugaguaggaa gucuagaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1260

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1320

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1380

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1440

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1500

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1560

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa     1620

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaa                    1666


<210>  7
<211>  354
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  human  OTC

<400>  7

Met Leu Phe Asn Leu Arg Ile Leu Leu Asn Asn Ala Ala Phe Arg Asn 
1               5                   10                  15      


Gly His Asn Phe Met Val Arg Asn Phe Arg Cys Gly Gln Pro Leu Gln 
            20                  25                  30          


Asn Lys Val Gln Leu Lys Gly Arg Asp Leu Leu Thr Leu Lys Asn Phe 
        35                  40                  45              


Thr Gly Glu Glu Ile Lys Tyr Met Leu Trp Leu Ser Ala Asp Leu Lys 
    50                  55                  60                  


Phe Arg Ile Lys Gln Lys Gly Glu Tyr Leu Pro Leu Leu Gln Gly Lys 
65                  70                  75                  80  


Ser Leu Gly Met Ile Phe Glu Lys Arg Ser Thr Arg Thr Arg Leu Ser 
                85                  90                  95      


Thr Glu Thr Gly Phe Ala Leu Leu Gly Gly His Pro Cys Phe Leu Thr 
            100                 105                 110         


Thr Gln Asp Ile His Leu Gly Val Asn Glu Ser Leu Thr Asp Thr Ala 
        115                 120                 125             


Arg Val Leu Ser Ser Met Ala Asp Ala Val Leu Ala Arg Val Tyr Lys 
    130                 135                 140                 


Gln Ser Asp Leu Asp Thr Leu Ala Lys Glu Ala Ser Ile Pro Ile Ile 
145                 150                 155                 160 


Asn Gly Leu Ser Asp Leu Tyr His Pro Ile Gln Ile Leu Ala Asp Tyr 
                165                 170                 175     


Leu Thr Leu Gln Glu His Tyr Ser Ser Leu Lys Gly Leu Thr Leu Ser 
            180                 185                 190         


Trp Ile Gly Asp Gly Asn Asn Ile Leu His Ser Ile Met Met Ser Ala 
        195                 200                 205             


Ala Lys Phe Gly Met His Leu Gln Ala Ala Thr Pro Lys Gly Tyr Glu 
    210                 215                 220                 


Pro Asp Ala Ser Val Thr Lys Leu Ala Glu Gln Tyr Ala Lys Glu Asn 
225                 230                 235                 240 


Gly Thr Lys Leu Leu Leu Thr Asn Asp Pro Leu Glu Ala Ala His Gly 
                245                 250                 255     


Gly Asn Val Leu Ile Thr Asp Thr Trp Ile Ser Met Gly Gln Glu Glu 
            260                 265                 270         


Glu Lys Lys Lys Arg Leu Gln Ala Phe Gln Gly Tyr Gln Val Thr Met 
        275                 280                 285             


Lys Thr Ala Lys Val Ala Ala Ser Asp Trp Thr Phe Leu His Cys Leu 
    290                 295                 300                 


Pro Arg Lys Pro Glu Glu Val Asp Asp Glu Val Phe Tyr Ser Pro Arg 
305                 310                 315                 320 


Ser Leu Val Phe Pro Glu Ala Glu Asn Arg Lys Trp Thr Ile Met Ala 
                325                 330                 335     


Val Met Val Ser Leu Leu Thr Asp Tyr Ser Pro Gln Leu Gln Lys Pro 
            340                 345                 350         


Lys Phe 
        


<210>  8
<211>  1226
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  DNA sequence for 3' UTR, human OTC open reading frame and 5' UTR

<400>  8
aggaaataag agagaaaaga agagtaagaa gaaatataag agccaccatg ctgtttaacc       60

tgaggattct gctgaacaac gctgcttttc ggaacggcca caactttatg gtgcggaact      120

ttcggtgcgg acagccactg cagaacaaag tgcagctgaa ggggagggac ctgctgaccc      180

tgaaaaattt cacaggagag gaaatcaagt acatgctgtg gctgtctgcc gatctgaagt      240

tccggatcaa gcagaagggc gaatatctgc cactgctgca gggcaaaagt ctggggatga      300

tcttcgaaaa gaggagtact cggaccagac tgtcaacaga gactggattc gctctgctgg      360

gaggacaccc atgctttctg accacacagg acattcatct gggcgtgaac gagtcactga      420

ccgacacagc tcgagtcctg agctccatgg cagatgccgt gctggcacgg gtctacaaac      480

agagcgacct ggataccctg gctaaggaag caagcatccc catcattaat gggctgtccg      540

acctgtatca ccctatccag attctggccg attacctgac cctgcaggag cattattcta      600

gtctgaaagg cctgacactg agctggattg gggacggaaa caatatcctg cactccatta      660

tgatgtctgc cgctaagttt ggaatgcatc tgcaggcagc cacaccaaaa ggctacgaac      720

ccgatgccag tgtgactaag ctggccgaac agtatgctaa agagaacggc actaagctgc      780

tgctgaccaa tgaccctctg gaggctgcac acggaggcaa cgtcctgatc actgatacct      840

ggatttccat gggccaggag gaagagaaga aaaagcgcct gcaggcattc caggggtacc      900

aggtgacaat gaaaactgcc aaggtcgccg cttctgattg gacttttctg cattgtctgc      960

cccgaaaacc tgaagaggtg gacgatgagg tcttctattc acctaggagc ctggtgtttc     1020

cagaagccga gaatcgcaag tggacaatca tggctgtgat ggtgtccctg ctgactgatt     1080

attcccccca gctgcagaaa cctaagttct gagcggccgc ttaattaagc tgccttctgc     1140

ggggcttgcc ttctggccat gcccttcttc tctcccttgc acctgtacct cttggtcttt     1200

gaataaagcc tgagtaggaa gtctag                                          1226


<210>  9
<211>  17
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  T7 Promoter

<400>  9
taatacgact cactata                                                      17


