                         SEQUENCE LISTING

<110>  Children's Hospitals and Clinics of Minnesota
 
<120>  VECTORS AND METHODS OF USE

<130>  00562.0003WOU1

<140>  New Filing
<141>  2018-10-02

<150>  US 62/567,631
<151>  2017-10-03

<150>  US 62/588,106
<151>  2017-11-17

<150>  US 62/640,787
<151>  2018-03-09

<160>  15    

<170>  PatentIn version 3.5

<210>  1
<211>  7696
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  synthetic sequence:   human fumarylacetoacetate hydrolase as 
       inserted into a lentiviral vector

<400>  1
acgcgccctg tagcggcgca ttaagcgcgg cgggtgtggt ggttacgcgc agcgtgaccg       60

ctacacttgc cagcgcccta gcgcccgctc ctttcgcttt cttcccttcc tttctcgcca      120

cgttcgccgg ctttccccgt caagctctaa atcgggggct ccctttaggg ttccgattta      180

gtgctttacg gcacctcgac cccaaaaaac ttgattaggg tgatggttca cgtagtgggc      240

catcgccctg atagacggtt tttcgccctt tgacgttgga gtccacgttc tttaatagtg      300

gactcttgtt ccaaactgga acaacactca accctatctc ggtctattct tttgatttat      360

aagggatttt gccgatttcg gcctattggt taaaaaatga gctgatttaa caaaaattta      420

acgcgaattt taacaaaata ttaacgctta caatttaggt ggcacttttc ggggaaatgt      480

gcgcggaacc cctatttgtt tatttttcta aatacattca aatatgtatc cgctcatgag      540

acaataaccc tgataaatgc ttcaataata gcacctagat caagagacag gatgaggatc      600

gtttcgcatg attgaacaag atggattgca cgcaggttct ccggccgctt gggtggagag      660

gctattcggc tatgactggg cacaacagac aatcggctgc tctgatgccg ccgtgttccg      720

gctgtcagcg caggggcgcc cggttctttt tgtcaagacc gacctgtccg gtgccctgaa      780

tgaactgcaa gacgaggcag cgcggctatc gtggctggcc acgacgggcg ttccttgcgc      840

agctgtgctc gacgttgtca ctgaagcggg aagggactgg ctgctattgg gcgaagtgcc      900

ggggcaggat ctcctgtcat ctcaccttgc tcctgccgag aaagtatcca tcatggctga      960

tgcaatgcgg cggctgcata cgcttgatcc ggctacctgc ccattcgacc accaagcgaa     1020

acatcgcatc gagcgagcac gtactcggat ggaagccggt cttgtcgatc aggatgatct     1080

ggacgaagag catcaggggc tcgcgccagc cgaactgttc gccaggctca aggcgagcat     1140

gcccgacggc gaggatctcg tcgtgaccca tggcgatgcc tgcttgccga atatcatggt     1200

ggaaaatggc cgcttttctg gattcatcga ctgtggccgg ctgggtgtgg cggaccgcta     1260

tcaggacata gcgttggcta cccgtgatat tgctgaagag cttggcggcg aatgggctga     1320

ccgcttcctc gtgctttacg gtatcgccgc tcccgattcg cagcgcatcg ccttctatcg     1380

ccttcttgac gagttcttct gaattattaa cgcttacaat ttcctgatgc ggtattttct     1440

ccttacgcat ctgtgcggta tttcacaccg catcaggtgg cacttttcgg ggaaatgtgc     1500

gcggaacccc tatttgttta tttttctaaa tacattcaaa tatgtatccg ctcatgacca     1560

aaatccctta acgtgagttt tcgttccact gagcgtcaga ccccgtagaa aagatcaaag     1620

gatcttcttg agatcctttt tttctgcgcg taatctgctg cttgcaaaca aaaaaaccac     1680

cgctaccagc ggtggtttgt ttgccggatc aagagctacc aactcttttt ccgaaggtaa     1740

ctggcttcag cagagcgcag ataccaaata ctgttcttct agtgtagccg tagttaggcc     1800

accacttcaa gaactctgta gcaccgccta catacctcgc tctgctaatc ctgttaccag     1860

tggctgctgc cagtggcgat aagtcgtgtc ttaccgggtt ggactcaaga cgatagttac     1920

cggataaggc gcagcggtcg ggctgaacgg gggggtcgtg cacacagccc agcttggagc     1980

gaacgaccta caccgaactg agatacctac agcgtgagct atgagaaagc gccacgcttc     2040

ccgaagggag aaaggcggac aggtatccgg taagcggcag ggtcggaaca ggagagcgca     2100

cgagggagct tccaggggga aacgcctggt atctttatag tcctgtcggg tttcgccacc     2160

tctgacttga gcgtcgattt ttgtgatgct cgtcaggggg gcggagccta tggaaaaacg     2220

ccagcaacgc ggccttttta cggttcctgg ccttttgctg gccttttgct cacatgttct     2280

ttcctgcgtt atcccctgat tctgtggata accgtattac cgcctttgag tgagctgata     2340

ccgctcgccg cagccgaacg accgagcgca gcgagtcagt gagcgaggaa gcggaagagc     2400

gcccaatacg caaaccgcct ctccccgcgc gttggccgat tcattaatgc agctggcacg     2460

acaggtttcc cgactggaaa gcgggcagtg agcgcaacgc aattaatgtg agttagctca     2520

ctcattaggc accccaggct ttacacttta tgcttccggc tcgtatgttg tgtggaattg     2580

tgagcggata acaatttcac acaggaaaca gctatgacca tgattacgcc aagcgcgcaa     2640

ttaaccctca ctaaagggaa caaaagctgg agctgcaagc ttggccattg catacgttgt     2700

atccatatca taatatgtac atttatattg gctcatgtcc aacattaccg ccatgttgac     2760

attgattatt gactagttat taatagtaat caattacggg gtcattagtt catagcccat     2820

atatggagtt ccgcgttaca taacttacgg taaatggccc gcctggctga ccgcccaacg     2880

acccccgccc attgacgtca ataatgacgt atgttcccat agtaacgcca atagggactt     2940

tccattgacg tcaatgggtg gagtatttac ggtaaactgc ccacttggca gtacatcaag     3000

tgtatcatat gccaagtacg ccccctattg acgtcaatga cggtaaatgg cccgcctggc     3060

attatgccca gtacatgacc ttatgggact ttcctacttg gcagtacatc tacgtattag     3120

tcatcgctat taccatggtg atgcggtttt ggcagtacat caatgggcgt ggatagcggt     3180

ttgactcacg gggatttcca agtctccacc ccattgacgt caatgggagt ttgttttggc     3240

accaaaatca acgggacttt ccaaaatgtc gtaacaactc cgccccattg acgcaaatgg     3300

gcggtaggcg tgtacggtgg gaggtctata taagcagagc tcgtttagtg aaccggggtc     3360

tctctggtta gaccagatct gagcctggga gctctctggc taactaggga acccactgct     3420

taagcctcaa taaagcttgc cttgagtgct tcaagtagtg tgtgcccgtc tgttgtgtga     3480

ctctggtaac tagagatccc tcagaccctt ttagtcagtg tggaaaatct ctagcagtgg     3540

cgcccgaaca gggacttgaa agcgaaaggg aaaccagagg agctctctcg acgcaggact     3600

cggcttgctg aagcgcgcac ggcaagaggc gaggggcggc gactggtgag tacgccaaaa     3660

attttgacta gcggaggcta gaaggagaga gatgggtgcg agagcgtcag tattaagcgg     3720

gggagaatta gatcgcgatg ggaaaaaatt cggttaaggc cagggggaaa gaaaaaatat     3780

aaattaaaac atatagtatg ggcaagcagg gagctagaac gattcgcagt taatcctggc     3840

ctgttagaaa catcagaagg ctgtagacaa atactgggac agctacaacc atcccttcag     3900

acaggatcag aagaacttag atcattatat aatacagtag caaccctcta ttgtgtgcat     3960

caaaggatag agataaaaga caccaaggaa gctttagaca agatagagga agagcaaaac     4020

aaaagtaaga ccaccgcaca gcaagcggcc gctgatcttc agacctggag gaggagatat     4080

gagggacaat tggagaagtg aattatataa atataaagta gtaaaaattg aaccattagg     4140

agtagcaccc accaaggcaa agagaagagt ggtgcagaga gaaaaaagag cagtgggaat     4200

aggagctttg ttccttgggt tcttgggagc agcaggaagc actatgggcg cagcgtcaat     4260

gacgctgacg gtacaggcca gacaattatt gtctggtata gtgcagcagc agaacaattt     4320

gctgagggct attgaggcgc aacagcatct gttgcaactc acagtctggg gcatcaagca     4380

gctccaggca agaatcctgg ctgtggaaag atacctaaag gatcaacagc tcctggggat     4440

ttggggttgc tctggaaaac tcatttgcac cactgctgtg ccttggaatg ctagttggag     4500

taataaatct ctggaacaga tttggaatca cacgacctgg atggagtggg acagagaaat     4560

taacaattac acaagcttaa tacactcctt aattgaagaa tcgcaaaacc agcaagaaaa     4620

gaatgaacaa gaattattgg aattagataa atgggcaagt ttgtggaatt ggtttaacat     4680

aacaaattgg ctgtggtata taaaattatt cataatgata gtaggaggct tggtaggttt     4740

aagaatagtt tttgctgtac tttctatagt gaatagagtt aggcagggat attcaccatt     4800

atcgtttcag acccacctcc caaccccgag gggacccgac aggcccgaag gaatagaaga     4860

agaaggtgga gagagagaca gagacagatc cattcgatta gtgaacggat ctcgacggta     4920

tcggttaact tttaaaagaa aaggggggat tggggggtac agtgcagggg aaagaatagt     4980

agacataata gcaacagaca tacaaactaa agaattacaa aaacaaatta caaaattcaa     5040

aattttatcg atagcgctac gcgtcctaaa atgggcaaac attgcaagca gcaaacagca     5100

aacacacagc cctccctgcc tgctgacctt ggagctgggg cagaggtcag agacctctct     5160

gggcccatgc cacctccaac atccactcga ccccttggaa tttcggtgga gaggagcaga     5220

ggttgtcctg gcgtggttta ggtagtgtga gagggcgcgc caatgactcc tttcggtaag     5280

tgcagtggaa gctgtacact gcccaggcaa agcgtccggg cagcgtaggc gggcgactca     5340

gatcccagcc agtggactta gcccctgttt gctcctccga taactggggt gaccttggtt     5400

aatattcacc agcagcctcc cccgttgccc ctctggatcc actgcttaaa tacggacgag     5460

gacagggccc tgtctcctca gcttcaggca ccaccactga cctgggacag tggctaagct     5520

tggtaccgag ctcggatccg cttagcatgt ccttcatccc ggtggccgag gattccgact     5580

tccccatcca caacctgccc tacggcgtct tctcgaccag aggcgaccca agaccgagga     5640

taggtgtggc cattggcgac cagatcctgg acctcagcat catcaagcac ctctttactg     5700

gtcctgtcct ctccaaacac caggatgtct tcaatcagcc tacactcaac agcttcatgg     5760

gcctgggtca ggctgcctgg aaggaggcga gagtgttctt gcagaacttg ctgtctgtga     5820

gccaagccag gctcagagat gacaccgaac ttcggaagtg tgcattcatc tcccaggctt     5880

ctgccacgat gcaccttcca gccaccatag gagactacac agacttctat tcctctcggc     5940

agcatgctac caacgtcgga atcatgttca gggacaagga gaatgcgttg atgccaaatt     6000

ggctgcactt accagtgggc taccatggcc gtgcctcctc tgtcgtggtg tctggcaccc     6060

caatccgaag gcccatggga cagatgaaac ctgatgactc taagcctccc gtatatggtg     6120

cctgcaagct cttggacatg gagctggaaa tggctttttt tgtaggccct ggaaacagat     6180

tgggagagcc gatccccatt tccaaggccc atgagcacat ttttggaatg gtccttatga     6240

acgactggag tgcacgagac attcagaagt gggagtatgt ccctctcggg ccattccttg     6300

ggaagagttt tgggaccact gtctctccgt gggtggtgcc catggatgct ctcatgccct     6360

ttgctgtgcc caacccgaag caggacccca ggcccctgcc gtatctgtgc catgacgagc     6420

cctacacatt tgacatcaac ctctctgtta acctgaaagg agaaggaatg agccaggcgg     6480

ctaccatatg caagtccaat tttaagtaca tgtactggac gatgctgcag cagctcactc     6540

accactctgt caacggctgc aacctgcggc cgggggacct cctggcttct gggaccatca     6600

gcgggccgga gccagaaaac ttcggctcca tgttggaact gtcgtggaag ggaacgaagc     6660

ccatagacct ggggaatggt cagaccagga agtttctgct ggacggggat gaagtcatca     6720

taacagggta ctgccagggg gatggttacc gcatcggctt tggccagtgt gctggaaaag     6780

tgctgcctgc tctcctgcca tcatgagaat tcgagctcgg tacctttaag accaatgact     6840

tacaaggcag ctgtagatct tagccacttt ttaaaagaaa aggggggact ggaagggcta     6900

attcactccc aacgaagaca agatctgctt tttgcttgta ctgggtctct ctggttagac     6960

cagatctgag cctgggagct ctctggctaa ctagggaacc cactgcttaa gcctcaataa     7020

agcttgcctt gagtgcttca agtagtgtgt gcccgtctgt tgtgtgactc tggtaactag     7080

agatccctca gaccctttta gtcagtgtgg aaaatctcta gcagtagtag ttcatgtcat     7140

cttattattc agtattataa cttgcaaaga aatgaatatc agagagtgag aggaacttgt     7200

ttattgcagc ttataatggt tacaaataaa gcaatagcat cacaaatttc acaaataaag     7260

catttttttc actgcattct agttgtggtt tgtccaaact catcaatgta tcttatcatg     7320

tctggctcta gctatcccgc ccctaactcc gcccatcccg cccctaactc cgcccagttc     7380

cgcccattct ccgccccatg gctgactaat tttttttatt tatgcagagg ccgaggccgc     7440

ctcggcctct gagctattcc agaagtagtg aggaggcttt tttggaggcc tagggacgta     7500

cccaattcgc cctatagtga gtcgtattac gcgcgctcac tggccgtcgt tttacaacgt     7560

cgtgactggg aaaaccctgg cgttacccaa cttaatcgcc ttgcagcaca tccccctttc     7620

gccagctggc gtaatagcga agaggcccgc accgatcgcc cttcccaaca gttgcgcagc     7680

ctgaatggcg aatggg                                                     7696


<210>  2
<211>  29
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Forward primer

<400>  2
tgtgtgactc tggtaactag agatccctc                                         29


<210>  3
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Reverse primer

<400>  3
agtagcaccc accaaggcaa                                                   20


<210>  4
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Forward primer

<400>  4
ggatgtcttc aatcagccta cac                                               23


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

<220>
<223>  Reverse primer

<400>  5
tgaaacctga tgactctaag cc                                                22


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

<220>
<223>  Forward primer

<400>  6
tgttggaact gtcgtggaag                                                   20


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

<220>
<223>  Reverse primer

<400>  7
taactaggga acccactgct                                                   20


<210>  8
<211>  1260
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleic acid sequence for human fumarylacetoacetate hydrolase

<400>  8
atgtccttca tcccggtggc cgaggattcc gacttcccca tccacaacct gccctacggc       60

gtcttctcga ccagaggcga cccaagaccg aggataggtg tggccattgg cgaccagatc      120

ctggacctca gcatcatcaa gcacctcttt actggtcctg tcctctccaa acaccaggat      180

gtcttcaatc agcctacact caacagcttc atgggcctgg gtcaggctgc ctggaaggag      240

gcgagagtgt tcttgcagaa cttgctgtct gtgagccaag ccaggctcag agatgacacc      300

gaacttcgga agtgtgcatt catctcccag gcttctgcca cgatgcacct tccagccacc      360

ataggagact acacagactt ctattcctct cggcagcatg ctaccaacgt cggaatcatg      420

ttcagggaca aggagaatgc gttgatgcca aattggctgc acttaccagt gggctaccat      480

ggccgtgcct cctctgtcgt ggtgtctggc accccaatcc gaaggcccat gggacagatg      540

aaacctgatg actctaagcc tcccgtatat ggtgcctgca agctcttgga catggagctg      600

gaaatggctt tttttgtagg ccctggaaac agattgggag agccgatccc catttccaag      660

gcccatgagc acatttttgg aatggtcctt atgaacgact ggagtgcacg agacattcag      720

aagtgggagt atgtccctct cgggccattc cttgggaaga gttttgggac cactgtctct      780

ccgtgggtgg tgcccatgga tgctctcatg ccctttgctg tgcccaaccc gaagcaggac      840

cccaggcccc tgccgtatct gtgccatgac gagccctaca catttgacat caacctctct      900

gttaacctga aaggagaagg aatgagccag gcggctacca tatgcaagtc caattttaag      960

tacatgtact ggacgatgct gcagcagctc actcaccact ctgtcaacgg ctgcaacctg     1020

cggccggggg acctcctggc ttctgggacc atcagcgggc cggagccaga aaacttcggc     1080

tccatgttgg aactgtcgtg gaagggaacg aagcccatag acctggggaa tggtcagacc     1140

aggaagtttc tgctggacgg ggatgaagtc atcataacag ggtactgcca gggggatggt     1200

taccgcatcg gctttggcca gtgtgctgga aaagtgctgc ctgctctcct gccatcatga     1260


<210>  9
<211>  419
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Protein sequence for human fumarylacetoacetate hydrolase

<400>  9

Met Ser Phe Ile Pro Val Ala Glu Asp Ser Asp Phe Pro Ile His Asn 
1               5                   10                  15      


Leu Pro Tyr Gly Val Phe Ser Thr Arg Gly Asp Pro Arg Pro Arg Ile 
            20                  25                  30          


Gly Val Ala Ile Gly Asp Gln Ile Leu Asp Leu Ser Ile Ile Lys His 
        35                  40                  45              


Leu Phe Thr Gly Pro Val Leu Ser Lys His Gln Asp Val Phe Asn Gln 
    50                  55                  60                  


Pro Thr Leu Asn Ser Phe Met Gly Leu Gly Gln Ala Ala Trp Lys Glu 
65                  70                  75                  80  


Ala Arg Val Phe Leu Gln Asn Leu Leu Ser Val Ser Gln Ala Arg Leu 
                85                  90                  95      


Arg Asp Asp Thr Glu Leu Arg Lys Cys Ala Phe Ile Ser Gln Ala Ser 
            100                 105                 110         


Ala Thr Met His Leu Pro Ala Thr Ile Gly Asp Tyr Thr Asp Phe Tyr 
        115                 120                 125             


Ser Ser Arg Gln His Ala Thr Asn Val Gly Ile Met Phe Arg Asp Lys 
    130                 135                 140                 


Glu Asn Ala Leu Met Pro Asn Trp Leu His Leu Pro Val Gly Tyr His 
145                 150                 155                 160 


Gly Arg Ala Ser Ser Val Val Val Ser Gly Thr Pro Ile Arg Arg Pro 
                165                 170                 175     


Met Gly Gln Met Lys Pro Asp Asp Ser Lys Pro Pro Val Tyr Gly Ala 
            180                 185                 190         


Cys Lys Leu Leu Asp Met Glu Leu Glu Met Ala Phe Phe Val Gly Pro 
        195                 200                 205             


Gly Asn Arg Leu Gly Glu Pro Ile Pro Ile Ser Lys Ala His Glu His 
    210                 215                 220                 


Ile Phe Gly Met Val Leu Met Asn Asp Trp Ser Ala Arg Asp Ile Gln 
225                 230                 235                 240 


Lys Trp Glu Tyr Val Pro Leu Gly Pro Phe Leu Gly Lys Ser Phe Gly 
                245                 250                 255     


Thr Thr Val Ser Pro Trp Val Val Pro Met Asp Ala Leu Met Pro Phe 
            260                 265                 270         


Ala Val Pro Asn Pro Lys Gln Asp Pro Arg Pro Leu Pro Tyr Leu Cys 
        275                 280                 285             


His Asp Glu Pro Tyr Thr Phe Asp Ile Asn Leu Ser Val Asn Leu Lys 
    290                 295                 300                 


Gly Glu Gly Met Ser Gln Ala Ala Thr Ile Cys Lys Ser Asn Phe Lys 
305                 310                 315                 320 


Tyr Met Tyr Trp Thr Met Leu Gln Gln Leu Thr His His Ser Val Asn 
                325                 330                 335     


Gly Cys Asn Leu Arg Pro Gly Asp Leu Leu Ala Ser Gly Thr Ile Ser 
            340                 345                 350         


Gly Pro Glu Pro Glu Asn Phe Gly Ser Met Leu Glu Leu Ser Trp Lys 
        355                 360                 365             


Gly Thr Lys Pro Ile Asp Leu Gly Asn Gly Gln Thr Arg Lys Phe Leu 
    370                 375                 380                 


Leu Asp Gly Asp Glu Val Ile Ile Thr Gly Tyr Cys Gln Gly Asp Gly 
385                 390                 395                 400 


Tyr Arg Ile Gly Phe Gly Gln Cys Ala Gly Lys Val Leu Pro Ala Leu 
                405                 410                 415     


Leu Pro Ser 
            


<210>  10
<211>  28
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic sequences: primers for identifying sites of integration
       (FIG.1)

<400>  10
atgtgactct ggtaactaga gatccctc                                          28


<210>  11
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic sequences: primers for identifying sites of integration
       (FIG.1)

<400>  11
gagatccctc agaccctttt agtcag                                            26


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

<220>
<223>  MseI linker

<400>  12
tagtccctta agcggag                                                      17


<210>  13
<211>  37
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  MseI linker

<400>  13
gtaatacgac tcactatagg gcctccgctt aagggac                                37


<210>  14
<211>  83
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Linker primer (reverse primer)

<400>  14
caagcagaag acggcatacg agatcggtct cggcattcct gctgaaccgc tcttccgatc       60

tgtaatacga ctcactatag ggc                                               83


<210>  15
<211>  90
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nested LSP

<400>  15
aatgatacgg cgaccaccga gatctacact ctttccctac acgacgctct tccgatctcg       60

atgtgagatc cctcagaccc ttttagtcag                                        90


