                         SEQUENCE LISTING

<110>  bluebird bio, Inc.
       Goss, Kendrick
       Parsons, Geoffrey
       Giniatullina, Asiya
 
<120>  GENE THERAPY OF NEURONAL CEROID LIPOFUSCINOSES

<130>  BLBD-070/02WO

<150>  US 62/457,498
<151>  2017-02-10

<150>  US 62/349,505
<151>  2016-06-13

<160>  20    

<170>  PatentIn version 3.5

<210>  1
<211>  7566
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleic acid sequence of a lentiviral vector encoding TTP1 
       (pMND-CLN2)

<400>  1
tcgcgcgttt cggtgatgac ggtgaaaacc tctgacacat gcagctcccg gagacggtca       60

cagcttgtct gtaagcggat gccgggagca gacaagcccg tcagggcgcg tcagcgggtg      120

ttggcgggtg tcggggctgg cttaactatg cggcatcaga gcagattgta ctgagagtgc      180

accatcatat gccagcctat ggtgacattg attattgact agttattaat agtaatcaat      240

tacggggtca ttagttcata gcccatatat ggagttccgc gttacataac ttacggtaaa      300

tggcccgcct ggctgaccgc ccaacgaccc ccgcccattg acgtcaataa tgacgtatgt      360

tcccatagta acgccaatag ggactttcca ttgacgtcaa tgggtggagt atttacggta      420

aactgcccac ttggcagtac atcaagtgta tcatatgcca agtacgcccc ctattgacgt      480

caatgacggt aaatggcccg cctggcatta tgcccagtac atgaccttat gggactttcc      540

tacttggcag tacatctacg tattagtcat cgctattacc atggtgatgc ggttttggca      600

gtacatcaat gggcgtggat agcggtttga ctcacgggga tttccaagtc tccaccccat      660

tgacgtcaat gggagtttgt tttggcacca aaatcaacgg gactttccaa aatgtcgtaa      720

caactccgcc ccattgacgc aaatgggcgg taggcgtgta cggtgggagg tctatataag      780

cagagctcgt ttagtgaacc gggtctctct ggttagacca gatctgagcc tgggagctct      840

ctggctaact agggaaccca ctgcttaagc ctcaataaag cttgccttga gtgctcaaag      900

tagtgtgtgc ccgtctgttg tgtgactctg gtaactagag atccctcaga cccttttagt      960

cagtgtggaa aatctctagc agtggcgccc gaacagggac ttgaaagcga aagtaaagcc     1020

agaggagatc tctcgacgca ggactcggct tgctgaagcg cgcacggcaa gaggcgaggg     1080

gcggcgactg gtgagtacgc caaaaatttt gactagcgga ggctagaagg agagagtagg     1140

gtgcgagagc gtcggtatta agcgggggag aattagataa atgggaaaaa attcggttaa     1200

ggccaggggg aaagaaacaa tataaactaa aacatatagt tagggcaagc agggagctag     1260

aacgattcgc agttaatcct ggccttttag agacatcaga aggctgtaga caaatactgg     1320

gacagctaca accatccctt cagacaggat cagaagaact tagatcatta tataatacaa     1380

tagcagtcct ctattgtgtg catcaaagga tagatgtaaa agacaccaag gaagccttag     1440

ataagataga ggaagagcaa aacaaaagta agaaaaaggc acagcaagca gcagctgaca     1500

caggaaacaa cagccaggtc agccaaaatt accctatagt gcagaacctc caggggcaaa     1560

tggtacatca ggccatatca cctagaactt taaattaaga cagcagtaca aatggcagta     1620

ttcatccaca attttaaaag aaaagggggg attggggggt acagtgcagg ggaaagaata     1680

gtagacataa tagcaacaga catacaaact aaagaattac aaaaacaaat tacaaaaatt     1740

caaaattttc gggtttatta cagggacagc agagatccag tttggaaagg accagcaaag     1800

ctcctctgga aaggtgaagg ggcagtagta atacaagata atagtgacat aaaagtagtg     1860

ccaagaagaa aagcaaagat catcagggat tatggaaaac agatggcagg tgatgattgt     1920

gtggcaagta gacaggatga ggattaacac atggaaaaga ttagtaaaac accatagctc     1980

tagagcgatc ccgatcttca gacctggagg aggagatatg agggacaatt ggagaagtga     2040

attatataaa tataaagtag taaaaattga accattagga gtagcaccca ccaaggcaaa     2100

gagaagagtg gtgcagagag aaaaaagagc agtgggaata ggagctttgt tccttgggtt     2160

cttgggagca gcaggaagca ctatgggcgc agcgtcaatg acgctgacgg tacaggccag     2220

acaattattg tctggtatag tgcagcagca gaacaatttg ctgagggcta ttgaggcgca     2280

acagcatctg ttgcaactca cagtctgggg catcaagcag ctccaggcaa gaatcctggc     2340

tgtggaaaga tacctaaagg atcaacagct cctggggatt tggggttgct ctggaaaact     2400

catttgcacc actgctgtgc cttggaatgc tagttggagt aataaatctc tggaacagat     2460

ttggaatcac acgacctgga tggagtggga cagagaaatt aacaattaca caagcttggt     2520

aggtttaaga atagtttttg ctgtactttc tatagtgaat agagttaggc agggatattc     2580

accattatcg tttcagaccc acctcccaac cccgagggga cccgacaggc ccgaaggaat     2640

agaagaagaa ggtggagaga gagacagaga cagatccatt cgattagtga acggatccat     2700

ctcgacggaa tgaaagaccc cacctgtagg tttggcaagc taggatcaag gttaggaaca     2760

gagagacagc agaatatggg ccaaacagga tatctgtggt aagcagttcc tgccccggct     2820

cagggccaag aacagttgga acagcagaat atgggccaaa caggatatct gtggtaagca     2880

gttcctgccc cggctcaggg ccaagaacag atggtcccca gatgcggtcc cgccctcagc     2940

agtttctaga gaaccatcag atgtttccag ggtgccccaa ggacctgaaa tgaccctgtg     3000

ccttatttga actaaccaat cagttcgctt ctcgcttctg ttcgcgcgct tctgctcccc     3060

gagctcaata aaagagccca caacccctca ctcggcgcga ttcacctgac gcgtctacgc     3120

caccatgggg ttgcaggctt gcctgctggg acttttcgct ctgatcctga gcggaaagtg     3180

ctcctactca cctgagccag accagaggag aactctgccc cccggatggg tgtccctggg     3240

aagggccgac cctgaggagg aactctcgct caccttcgca ctgcggcagc agaacgtgga     3300

aagactgtcc gaactggtgc aggcagtgtc cgacccctcg agcccgcagt acggaaagta     3360

cctgaccctc gaaaacgtgg cagacttggt ccggccctcc cctctcaccc tgcacaccgt     3420

gcaaaaatgg ctgctggccg ccggagctca gaagtgccat tccgtgatta cacaggactt     3480

ccttacctgt tggcttagca tccgccaagc ggagctgctg ctgcctggtg ccgagttcca     3540

ccactacgtg ggcgggccaa ctgaaaccca cgtcgtgcgc agcccgcacc cgtatcagct     3600

gccccaggcg ctggctcctc atgtggactt cgtgggaggt ctgcaccggt tcccaccgac     3660

ttcaagcctc cggcagcgcc ccgaacctca agtcaccgga actgtggggc tccacctcgg     3720

cgtcacccct tccgtgatcc ggaagcggta caatctgacc tcgcaagacg tgggctcggg     3780

aacctcaaac aacagccagg cctgcgccca atttctggaa cagtacttcc acgatagcga     3840

tctggcccag ttcatgcgac ttttcggggg gaatttcgcc caccaagcca gcgtggcccg     3900

cgtggtcggg caacaggggc gcggaagggc gggcatcgag gcttccctgg atgtccagta     3960

cctcatgtcc gccggggcca acatctccac ttgggtgtac tcctcacctg gccgccacga     4020

ggggcaggaa ccgtttctgc aatggctgat gctgctgagc aacgaatccg cactcccgca     4080

cgtgcatact gtctcgtacg gcgacgatga ggactcactg tcctccgcgt acatccagag     4140

agtgaacact gagctcatga aggccgccgc gcggggcctg actttgttgt tcgcaagcgg     4200

cgattcggga gcgggatgtt ggtcggtgtc cggacgccat cagttccgcc cgaccttccc     4260

tgcctcaagc ccctacgtga caaccgtggg aggcaccagc tttcaggagc cgtttctgat     4320

taccaacgaa atcgtcgact acatttcggg cggcggtttc tccaacgtgt tcccacgccc     4380

ctcgtaccaa gaagaggccg tcaccaagtt cctgtcctcc tcccctcatc tcccgccatc     4440

ctcctacttt aacgcctccg gtcgggccta tcccgatgtg gccgccctgt cggacggcta     4500

ctgggtggtg tcgaataggg tgccgatccc ctgggtcagc ggaacttccg cgtccactcc     4560

tgtgtttggc ggcattcttt ccttgatcaa cgagcaccgg attctgtcgg gtagaccgcc     4620

gctgggattc ctcaacccgc ggctgtacca gcagcacggt gccggactgt tcgacgtgac     4680

gagagggtgc cacgagtcct gcctggacga ggaagtggaa ggacagggat tctgctctgg     4740

acccggatgg gatccggtca ccggctgggg caccccgaac ttccctgcgc tgctcaagac     4800

cctcctgaac ccctgatagt aatgacaggt acctttaaga ccaatgactt acaaggcagc     4860

tgtagatctt agccactttt taaaagaaaa ggggggactg gaagggctaa ttcactccca     4920

aagaagacaa gatctgcttt ttgcctgtac tgggtctctc tggttagacc agatctgagc     4980

ctgggagctc tctggctaac tagggaaccc actgcttaag cctcaataaa gcttgccttg     5040

agtgcttcaa tgtgtgtgtt ggttttttgt gtgtcgaaat tctagcgatt ctagcttggc     5100

gtaatcatgg tcatagctgt ttcctgtgtg aaattgttat ccgctcacaa ttccacacaa     5160

catacgagcc ggaagcataa agtgtaaagc ctggggtgcc taatgagtga gctaactcac     5220

attaattgcg ttgcgctcac tgcccgcttt ccagtcggga aacctgtcgt gccagctgca     5280

ttaatgaatc ggccaacgcg cggggagagg cggtttgcgt attgggcgct cttccgcttc     5340

ctcgctcact gactcgctgc gctcggtcgt tcggctgcgg cgagcggtat cagctcactc     5400

aaaggcggta atacggttat ccacagaatc aggggataac gcaggaaaga acatgtgagc     5460

aaaaggccag caaaaggcca ggaaccgtaa aaaggccgcg ttgctggcgt ttttccatag     5520

gctccgcccc cctgacgagc atcacaaaaa tcgacgctca agtcagaggt ggcgaaaccc     5580

gacaggacta taaagatacc aggcgtttcc ccctggaagc tccctcgtgc gctctcctgt     5640

tccgaccctg ccgcttaccg gatacctgtc cgcctttctc ccttcgggaa gcgtggcgct     5700

ttctcatagc tcacgctgta ggtatctcag ttcggtgtag gtcgttcgct ccaagctggg     5760

ctgtgtgcac gaaccccccg ttcagcccga ccgctgcgcc ttatccggta actatcgtct     5820

tgagtccaac ccggtaagac acgacttatc gccactggca gcagccactg gtaacaggat     5880

tagcagagcg aggtatgtag gcggtgctac agagttcttg aagtggtggc ctaactacgg     5940

ctacactaga agaacagtat ttggtatctg cgctctgctg aagccagtta ccttcggaaa     6000

aagagttggt agctcttgat ccggcaaaca aaccaccgct ggtagcggtg gtttttttgt     6060

ttgcaagcag cagattacgc gcagaaaaaa aggatctcaa gaagatcctt tgatcttttc     6120

tacggggtct gacgctcagt ggaacgaaaa ctcacgttaa gggattttgg tcatgagatt     6180

atcaaaaagg atcttcacct agatcctttt aaattaaaaa tgaagtttta aatcaatcta     6240

aagtatatat gagtaaactt ggtctgacag ttaccaatgc ttaatcagtg aggcacctat     6300

ctcagcgatc tgtctatttc gttcatccat agttgcctga ctccccgtcg tgtagataac     6360

tacgatacgg gagggcttac catctggccc cagtgctgca atgataccgc gagacccacg     6420

ctcaccggct ccagatttat cagcaataaa ccagccagcc ggaagggccg agcgcagaag     6480

tggtcctgca actttatccg cctccatcca gtctattaat tgttgccggg aagctagagt     6540

aagtagttcg ccagttaata gtttgcgcaa cgttgttgcc attgctacag gcatcgtggt     6600

gtcacgctcg tcgtttggta tggcttcatt cagctccggt tcccaacgat caaggcgagt     6660

tacatgatcc cccatgttgt gcaaaaaagc ggttagctcc ttcggtcctc cgatcgttgt     6720

cagaagtaag ttggccgcag tgttatcact catggttatg gcagcactgc ataattctct     6780

tactgtcatg ccatccgtaa gatgcttttc tgtgactggt gagtactcaa ccaagtcatt     6840

ctgagaatag tgtatgcggc gaccgagttg ctcttgcccg gcgtcaatac gggataatac     6900

cgcgccacat agcagaactt taaaagtgct catcattgga aaacgttctt cggggcgaaa     6960

actctcaagg atcttaccgc tgttgagatc cagttcgatg taacccactc gtgcacccaa     7020

ctgatcttca gcatctttta ctttcaccag cgtttctggg tgagcaaaaa caggaaggca     7080

aaatgccgca aaaaagggaa taagggcgac acggaaatgt tgaatactca tactcttcct     7140

ttttcaatat tattgaagca tttatcaggg ttattgtctc atgagcggat acatatttga     7200

atgtatttag aaaaataaac aaataggggt tccgcgcaca tttccccgaa aagtgccacc     7260

tgggactagc tttttgcaaa agcctaggcc tccaaaaaag cctcctcact acttctggaa     7320

tagctcagag gccgaggcgg cctcggcctc tgcataaata aaaaaaatta gtcagccatg     7380

gggcggagaa tgggcggaac tgggcggagt taggggcggg atgggcggag ttaggggcgg     7440

gactatggtt gctgactaat tgagatgagc ttgcatgccg acattgatta ttgactagtc     7500

cctaagaaac cattcttatc atgacattaa cctataaaaa taggcgtatc acgaggccct     7560

ttcgtc                                                                7566


<210>  2
<211>  7700
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleic acid sequence of a lentiviral vector encoding TTP1 
       (pEF1alpha-CLN2)

<400>  2
tcgcgcgttt cggtgatgac ggtgaaaacc tctgacacat gcagctcccg gagacggtca       60

cagcttgtct gtaagcggat gccgggagca gacaagcccg tcagggcgcg tcagcgggtg      120

ttggcgggtg tcggggctgg cttaactatg cggcatcaga gcagattgta ctgagagtgc      180

accatcatat gccagcctat ggtgacattg attattgact agttattaat agtaatcaat      240

tacggggtca ttagttcata gcccatatat ggagttccgc gttacataac ttacggtaaa      300

tggcccgcct ggctgaccgc ccaacgaccc ccgcccattg acgtcaataa tgacgtatgt      360

tcccatagta acgccaatag ggactttcca ttgacgtcaa tgggtggagt atttacggta      420

aactgcccac ttggcagtac atcaagtgta tcatatgcca agtacgcccc ctattgacgt      480

caatgacggt aaatggcccg cctggcatta tgcccagtac atgaccttat gggactttcc      540

tacttggcag tacatctacg tattagtcat cgctattacc atggtgatgc ggttttggca      600

gtacatcaat gggcgtggat agcggtttga ctcacgggga tttccaagtc tccaccccat      660

tgacgtcaat gggagtttgt tttggcacca aaatcaacgg gactttccaa aatgtcgtaa      720

caactccgcc ccattgacgc aaatgggcgg taggcgtgta cggtgggagg tctatataag      780

cagagctcgt ttagtgaacc gggtctctct ggttagacca gatctgagcc tgggagctct      840

ctggctaact agggaaccca ctgcttaagc ctcaataaag cttgccttga gtgctcaaag      900

tagtgtgtgc ccgtctgttg tgtgactctg gtaactagag atccctcaga cccttttagt      960

cagtgtggaa aatctctagc agtggcgccc gaacagggac ttgaaagcga aagtaaagcc     1020

agaggagatc tctcgacgca ggactcggct tgctgaagcg cgcacggcaa gaggcgaggg     1080

gcggcgactg gtgagtacgc caaaaatttt gactagcgga ggctagaagg agagagtagg     1140

gtgcgagagc gtcggtatta agcgggggag aattagataa atgggaaaaa attcggttaa     1200

ggccaggggg aaagaaacaa tataaactaa aacatatagt tagggcaagc agggagctag     1260

aacgattcgc agttaatcct ggccttttag agacatcaga aggctgtaga caaatactgg     1320

gacagctaca accatccctt cagacaggat cagaagaact tagatcatta tataatacaa     1380

tagcagtcct ctattgtgtg catcaaagga tagatgtaaa agacaccaag gaagccttag     1440

ataagataga ggaagagcaa aacaaaagta agaaaaaggc acagcaagca gcagctgaca     1500

caggaaacaa cagccaggtc agccaaaatt accctatagt gcagaacctc caggggcaaa     1560

tggtacatca ggccatatca cctagaactt taaattaaga cagcagtaca aatggcagta     1620

ttcatccaca attttaaaag aaaagggggg attggggggt acagtgcagg ggaaagaata     1680

gtagacataa tagcaacaga catacaaact aaagaattac aaaaacaaat tacaaaaatt     1740

caaaattttc gggtttatta cagggacagc agagatccag tttggaaagg accagcaaag     1800

ctcctctgga aaggtgaagg ggcagtagta atacaagata atagtgacat aaaagtagtg     1860

ccaagaagaa aagcaaagat catcagggat tatggaaaac agatggcagg tgatgattgt     1920

gtggcaagta gacaggatga ggattaacac atggaaaaga ttagtaaaac accatagctc     1980

tagagcgatc ccgatcttca gacctggagg aggagatatg agggacaatt ggagaagtga     2040

attatataaa tataaagtag taaaaattga accattagga gtagcaccca ccaaggcaaa     2100

gagaagagtg gtgcagagag aaaaaagagc agtgggaata ggagctttgt tccttgggtt     2160

cttgggagca gcaggaagca ctatgggcgc agcgtcaatg acgctgacgg tacaggccag     2220

acaattattg tctggtatag tgcagcagca gaacaatttg ctgagggcta ttgaggcgca     2280

acagcatctg ttgcaactca cagtctgggg catcaagcag ctccaggcaa gaatcctggc     2340

tgtggaaaga tacctaaagg atcaacagct cctggggatt tggggttgct ctggaaaact     2400

catttgcacc actgctgtgc cttggaatgc tagttggagt aataaatctc tggaacagat     2460

ttggaatcac acgacctgga tggagtggga cagagaaatt aacaattaca caagcttggt     2520

aggtttaaga atagtttttg ctgtactttc tatagtgaat agagttaggc agggatattc     2580

accattatcg tttcagaccc acctcccaac cccgagggga cccgacaggc ccgaaggaat     2640

agaagaagaa ggtggagaga gagacagaga cagatccatt cgattagtga acggatccaa     2700

ggatctgcga tcgctccggt gcccgtcagt gggcagagcg cacatcgccc acagtccccg     2760

agaagttggg gggaggggtc ggcaattgaa cgggtgccta gagaaggtgg cgcggggtaa     2820

actgggaaag tgatgtcgtg tactggctcc gcctttttcc cgagggtggg ggagaaccgt     2880

atataagtgc agtagtcgcc gtgaacgttc tttttcgcaa cgggtttgcc gccagaacac     2940

agctgaagct tcgaggggct cgcatctctc cttcacgcgc ccgccgccct acctgaggcc     3000

gccatccacg ccggttgagt cgcgttctgc cgcctcccgc ctgtggtgcc tcctgaactg     3060

cgtccgccgt ctaggtaagt ttaaagctca ggtcgagacc gggcctttgt ccggcgctcc     3120

cttggagcct acctagactc agccggctct ccacgctttg cctgaccctg cttgctcaac     3180

tctacgtctt tgtttcgttt tctgttctgc gccgttacag atccaagctg tgaccggcgc     3240

ctacgcgtct acgccaccat ggggttgcag gcttgcctgc tgggactttt cgctctgatc     3300

ctgagcggaa agtgctccta ctcacctgag ccagaccaga ggagaactct gccccccgga     3360

tgggtgtccc tgggaagggc cgaccctgag gaggaactct cgctcacctt cgcactgcgg     3420

cagcagaacg tggaaagact gtccgaactg gtgcaggcag tgtccgaccc ctcgagcccg     3480

cagtacggaa agtacctgac cctcgaaaac gtggcagact tggtccggcc ctcccctctc     3540

accctgcaca ccgtgcaaaa atggctgctg gccgccggag ctcagaagtg ccattccgtg     3600

attacacagg acttccttac ctgttggctt agcatccgcc aagcggagct gctgctgcct     3660

ggtgccgagt tccaccacta cgtgggcggg ccaactgaaa cccacgtcgt gcgcagcccg     3720

cacccgtatc agctgcccca ggcgctggct cctcatgtgg acttcgtggg aggtctgcac     3780

cggttcccac cgacttcaag cctccggcag cgccccgaac ctcaagtcac cggaactgtg     3840

gggctccacc tcggcgtcac cccttccgtg atccggaagc ggtacaatct gacctcgcaa     3900

gacgtgggct cgggaacctc aaacaacagc caggcctgcg cccaatttct ggaacagtac     3960

ttccacgata gcgatctggc ccagttcatg cgacttttcg gggggaattt cgcccaccaa     4020

gccagcgtgg cccgcgtggt cgggcaacag gggcgcggaa gggcgggcat cgaggcttcc     4080

ctggatgtcc agtacctcat gtccgccggg gccaacatct ccacttgggt gtactcctca     4140

cctggccgcc acgaggggca ggaaccgttt ctgcaatggc tgatgctgct gagcaacgaa     4200

tccgcactcc cgcacgtgca tactgtctcg tacggcgacg atgaggactc actgtcctcc     4260

gcgtacatcc agagagtgaa cactgagctc atgaaggccg ccgcgcgggg cctgactttg     4320

ttgttcgcaa gcggcgattc gggagcggga tgttggtcgg tgtccggacg ccatcagttc     4380

cgcccgacct tccctgcctc aagcccctac gtgacaaccg tgggaggcac cagctttcag     4440

gagccgtttc tgattaccaa cgaaatcgtc gactacattt cgggcggcgg tttctccaac     4500

gtgttcccac gcccctcgta ccaagaagag gccgtcacca agttcctgtc ctcctcccct     4560

catctcccgc catcctccta ctttaacgcc tccggtcggg cctatcccga tgtggccgcc     4620

ctgtcggacg gctactgggt ggtgtcgaat agggtgccga tcccctgggt cagcggaact     4680

tccgcgtcca ctcctgtgtt tggcggcatt ctttccttga tcaacgagca ccggattctg     4740

tcgggtagac cgccgctggg attcctcaac ccgcggctgt accagcagca cggtgccgga     4800

ctgttcgacg tgacgagagg gtgccacgag tcctgcctgg acgaggaagt ggaaggacag     4860

ggattctgct ctggacccgg atgggatccg gtcaccggct ggggcacccc gaacttccct     4920

gcgctgctca agaccctcct gaacccctga tagtaatgac aggtaccttt aagaccaatg     4980

acttacaagg cagctgtaga tcttagccac tttttaaaag aaaagggggg actggaaggg     5040

ctaattcact cccaaagaag acaagatctg ctttttgcct gtactgggtc tctctggtta     5100

gaccagatct gagcctggga gctctctggc taactaggga acccactgct taagcctcaa     5160

taaagcttgc cttgagtgct tcaatgtgtg tgttggtttt ttgtgtgtcg aaattctagc     5220

gattctagct tggcgtaatc atggtcatag ctgtttcctg tgtgaaattg ttatccgctc     5280

acaattccac acaacatacg agccggaagc ataaagtgta aagcctgggg tgcctaatga     5340

gtgagctaac tcacattaat tgcgttgcgc tcactgcccg ctttccagtc gggaaacctg     5400

tcgtgccagc tgcattaatg aatcggccaa cgcgcgggga gaggcggttt gcgtattggg     5460

cgctcttccg cttcctcgct cactgactcg ctgcgctcgg tcgttcggct gcggcgagcg     5520

gtatcagctc actcaaaggc ggtaatacgg ttatccacag aatcagggga taacgcagga     5580

aagaacatgt gagcaaaagg ccagcaaaag gccaggaacc gtaaaaaggc cgcgttgctg     5640

gcgtttttcc ataggctccg cccccctgac gagcatcaca aaaatcgacg ctcaagtcag     5700

aggtggcgaa acccgacagg actataaaga taccaggcgt ttccccctgg aagctccctc     5760

gtgcgctctc ctgttccgac cctgccgctt accggatacc tgtccgcctt tctcccttcg     5820

ggaagcgtgg cgctttctca tagctcacgc tgtaggtatc tcagttcggt gtaggtcgtt     5880

cgctccaagc tgggctgtgt gcacgaaccc cccgttcagc ccgaccgctg cgccttatcc     5940

ggtaactatc gtcttgagtc caacccggta agacacgact tatcgccact ggcagcagcc     6000

actggtaaca ggattagcag agcgaggtat gtaggcggtg ctacagagtt cttgaagtgg     6060

tggcctaact acggctacac tagaagaaca gtatttggta tctgcgctct gctgaagcca     6120

gttaccttcg gaaaaagagt tggtagctct tgatccggca aacaaaccac cgctggtagc     6180

ggtggttttt ttgtttgcaa gcagcagatt acgcgcagaa aaaaaggatc tcaagaagat     6240

cctttgatct tttctacggg gtctgacgct cagtggaacg aaaactcacg ttaagggatt     6300

ttggtcatga gattatcaaa aaggatcttc acctagatcc ttttaaatta aaaatgaagt     6360

tttaaatcaa tctaaagtat atatgagtaa acttggtctg acagttacca atgcttaatc     6420

agtgaggcac ctatctcagc gatctgtcta tttcgttcat ccatagttgc ctgactcccc     6480

gtcgtgtaga taactacgat acgggagggc ttaccatctg gccccagtgc tgcaatgata     6540

ccgcgagacc cacgctcacc ggctccagat ttatcagcaa taaaccagcc agccggaagg     6600

gccgagcgca gaagtggtcc tgcaacttta tccgcctcca tccagtctat taattgttgc     6660

cgggaagcta gagtaagtag ttcgccagtt aatagtttgc gcaacgttgt tgccattgct     6720

acaggcatcg tggtgtcacg ctcgtcgttt ggtatggctt cattcagctc cggttcccaa     6780

cgatcaaggc gagttacatg atcccccatg ttgtgcaaaa aagcggttag ctccttcggt     6840

cctccgatcg ttgtcagaag taagttggcc gcagtgttat cactcatggt tatggcagca     6900

ctgcataatt ctcttactgt catgccatcc gtaagatgct tttctgtgac tggtgagtac     6960

tcaaccaagt cattctgaga atagtgtatg cggcgaccga gttgctcttg cccggcgtca     7020

atacgggata ataccgcgcc acatagcaga actttaaaag tgctcatcat tggaaaacgt     7080

tcttcggggc gaaaactctc aaggatctta ccgctgttga gatccagttc gatgtaaccc     7140

actcgtgcac ccaactgatc ttcagcatct tttactttca ccagcgtttc tgggtgagca     7200

aaaacaggaa ggcaaaatgc cgcaaaaaag ggaataaggg cgacacggaa atgttgaata     7260

ctcatactct tcctttttca atattattga agcatttatc agggttattg tctcatgagc     7320

ggatacatat ttgaatgtat ttagaaaaat aaacaaatag gggttccgcg cacatttccc     7380

cgaaaagtgc cacctgggac tagctttttg caaaagccta ggcctccaaa aaagcctcct     7440

cactacttct ggaatagctc agaggccgag gcggcctcgg cctctgcata aataaaaaaa     7500

attagtcagc catggggcgg agaatgggcg gaactgggcg gagttagggg cgggatgggc     7560

ggagttaggg gcgggactat ggttgctgac taattgagat gagcttgcat gccgacattg     7620

attattgact agtccctaag aaaccattct tatcatgaca ttaacctata aaaataggcg     7680

tatcacgagg ccctttcgtc                                                 7700


<210>  3
<211>  1719
<212>  DNA
<213>  Homo sapiens

<400>  3
atgacagcag atccgcggaa gggcagaatg ggactccaag cctgcctcct agggctcttt       60

gccctcatcc tctctggcaa atgcagttac agcccggagc ccgaccagcg gaggacgctg      120

cccccaggct gggtgtccct gggccgtgcg gaccctgagg aagagctgag tctcaccttt      180

gccctgagac agcagaatgt ggaaagactc tcggagctgg tgcaggctgt gtcggatccc      240

agctctcctc aatacggaaa atacctgacc ctagagaatg tggctgatct ggtgaggcca      300

tccccactga ccctccacac ggtgcaaaaa tggctcttgg cagccggagc ccagaagtgc      360

cattctgtga tcacacagga ctttctgact tgctggctga gcatccgaca agcagagctg      420

ctgctccctg gggctgagtt tcatcactat gtgggaggac ctacggaaac ccatgttgta      480

aggtccccac atccctacca gcttccacag gccttggccc cccatgtgga ctttgtgggg      540

ggactgcacc gttttccccc aacatcatcc ctgaggcaac gtcctgagcc gcaggtgaca      600

gggactgtag gcctgcatct gggggtaacc ccctctgtga tccgtaagcg atacaacttg      660

acctcacaag acgtgggctc tggcaccagc aataacagcc aagcctgtgc ccagttcctg      720

gagcagtatt tccatgactc agacctggct cagttcatgc gcctcttcgg tggcaacttt      780

gcacatcagg catcagtagc ccgtgtggtt ggacaacagg gccggggccg ggccgggatt      840

gaggccagtc tagatgtgca gtacctgatg agtgctggtg ccaacatctc cacctgggtc      900

tacagtagcc ctggccggca tgagggacag gagcccttcc tgcagtggct catgctgctc      960

agtaatgagt cagccctgcc acatgtgcat actgtgagct atggagatga tgaggactcc     1020

ctcagcagcg cctacatcca gcgggtcaac actgagctca tgaaggctgc cgctcggggt     1080

ctcaccctgc tcttcgcctc aggtgacagt ggggccgggt gttggtctgt ctctggaaga     1140

caccagttcc gccctacctt ccctgcctcc agcccctatg tcaccacagt gggaggcaca     1200

tccttccagg aacctttcct catcacaaat gaaattgttg actatatcag tggtggtggc     1260

ttcagcaatg tgttcccacg gccttcatac caggaggaag ctgtaacgaa gttcctgagc     1320

tctagccccc acctgccacc atccagttac ttcaatgcca gtggccgtgc ctacccagat     1380

gtggctgcac tttctgatgg ctactgggtg gtcagcaaca gagtgcccat tccatgggtg     1440

tccggaacct cggcctctac tccagtgttt ggggggatcc tatccttgat caatgagcac     1500

aggatcctta gtggccgccc ccctcttggc tttctcaacc caaggctcta ccagcagcat     1560

ggggcaggac tctttgatgt aacccgtggc tgccatgagt cctgtctgga tgaagaggta     1620

gagggccagg gtttctgctc tggtcctggc tgggatcctg taacaggctg gggaacaccc     1680

aacttcccag ctttgctgaa gactctactc aacccctga                            1719


<210>  4
<211>  1692
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon-optimized polynucleotide sequence encoding a human 
       tripeptidyl peptidase 1 (TPP1)

<400>  4
atggggttgc aggcttgcct gctgggactt ttcgctctga tcctgagcgg aaagtgctcc       60

tactcacctg agccagacca gaggagaact ctgccccccg gatgggtgtc cctgggaagg      120

gccgaccctg aggaggaact ctcgctcacc ttcgcactgc ggcagcagaa cgtggaaaga      180

ctgtccgaac tggtgcaggc agtgtccgac ccctcgagcc cgcagtacgg aaagtacctg      240

accctcgaaa acgtggcaga cttggtccgg ccctcccctc tcaccctgca caccgtgcaa      300

aaatggctgc tggccgccgg agctcagaag tgccattccg tgattacaca ggacttcctt      360

acctgttggc ttagcatccg ccaagcggag ctgctgctgc ctggtgccga gttccaccac      420

tacgtgggcg ggccaactga aacccacgtc gtgcgcagcc cgcacccgta tcagctgccc      480

caggcgctgg ctcctcatgt ggacttcgtg ggaggtctgc accggttccc accgacttca      540

agcctccggc agcgccccga acctcaagtc accggaactg tggggctcca cctcggcgtc      600

accccttccg tgatccggaa gcggtacaat ctgacctcgc aagacgtggg ctcgggaacc      660

tcaaacaaca gccaggcctg cgcccaattt ctggaacagt acttccacga tagcgatctg      720

gcccagttca tgcgactttt cggggggaat ttcgcccacc aagccagcgt ggcccgcgtg      780

gtcgggcaac aggggcgcgg aagggcgggc atcgaggctt ccctggatgt ccagtacctc      840

atgtccgccg gggccaacat ctccacttgg gtgtactcct cacctggccg ccacgagggg      900

caggaaccgt ttctgcaatg gctgatgctg ctgagcaacg aatccgcact cccgcacgtg      960

catactgtct cgtacggcga cgatgaggac tcactgtcct ccgcgtacat ccagagagtg     1020

aacactgagc tcatgaaggc cgccgcgcgg ggcctgactt tgttgttcgc aagcggcgat     1080

tcgggagcgg gatgttggtc ggtgtccgga cgccatcagt tccgcccgac cttccctgcc     1140

tcaagcccct acgtgacaac cgtgggaggc accagctttc aggagccgtt tctgattacc     1200

aacgaaatcg tcgactacat ttcgggcggc ggtttctcca acgtgttccc acgcccctcg     1260

taccaagaag aggccgtcac caagttcctg tcctcctccc ctcatctccc gccatcctcc     1320

tactttaacg cctccggtcg ggcctatccc gatgtggccg ccctgtcgga cggctactgg     1380

gtggtgtcga atagggtgcc gatcccctgg gtcagcggaa cttccgcgtc cactcctgtg     1440

tttggcggca ttctttcctt gatcaacgag caccggattc tgtcgggtag accgccgctg     1500

ggattcctca acccgcggct gtaccagcag cacggtgccg gactgttcga cgtgacgaga     1560

gggtgccacg agtcctgcct ggacgaggaa gtggaaggac agggattctg ctctggaccc     1620

ggatgggatc cggtcaccgg ctggggcacc ccgaacttcc ctgcgctgct caagaccctc     1680

ctgaacccct ga                                                         1692


<210>  5
<211>  563
<212>  PRT
<213>  Homo sapiens

<400>  5

Met Gly Leu Gln Ala Cys Leu Leu Gly Leu Phe Ala Leu Ile Leu Ser 
1               5                   10                  15      


Gly Lys Cys Ser Tyr Ser Pro Glu Pro Asp Gln Arg Arg Thr Leu Pro 
            20                  25                  30          


Pro Gly Trp Val Ser Leu Gly Arg Ala Asp Pro Glu Glu Glu Leu Ser 
        35                  40                  45              


Leu Thr Phe Ala Leu Arg Gln Gln Asn Val Glu Arg Leu Ser Glu Leu 
    50                  55                  60                  


Val Gln Ala Val Ser Asp Pro Ser Ser Pro Gln Tyr Gly Lys Tyr Leu 
65                  70                  75                  80  


Thr Leu Glu Asn Val Ala Asp Leu Val Arg Pro Ser Pro Leu Thr Leu 
                85                  90                  95      


His Thr Val Gln Lys Trp Leu Leu Ala Ala Gly Ala Gln Lys Cys His 
            100                 105                 110         


Ser Val Ile Thr Gln Asp Phe Leu Thr Cys Trp Leu Ser Ile Arg Gln 
        115                 120                 125             


Ala Glu Leu Leu Leu Pro Gly Ala Glu Phe His His Tyr Val Gly Gly 
    130                 135                 140                 


Pro Thr Glu Thr His Val Val Arg Ser Pro His Pro Tyr Gln Leu Pro 
145                 150                 155                 160 


Gln Ala Leu Ala Pro His Val Asp Phe Val Gly Gly Leu His Arg Phe 
                165                 170                 175     


Pro Pro Thr Ser Ser Leu Arg Gln Arg Pro Glu Pro Gln Val Thr Gly 
            180                 185                 190         


Thr Val Gly Leu His Leu Gly Val Thr Pro Ser Val Ile Arg Lys Arg 
        195                 200                 205             


Tyr Asn Leu Thr Ser Gln Asp Val Gly Ser Gly Thr Ser Asn Asn Ser 
    210                 215                 220                 


Gln Ala Cys Ala Gln Phe Leu Glu Gln Tyr Phe His Asp Ser Asp Leu 
225                 230                 235                 240 


Ala Gln Phe Met Arg Leu Phe Gly Gly Asn Phe Ala His Gln Ala Ser 
                245                 250                 255     


Val Ala Arg Val Val Gly Gln Gln Gly Arg Gly Arg Ala Gly Ile Glu 
            260                 265                 270         


Ala Ser Leu Asp Val Gln Tyr Leu Met Ser Ala Gly Ala Asn Ile Ser 
        275                 280                 285             


Thr Trp Val Tyr Ser Ser Pro Gly Arg His Glu Gly Gln Glu Pro Phe 
    290                 295                 300                 


Leu Gln Trp Leu Met Leu Leu Ser Asn Glu Ser Ala Leu Pro His Val 
305                 310                 315                 320 


His Thr Val Ser Tyr Gly Asp Asp Glu Asp Ser Leu Ser Ser Ala Tyr 
                325                 330                 335     


Ile Gln Arg Val Asn Thr Glu Leu Met Lys Ala Ala Ala Arg Gly Leu 
            340                 345                 350         


Thr Leu Leu Phe Ala Ser Gly Asp Ser Gly Ala Gly Cys Trp Ser Val 
        355                 360                 365             


Ser Gly Arg His Gln Phe Arg Pro Thr Phe Pro Ala Ser Ser Pro Tyr 
    370                 375                 380                 


Val Thr Thr Val Gly Gly Thr Ser Phe Gln Glu Pro Phe Leu Ile Thr 
385                 390                 395                 400 


Asn Glu Ile Val Asp Tyr Ile Ser Gly Gly Gly Phe Ser Asn Val Phe 
                405                 410                 415     


Pro Arg Pro Ser Tyr Gln Glu Glu Ala Val Thr Lys Phe Leu Ser Ser 
            420                 425                 430         


Ser Pro His Leu Pro Pro Ser Ser Tyr Phe Asn Ala Ser Gly Arg Ala 
        435                 440                 445             


Tyr Pro Asp Val Ala Ala Leu Ser Asp Gly Tyr Trp Val Val Ser Asn 
    450                 455                 460                 


Arg Val Pro Ile Pro Trp Val Ser Gly Thr Ser Ala Ser Thr Pro Val 
465                 470                 475                 480 


Phe Gly Gly Ile Leu Ser Leu Ile Asn Glu His Arg Ile Leu Ser Gly 
                485                 490                 495     


Arg Pro Pro Leu Gly Phe Leu Asn Pro Arg Leu Tyr Gln Gln His Gly 
            500                 505                 510         


Ala Gly Leu Phe Asp Val Thr Arg Gly Cys His Glu Ser Cys Leu Asp 
        515                 520                 525             


Glu Glu Val Glu Gly Gln Gly Phe Cys Ser Gly Pro Gly Trp Asp Pro 
    530                 535                 540                 


Val Thr Gly Trp Gly Thr Pro Asn Phe Pro Ala Leu Leu Lys Thr Leu 
545                 550                 555                 560 


Leu Asn Pro 
            


<210>  6
<211>  3
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  6

Gly Gly Gly 
1           


<210>  7
<211>  5
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  7

Asp Gly Gly Gly Ser 
1               5   


<210>  8
<211>  5
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  8

Thr Gly Glu Lys Pro 
1               5   


<210>  9
<211>  4
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  9

Gly Gly Arg Arg 
1               


<210>  10
<211>  5
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  10

Gly Gly Gly Gly Ser 
1               5   


<210>  11
<211>  14
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  11

Glu Gly Lys Ser Ser Gly Ser Gly Ser Glu Ser Lys Val Asp 
1               5                   10                  


<210>  12
<211>  18
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  12

Lys Glu Ser Gly Ser Val Ser Ser Glu Gln Leu Ala Gln Phe Arg Ser 
1               5                   10                  15      


Leu Asp 
        


<210>  13
<211>  8
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  13

Gly Gly Arg Arg Gly Gly Gly Ser 
1               5               


<210>  14
<211>  9
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  14

Leu Arg Gln Arg Asp Gly Glu Arg Pro 
1               5                   


<210>  15
<211>  12
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  15

Leu Arg Gln Lys Asp Gly Gly Gly Ser Glu Arg Pro 
1               5                   10          


<210>  16
<211>  16
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Exemplary linker sequence

<400>  16

Leu Arg Gln Lys Asp Gly Gly Gly Ser Gly Gly Gly Ser Glu Arg Pro 
1               5                   10                  15      


<210>  17
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  TEV protease cleavage sequence motif


<220>
<221>  MISC_FEATURE
<222>  (2)..(3)
<223>  Xaa is any amino acid

<220>
<221>  MISC_FEATURE
<222>  (5)..(5)
<223>  Xaa is any amino acid

<220>
<221>  MISC_FEATURE
<222>  (7)..(7)
<223>  Xaa is Gly or Ser

<400>  17

Glu Xaa Xaa Tyr Xaa Gln Xaa 
1               5           


<210>  18
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Cleavage sequence by TEV protease

<400>  18

Glu Asn Leu Tyr Phe Gln Gly 
1               5           


<210>  19
<211>  7
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Cleavage sequence by TEV protease

<400>  19

Glu Asn Leu Tyr Phe Gln Ser 
1               5           


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

<220>
<223>  Consensus Kozak sequence

<400>  20
gccrccatgg                                                              10


