                               SEQUENCE LISTING

<110> DEROSA, FRANK
      HEARTLEIN, MICHAEL
      DIAS, ANUSHA
 
<120> MRNA THERAPY FOR PHENYLKETONURIA

<130> 2006685-0692

<140>
<141>

<150> 61/894,303
<151> 2013-10-22

<160> 12    

<170> PatentIn version 3.5

<210> 1
<211> 2680
<212> RNA
<213> Homo sapiens

<400> 1
cagcuggggg uaaggggggc ggauuauuca uauaauuguu auaccagacg gucgcaggcu       60

uaguccaauu gcagagaacu cgcuucccag gcuucugaga gucccggaag ugccuaaacc      120

ugucuaaucg acggggcuug gguggcccgu cgcucccugg cuucuucccu uuacccaggg      180

cgggcagcga aguggugccu ccugcguccc ccacacccuc ccucagcccc uccccuccgg      240

cccguccugg gcaggugacc uggagcaucc ggcaggcugc ccuggccucc ugcgucagga      300

caagcccacg aggggcguua cugugcggag augcaccacg caagagacac ccuuuguaac      360

ucucuucucc ucccuagugc gagguuaaaa ccuucagccc cacgugcugu uugcaaaccu      420

gccuguaccu gaggcccuaa aaagccagag accucacucc cggggagcca gcauguccac      480

ugcgguccug gaaaacccag gcuugggcag gaaacucucu gacuuuggac aggaaacaag      540

cuauauugaa gacaacugca aucaaaaugg ugccauauca cugaucuucu cacucaaaga      600

agaaguuggu gcauuggcca aaguauugcg cuuauuugag gagaaugaug uaaaccugac      660

ccacauugaa ucuagaccuu cucguuuaaa gaaagaugag uaugaauuuu ucacccauuu      720

ggauaaacgu agccugccug cucugacaaa caucaucaag aucuugaggc augacauugg      780

ugccacuguc caugagcuuu cacgagauaa gaagaaagac acagugcccu gguucccaag      840

aaccauucaa gagcuggaca gauuugccaa ucagauucuc agcuauggag cggaacugga      900

ugcugaccac ccugguuuua aagauccugu guaccgugca agacggaagc aguuugcuga      960

cauugccuac aacuaccgcc augggcagcc caucccucga guggaauaca uggaggaaga     1020

aaagaaaaca uggggcacag uguucaagac ucugaagucc uuguauaaaa cccaugcuug     1080

cuaugaguac aaucacauuu uuccacuucu ugaaaaguac uguggcuucc augaagauaa     1140

cauuccccag cuggaagacg uuucucaauu ccugcagacu ugcacugguu uccgccuccg     1200

accuguggcu ggccugcuuu ccucucggga uuucuugggu ggccuggccu uccgagucuu     1260

ccacugcaca caguacauca gacauggauc caagcccaug uauacccccg aaccugacau     1320

cugccaugag cuguugggac augugcccuu guuuucagau cgcagcuuug cccaguuuuc     1380

ccaggaaauu ggccuugccu cucugggugc accugaugaa uacauugaaa agcucgccac     1440

aauuuacugg uuuacugugg aguuugggcu cugcaaacaa ggagacucca uaaaggcaua     1500

uggugcuggg cuccugucau ccuuugguga auuacaguac ugcuuaucag agaagccaaa     1560

gcuucucccc cuggagcugg agaagacagc cauccaaaau uacacuguca cggaguucca     1620

gccccuguau uacguggcag agaguuuuaa ugaugccaag gagaaaguaa ggaacuuugc     1680

ugccacaaua ccucggcccu ucucaguucg cuacgaccca uacacccaaa ggauugaggu     1740

cuuggacaau acccagcagc uuaagauuuu ggcugauucc auuaacagug aaauuggaau     1800

ccuuugcagu gcccuccaga aaauaaagua aagccaugga cagaaugugg ucugucagcu     1860

gugaaucugu ugauggagau ccaacuauuu cuuucaucag aaaaaguccg aaaagcaaac     1920

cuuaauuuga aauaacagcc uuaaauccuu uacaagaugg agaaacaaca aauaagucaa     1980

aauaaucuga aaugacagga uaugaguaca uacucaagag cauaauggua aaucuuuugg     2040

ggucaucuuu gauuuagaga ugauaauccc auacucucaa uugaguuaaa ucaguaaucu     2100

gucgcauuuc aucaagauua auuaaaauuu gggaccugcu ucauucaagc uucauauaug     2160

cuuugcagag aacucauaaa ggagcauaua aggcuaaaug uaaaacacaa gacugucauu     2220

agaauugaau uauugggcuu aauauaaauc guaaccuaug aaguuuauuu ucuauuuuag     2280

uuaacuauga uuccaauuac uacuuuguua uuguaccuaa guaaauuuuc uuuaggucag     2340

aagcccauua aaauaguuac aagcauugaa cuucuuuagu auuauauuaa uauaaaaaca     2400

uuuuuguaug uuuuauugua aucauaaaua cugcuguaua agguaauaaa acucugcacc     2460

uaauccccau aacuuccagu aucauuuucc aauuaauuau caagucuguu uugggaaaca     2520

cuuugaggac auuuaugaug cagcagaugu ugacuaaagg cuugguuggu agauauucag     2580

gaaauguuca cugaauaaau aaguaaauac auuauugaaa agcaaaucug uauaaaugug     2640

aaauuuuuau uuguauuagu aauaaaacau uaguaguuua                           2680


<210> 2
<211> 452
<212> PRT
<213> Homo sapiens

<400> 2
Met Ser Thr Ala Val Leu Glu Asn Pro Gly Leu Gly Arg Lys Leu Ser 
1               5                   10                  15      


Asp Phe Gly Gln Glu Thr Ser Tyr Ile Glu Asp Asn Cys Asn Gln Asn 
            20                  25                  30          


Gly Ala Ile Ser Leu Ile Phe Ser Leu Lys Glu Glu Val Gly Ala Leu 
        35                  40                  45              


Ala Lys Val Leu Arg Leu Phe Glu Glu Asn Asp Val Asn Leu Thr His 
    50                  55                  60                  


Ile Glu Ser Arg Pro Ser Arg Leu Lys Lys Asp Glu Tyr Glu Phe Phe 
65                  70                  75                  80  


Thr His Leu Asp Lys Arg Ser Leu Pro Ala Leu Thr Asn Ile Ile Lys 
                85                  90                  95      


Ile Leu Arg His Asp Ile Gly Ala Thr Val His Glu Leu Ser Arg Asp 
            100                 105                 110         


Lys Lys Lys Asp Thr Val Pro Trp Phe Pro Arg Thr Ile Gln Glu Leu 
        115                 120                 125             


Asp Arg Phe Ala Asn Gln Ile Leu Ser Tyr Gly Ala Glu Leu Asp Ala 
    130                 135                 140                 


Asp His Pro Gly Phe Lys Asp Pro Val Tyr Arg Ala Arg Arg Lys Gln 
145                 150                 155                 160 


Phe Ala Asp Ile Ala Tyr Asn Tyr Arg His Gly Gln Pro Ile Pro Arg 
                165                 170                 175     


Val Glu Tyr Met Glu Glu Glu Lys Lys Thr Trp Gly Thr Val Phe Lys 
            180                 185                 190         


Thr Leu Lys Ser Leu Tyr Lys Thr His Ala Cys Tyr Glu Tyr Asn His 
        195                 200                 205             


Ile Phe Pro Leu Leu Glu Lys Tyr Cys Gly Phe His Glu Asp Asn Ile 
    210                 215                 220                 


Pro Gln Leu Glu Asp Val Ser Gln Phe Leu Gln Thr Cys Thr Gly Phe 
225                 230                 235                 240 


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


Gly Leu Ala Phe Arg Val Phe His Cys Thr Gln Tyr Ile Arg His Gly 
            260                 265                 270         


Ser Lys Pro Met Tyr Thr Pro Glu Pro Asp Ile Cys His Glu Leu Leu 
        275                 280                 285             


Gly His Val Pro Leu Phe Ser Asp Arg Ser Phe Ala Gln Phe Ser Gln 
    290                 295                 300                 


Glu Ile Gly Leu Ala Ser Leu Gly Ala Pro Asp Glu Tyr Ile Glu Lys 
305                 310                 315                 320 


Leu Ala Thr Ile Tyr Trp Phe Thr Val Glu Phe Gly Leu Cys Lys Gln 
                325                 330                 335     


Gly Asp Ser Ile Lys Ala Tyr Gly Ala Gly Leu Leu Ser Ser Phe Gly 
            340                 345                 350         


Glu Leu Gln Tyr Cys Leu Ser Glu Lys Pro Lys Leu Leu Pro Leu Glu 
        355                 360                 365             


Leu Glu Lys Thr Ala Ile Gln Asn Tyr Thr Val Thr Glu Phe Gln Pro 
    370                 375                 380                 


Leu Tyr Tyr Val Ala Glu Ser Phe Asn Asp Ala Lys Glu Lys Val Arg 
385                 390                 395                 400 


Asn Phe Ala Ala Thr Ile Pro Arg Pro Phe Ser Val Arg Tyr Asp Pro 
                405                 410                 415     


Tyr Thr Gln Arg Ile Glu Val Leu Asp Asn Thr Gln Gln Leu Lys Ile 
            420                 425                 430         


Leu Ala Asp Ser Ile Asn Ser Glu Ile Gly Ile Leu Cys Ser Ala Leu 
        435                 440                 445             


Gln Lys Ile Lys 
    450     


<210> 3
<211> 1359
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 3
augagcaccg ccgugcugga gaaccccggc cugggccgca agcugagcga cuucggccag       60

gagaccagcu acaucgagga caacugcaac cagaacggcg ccaucagccu gaucuucagc      120

cugaaggagg aggugggcgc ccuggccaag gugcugcgcc uguucgagga gaacgacgug      180

aaccugaccc acaucgagag ccgccccagc cgccugaaga aggacgagua cgaguucuuc      240

acccaccugg acaagcgcag ccugcccgcc cugaccaaca ucaucaagau ccugcgccac      300

gacaucggcg ccaccgugca cgagcugagc cgcgacaaga agaaggacac cgugcccugg      360

uucccccgca ccauccagga gcuggaccgc uucgccaacc agauccugag cuacggcgcc      420

gagcuggacg ccgaccaccc cggcuucaag gaccccgugu accgcgcccg ccgcaagcag      480

uucgccgaca ucgccuacaa cuaccgccac ggccagccca ucccccgcgu ggaguacaug      540

gaggaggaga agaagaccug gggcaccgug uucaagaccc ugaagagccu guacaagacc      600

cacgccugcu acgaguacaa ccacaucuuc ccccugcugg agaaguacug cggcuuccac      660

gaggacaaca ucccccagcu ggaggacgug agccaguucc ugcagaccug caccggcuuc      720

cgccugcgcc ccguggccgg ccugcugagc agccgcgacu uccugggcgg ccuggccuuc      780

cgcguguucc acugcaccca guacauccgc cacggcagca agcccaugua cacccccgag      840

cccgacaucu gccacgagcu gcugggccac gugccccugu ucagcgaccg cagcuucgcc      900

caguucagcc aggagaucgg ccuggccagc cugggcgccc ccgacgagua caucgagaag      960

cuggccacca ucuacugguu caccguggag uucggccugu gcaagcaggg cgacagcauc     1020

aaggccuacg gcgccggccu gcugagcagc uucggcgagc ugcaguacug ccugagcgag     1080

aagcccaagc ugcugccccu ggagcuggag aagaccgcca uccagaacua caccgugacc     1140

gaguuccagc cccuguacua cguggccgag agcuucaacg acgccaagga gaaggugcgc     1200

aacuucgccg ccaccauccc ccgccccuuc agcgugcgcu acgaccccua cacccagcgc     1260

aucgaggugc uggacaacac ccagcagcug aagauccugg ccgacagcau caacagcgag     1320

aucggcaucc ugugcagcgc ccugcagaag aucaaguaa                            1359


<210> 4
<211> 140
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 4
ggacagaucg ccuggagacg ccauccacgc uguuuugacc uccauagaag acaccgggac       60

cgauccagcc uccgcggccg ggaacggugc auuggaacgc ggauuccccg ugccaagagu      120

gacucaccgu ccuugacacg                                                  140


<210> 5
<211> 105
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 5
ggguggcauc ccugugaccc cuccccagug ccucuccugg cccuggaagu ugccacucca       60

gugcccacca gccuuguccu aauaaaauua aguugcauca aagcu                      105


<210> 6
<211> 105
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 6
cggguggcau cccugugacc ccuccccagu gccucuccug gcccuggaag uugccacucc       60

agugcccacc agccuugucc uaauaaaauu aaguugcauc aagcu                      105


<210> 7
<211> 1604
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 7
ggacagaucg ccuggagacg ccauccacgc uguuuugacc uccauagaag acaccgggac       60

cgauccagcc uccgcggccg ggaacggugc auuggaacgc ggauuccccg ugccaagagu      120

gacucaccgu ccuugacacg augagcaccg ccgugcugga gaaccccggc cugggccgca      180

agcugagcga cuucggccag gagaccagcu acaucgagga caacugcaac cagaacggcg      240

ccaucagccu gaucuucagc cugaaggagg aggugggcgc ccuggccaag gugcugcgcc      300

uguucgagga gaacgacgug aaccugaccc acaucgagag ccgccccagc cgccugaaga      360

aggacgagua cgaguucuuc acccaccugg acaagcgcag ccugcccgcc cugaccaaca      420

ucaucaagau ccugcgccac gacaucggcg ccaccgugca cgagcugagc cgcgacaaga      480

agaaggacac cgugcccugg uucccccgca ccauccagga gcuggaccgc uucgccaacc      540

agauccugag cuacggcgcc gagcuggacg ccgaccaccc cggcuucaag gaccccgugu      600

accgcgcccg ccgcaagcag uucgccgaca ucgccuacaa cuaccgccac ggccagccca      660

ucccccgcgu ggaguacaug gaggaggaga agaagaccug gggcaccgug uucaagaccc      720

ugaagagccu guacaagacc cacgccugcu acgaguacaa ccacaucuuc ccccugcugg      780

agaaguacug cggcuuccac gaggacaaca ucccccagcu ggaggacgug agccaguucc      840

ugcagaccug caccggcuuc cgccugcgcc ccguggccgg ccugcugagc agccgcgacu      900

uccugggcgg ccuggccuuc cgcguguucc acugcaccca guacauccgc cacggcagca      960

agcccaugua cacccccgag cccgacaucu gccacgagcu gcugggccac gugccccugu     1020

ucagcgaccg cagcuucgcc caguucagcc aggagaucgg ccuggccagc cugggcgccc     1080

ccgacgagua caucgagaag cuggccacca ucuacugguu caccguggag uucggccugu     1140

gcaagcaggg cgacagcauc aaggccuacg gcgccggccu gcugagcagc uucggcgagc     1200

ugcaguacug ccugagcgag aagcccaagc ugcugccccu ggagcuggag aagaccgcca     1260

uccagaacua caccgugacc gaguuccagc cccuguacua cguggccgag agcuucaacg     1320

acgccaagga gaaggugcgc aacuucgccg ccaccauccc ccgccccuuc agcgugcgcu     1380

acgaccccua cacccagcgc aucgaggugc uggacaacac ccagcagcug aagauccugg     1440

ccgacagcau caacagcgag aucggcaucc ugugcagcgc ccugcagaag aucaaguaag     1500

gguggcaucc cugugacccc uccccagugc cucuccuggc ccuggaaguu gccacuccag     1560

ugcccaccag ccuuguccua auaaaauuaa guugcaucaa agcu                      1604


<210> 8
<211> 1604
<212> RNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 8
ggacagaucg ccuggagacg ccauccacgc uguuuugacc uccauagaag acaccgggac       60

cgauccagcc uccgcggccg ggaacggugc auuggaacgc ggauuccccg ugccaagagu      120

gacucaccgu ccuugacacg augagcaccg ccgugcugga gaaccccggc cugggccgca      180

agcugagcga cuucggccag gagaccagcu acaucgagga caacugcaac cagaacggcg      240

ccaucagccu gaucuucagc cugaaggagg aggugggcgc ccuggccaag gugcugcgcc      300

uguucgagga gaacgacgug aaccugaccc acaucgagag ccgccccagc cgccugaaga      360

aggacgagua cgaguucuuc acccaccugg acaagcgcag ccugcccgcc cugaccaaca      420

ucaucaagau ccugcgccac gacaucggcg ccaccgugca cgagcugagc cgcgacaaga      480

agaaggacac cgugcccugg uucccccgca ccauccagga gcuggaccgc uucgccaacc      540

agauccugag cuacggcgcc gagcuggacg ccgaccaccc cggcuucaag gaccccgugu      600

accgcgcccg ccgcaagcag uucgccgaca ucgccuacaa cuaccgccac ggccagccca      660

ucccccgcgu ggaguacaug gaggaggaga agaagaccug gggcaccgug uucaagaccc      720

ugaagagccu guacaagacc cacgccugcu acgaguacaa ccacaucuuc ccccugcugg      780

agaaguacug cggcuuccac gaggacaaca ucccccagcu ggaggacgug agccaguucc      840

ugcagaccug caccggcuuc cgccugcgcc ccguggccgg ccugcugagc agccgcgacu      900

uccugggcgg ccuggccuuc cgcguguucc acugcaccca guacauccgc cacggcagca      960

agcccaugua cacccccgag cccgacaucu gccacgagcu gcugggccac gugccccugu     1020

ucagcgaccg cagcuucgcc caguucagcc aggagaucgg ccuggccagc cugggcgccc     1080

ccgacgagua caucgagaag cuggccacca ucuacugguu caccguggag uucggccugu     1140

gcaagcaggg cgacagcauc aaggccuacg gcgccggccu gcugagcagc uucggcgagc     1200

ugcaguacug ccugagcgag aagcccaagc ugcugccccu ggagcuggag aagaccgcca     1260

uccagaacua caccgugacc gaguuccagc cccuguacua cguggccgag agcuucaacg     1320

acgccaagga gaaggugcgc aacuucgccg ccaccauccc ccgccccuuc agcgugcgcu     1380

acgaccccua cacccagcgc aucgaggugc uggacaacac ccagcagcug aagauccugg     1440

ccgacagcau caacagcgag aucggcaucc ugugcagcgc ccugcagaag aucaaguaac     1500

ggguggcauc ccugugaccc cuccccagug ccucuccugg cccuggaagu ugccacucca     1560

gugcccacca gccuuguccu aauaaaauua aguugcauca agcu                      1604


<210> 9
<211> 300
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide


<220>
<221> misc_feature
<222> (1)..(300)
<223> This sequence may encompass 10-300 nucleotides

<220> 
<223> See specification as filed for detailed description of
      substitutions and preferred embodiments

<400> 9
aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa       60

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      120

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      180

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      240

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      300


<210> 10
<211> 200
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide


<220>
<221> misc_feature
<222> (1)..(200)
<223> This sequence may encompass 10-200 nucleotides

<220> 
<223> See specification as filed for detailed description of
      substitutions and preferred embodiments

<400> 10
cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc       60

cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc      120

cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc cccccccccc      180

cccccccccc cccccccccc                                                  200


<210> 11
<211> 500
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide


<220> 
<221> misc_feature
<222> (1)..(500)
<223> This sequence may encompass 10-500 nucleotides

<220> 
<223> See specification as filed for detailed description of
      substitutions and preferred embodiments

<400> 11
aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa       60

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      120

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      180

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      240

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      300

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      360

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      420

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      480

aaaaaaaaaa aaaaaaaaaa                                                  500


<210> 12
<211> 250
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide


<220> 
<223> See specification as filed for detailed description of
      substitutions and preferred embodiments

<400> 12
aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa       60

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      120

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      180

aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa aaaaaaaaaa      240

aaaaaaaaaa                                                             250


