                         SEQUENCE LISTING

<110>  Research Institute at Nationwide Childrens Hospital
 
<120>  Materials and methods for the treatment of Lysosomal Acid Lipase 
       Deficiency (LAL-D)

<130>  28335/54743/PC

<150>  US 63/142,257
<151>  2021-01-27

<160>  7     

<170>  PatentIn version 3.5

<210>  1
<211>  1200
<212>  DNA
<213>  Homo sapiens

<400>  1
atgaaaatgc ggttcttggg gttggtggtc tgtttggttc tctggaccct gcattctgag       60

gggtctggag ggaaactgac agctgtggat cctgaaacaa acatgaatgt gagtgaaatt      120

atctcttact ggggattccc tagtgaggaa tacctagttg agacagaaga tggatatatt      180

ctgtgcctta accgaattcc tcatgggagg aagaaccatt ctgacaaagg tcccaaacca      240

gttgtcttcc tgcaacatgg cttgctggca gattctagta actgggtcac aaaccttgcc      300

aacagcagcc tgggcttcat tcttgctgat gctggttttg acgtgtggat gggcaacagc      360

agaggaaata cctggtctcg gaaacataag acactctcag tttctcagga tgaattctgg      420

gctttcagtt atgatgagat ggcaaaatat gacctaccag cttccattaa cttcattctg      480

aataaaactg gccaagaaca agtgtattat gtgggtcatt ctcaaggcac cactataggt      540

tttatagcat tttcacagat ccctgagctg gctaaaagga ttaaaatgtt ttttgccctg      600

ggtcctgtgg cttccgtcgc cttctgtact agccctatgg ccaaattagg acgattacca      660

gatcatctca ttaaggactt atttggagac aaagaatttc ttccccagag tgcgtttttg      720

aagtggctgg gtacccacgt ttgcactcat gtcatactga aggagctctg tggaaatctc      780

tgttttcttc tgtgtggatt taatgagaga aatttaaata tgtctagagt ggatgtatat      840

acaacacatt ctcctgctgg aacttctgtg caaaacatgt tacactggag ccaggctgtt      900

aaattccaaa agtttcaagc ctttgactgg ggaagcagtg ccaagaatta ttttcattac      960

aaccagagtt atcctcccac atacaatgtg aaggacatgc ttgtgccgac tgcagtctgg     1020

agcgggggtc acgactggct tgcagatgtc tacgacgtca atatcttact gactcagatc     1080

accaacttgg tgttccatga gagcattccg gaatgggagc atcttgactt catttggggc     1140

ctggatgccc cttggaggct ttataataaa attattaatc taatgaggaa atatcagtga     1200


<210>  2
<211>  399
<212>  PRT
<213>  Homo sapiens

<400>  2

Met Lys Met Arg Phe Leu Gly Leu Val Val Cys Leu Val Leu Trp Thr 
1               5                   10                  15      


Leu His Ser Glu Gly Ser Gly Gly Lys Leu Thr Ala Val Asp Pro Glu 
            20                  25                  30          


Thr Asn Met Asn Val Ser Glu Ile Ile Ser Tyr Trp Gly Phe Pro Ser 
        35                  40                  45              


Glu Glu Tyr Leu Val Glu Thr Glu Asp Gly Tyr Ile Leu Cys Leu Asn 
    50                  55                  60                  


Arg Ile Pro His Gly Arg Lys Asn His Ser Asp Lys Gly Pro Lys Pro 
65                  70                  75                  80  


Val Val Phe Leu Gln His Gly Leu Leu Ala Asp Ser Ser Asn Trp Val 
                85                  90                  95      


Thr Asn Leu Ala Asn Ser Ser Leu Gly Phe Ile Leu Ala Asp Ala Gly 
            100                 105                 110         


Phe Asp Val Trp Met Gly Asn Ser Arg Gly Asn Thr Trp Ser Arg Lys 
        115                 120                 125             


His Lys Thr Leu Ser Val Ser Gln Asp Glu Phe Trp Ala Phe Ser Tyr 
    130                 135                 140                 


Asp Glu Met Ala Lys Tyr Asp Leu Pro Ala Ser Ile Asn Phe Ile Leu 
145                 150                 155                 160 


Asn Lys Thr Gly Gln Glu Gln Val Tyr Tyr Val Gly His Ser Gln Gly 
                165                 170                 175     


Thr Thr Ile Gly Phe Ile Ala Phe Ser Gln Ile Pro Glu Leu Ala Lys 
            180                 185                 190         


Arg Ile Lys Met Phe Phe Ala Leu Gly Pro Val Ala Ser Val Ala Phe 
        195                 200                 205             


Cys Thr Ser Pro Met Ala Lys Leu Gly Arg Leu Pro Asp His Leu Ile 
    210                 215                 220                 


Lys Asp Leu Phe Gly Asp Lys Glu Phe Leu Pro Gln Ser Ala Phe Leu 
225                 230                 235                 240 


Lys Trp Leu Gly Thr His Val Cys Thr His Val Ile Leu Lys Glu Leu 
                245                 250                 255     


Cys Gly Asn Leu Cys Phe Leu Leu Cys Gly Phe Asn Glu Arg Asn Leu 
            260                 265                 270         


Asn Met Ser Arg Val Asp Val Tyr Thr Thr His Ser Pro Ala Gly Thr 
        275                 280                 285             


Ser Val Gln Asn Met Leu His Trp Ser Gln Ala Val Lys Phe Gln Lys 
    290                 295                 300                 


Phe Gln Ala Phe Asp Trp Gly Ser Ser Ala Lys Asn Tyr Phe His Tyr 
305                 310                 315                 320 


Asn Gln Ser Tyr Pro Pro Thr Tyr Asn Val Lys Asp Met Leu Val Pro 
                325                 330                 335     


Thr Ala Val Trp Ser Gly Gly His Asp Trp Leu Ala Asp Val Tyr Asp 
            340                 345                 350         


Val Asn Ile Leu Leu Thr Gln Ile Thr Asn Leu Val Phe His Glu Ser 
        355                 360                 365             


Ile Pro Glu Trp Glu His Leu Asp Phe Ile Trp Gly Leu Asp Ala Pro 
    370                 375                 380                 


Trp Arg Leu Tyr Asn Lys Ile Ile Asn Leu Met Arg Lys Tyr Gln 
385                 390                 395                 


<210>  3
<211>  228
<212>  DNA
<213>  Homo sapiens

<400>  3
tcgttacata acttacggta aatggcccgc ctggctgacc gcccaacgac ccccggactc       60

acggggattt ccaagtctcc accccattga cgtcaatggg agtttgtttt ggcaccaaaa      120

tcaacgggac tttccaaaat gtcgtaacaa ctccgcccca ttgacgcaaa tgggcggtag      180

gcgtgtacgg tgggaggtct atataagcag agctcgttta gtgaaccg                   228


<210>  4
<211>  5626
<212>  DNA
<213>  Homo sapiens

<400>  4
acccaactga tcttcagcat cttttacttt caccagcgtt tctgggtgag caaaaacagg       60

aaggcaaaat gccgcaaaaa agggaataag ggcgacacgg aaatgttgaa tactcatact      120

cttccttttt caatattatt gaagcattta tcagggttat tgtctcatga gcggatacat      180

atttgaatgt atttagaaaa ataaacaaat aggggttccg cgcacatttc cccgaaaagt      240

gccacctgac gtctaagaaa ccattattat catgacatta acctataaaa ataggcgtat      300

cacgaggccc tttcgtctcg cgcgtttcgg tgatgacggt gaaaacctct gacacatgca      360

gctcccggag acggtcacag cttgtctgta agcggatgcc gggagcagac aagcccgtca      420

gggcgcgtca gcgtgtgttg gcgggtgtcg gggctggctt aactatgcgg catcagagca      480

gattgtactg agagtgcacc atatgcggtg tgaaataccg cacagatgcg taaggagaaa      540

ataccgcatc aggcgattcc aacatccaat aaatcataca ggcaaggcaa agaattagca      600

aaattaagca ataaagcctc agagcataaa gctaaatcgg ttgtaccaaa aacattatga      660

ccctgtaata cttttgcggg agaagccttt atttcaacgc aaggataaaa atttttagaa      720

ccctcatata ttttaaatgc aatgcctgag taatgtgtag gtaaagattc aaacgggtga      780

gaaaggccgg agacagtcaa atcaccatca atatgatatt caaccgttct agctgataaa      840

ttcatgccgg agagggtagc tatttttgag aggtctctac aaaggctatc aggtcattgc      900

ctgagagtct ggagcaaaca agagaatcga tgaacggtaa tcgtaaaact agcatgtcaa      960

tcatatgtac cccggttgat aatcagaaaa gccccaaaaa caggaagatt gtataagcaa     1020

atatttaaat tgtaagcgtt aatattttgt taaaattcgc gttaaatttt tgttaaatca     1080

gctcattttt taaccaatag gccgaaatcg gcaaaatccc ttataaatca aaagaataga     1140

ccgagatagg gttgagtgtt gttccagttt ggaacaagag tccactatta aagaacgtgg     1200

actccaacgt caaagggcga aaaaccgtct atcagggcga tggcccacta cgtgaaccat     1260

caccctaatc aagttttttg gggtcgaggt gccgtaaagc actaaatcgg aaccctaaag     1320

ggagcccccg atttagagct tgacggggaa agccggcgaa cgtggcgaga aaggaaggga     1380

agaaagcgaa aggagcgggc gctagggcgc tggcaagtgt agcggtcacg ctgcgcgtaa     1440

ccaccacacc cgccgcgctt aatgcgccgc tacagggcgc gtactatggt tgctttgacg     1500

agcacgtata acgtgctttc ctcgttagaa tcagagcggg agctaaacag gaggccgatt     1560

aaagggattt tagacaggaa cggtacgcca gaatcctgag aagtgttttt ataatcagtg     1620

aggccaccga gtaaaagagt ctgtccatca cgcaaattaa ccgttgtcgc aatacttctt     1680

tgattagtaa taacatcact tgcctgagta gaagaactca aactatcggc cttgctggta     1740

atatccagaa caatattacc gccagccatt gcaacggaat cgccattcgc cattcaggct     1800

gcgcaactgt tgggaagggc gatcggtgcg ggcctcttcg ctattacgcc agctgcgcgc     1860

tcgctcgctc actgaggccg cccgggcaaa gcccgggcgt cgggcgacct ttggtcgccc     1920

ggcctcagtg agcgagcgag cgcgcagaga gggagtggcc aactccatca ctaggggttc     1980

ctaggaagct ttcgttacat aacttacggt aaatggcccg cctggctgac cgcccaacga     2040

cccccggact cacggggatt tccaagtctc caccccattg acgtcaatgg gagtttgttt     2100

tggcaccaaa atcaacggga ctttccaaaa tgtcgtaaca actccgcccc attgacgcaa     2160

atgggcggta ggcgtgtacg gtgggaggtc tatataagca gagctcgttt agtgaaccgc     2220

tcgaccgggg tacccggccg gctagccgcc accatgaaaa tgcggttctt ggggttggtg     2280

gtctgtttgg ttctctggac cctgcattct gaggggtctg gagggaaact gacagctgtg     2340

gatcctgaaa caaacatgaa tgtgagtgaa attatctctt actggggatt ccctagtgag     2400

gaatacctag ttgagacaga agatggatat attctgtgcc ttaaccgaat tcctcatggg     2460

aggaagaacc attctgacaa aggtcccaaa ccagttgtct tcctgcaaca tggcttgctg     2520

gcagattcta gtaactgggt cacaaacctt gccaacagca gcctgggctt cattcttgct     2580

gatgctggtt ttgacgtgtg gatgggcaac agcagaggaa atacctggtc tcggaaacat     2640

aagacactct cagtttctca ggatgaattc tgggctttca gttatgatga gatggcaaaa     2700

tatgacctac cagcttccat taacttcatt ctgaataaaa ctggccaaga acaagtgtat     2760

tatgtgggtc attctcaagg caccactata ggttttatag cattttcaca gatccctgag     2820

ctggctaaaa ggattaaaat gttttttgcc ctgggtcctg tggcttccgt cgccttctgt     2880

actagcccta tggccaaatt aggacgatta ccagatcatc tcattaagga cttatttgga     2940

gacaaagaat ttcttcccca gagtgcgttt ttgaagtggc tgggtaccca cgtttgcact     3000

catgtcatac tgaaggagct ctgtggaaat ctctgttttc ttctgtgtgg atttaatgag     3060

agaaatttaa atatgtctag agtggatgta tatacaacac attctcctgc tggaacttct     3120

gtgcaaaaca tgttacactg gagccaggct gttaaattcc aaaagtttca agcctttgac     3180

tggggaagca gtgccaagaa ttattttcat tacaaccaga gttatcctcc cacatacaat     3240

gtgaaggaca tgcttgtgcc gactgcagtc tggagcgggg gtcacgactg gcttgcagat     3300

gtctacgacg tcaatatctt actgactcag atcaccaact tggtgttcca tgagagcatt     3360

ccggaatggg agcatcttga cttcatttgg ggcctggatg ccccttggag gctttataat     3420

aaaattatta atctaatgag gaaatatcag tgagcatgca ctagtgcggc cgcggatctc     3480

agacatgata agatacattg atgagtttgg acaaaccaca actagaatgc agtgaaaaaa     3540

atgctttatt tgtgaaattt gtgatgctat tgctttattt gtaaccatta taagctgcaa     3600

taaacaagtt aacaacaaca attgcattca ttttatgttt caggttcagg gggaggtgtg     3660

ggaggttttt taggtttaaa ccccactccc tctctgcgcg ctcgctcgct cactgaggcc     3720

gggcgaccaa aggtcgcccg acgcccgggc tttgcccggg cggcctcagt gagcgagcga     3780

gcgcgcagct gcattaatga atcggccaac gcgcggggag aggcggtttg cgtattgggc     3840

gctcttccgc ttcctcgctc actgactcgc tgcgctcggt cgttcggctg cggcgagcgg     3900

tatcagctca ctcaaaggcg gtaatacggt tatccacaga atcaggggat aacgcaggaa     3960

agaacatgtg agcaaaaggc cagcaaaagg ccaggaaccg taaaaaggcc gcgttgctgg     4020

cgtttttcca taggctccgc ccccctgacg agcatcacaa aaatcgacgc tcaagtcaga     4080

ggtggcgaaa cccgacagga ctataaagat accaggcgtt tccccctgga agctccctcg     4140

tgcgctctcc tgttccgacc ctgccgctta ccggatacct gtccgccttt ctcccttcgg     4200

gaagcgtggc gctttctcat agctcacgct gtaggtatct cagttcggtg taggtcgttc     4260

gctccaagct gggctgtgtg cacgaacccc ccgttcagcc cgaccgctgc gccttatccg     4320

gtaactatcg tcttgagtcc aacccggtaa gacacgactt atcgccactg gcagcagcca     4380

ctggtaacag gattagcaga gcgaggtatg taggcggtgc tacagagttc ttgaagtggt     4440

ggcctaacta cggctacact agaaggacag tatttggtat ctgcgctctg ctgaagccag     4500

ttaccttcgg aaaaagagtt ggtagctctt gatccggcaa acaaaccacc gctggtagcg     4560

gtggtttttt tgtttgcaag cagcagatta cgcgcagaaa aaaaggatct caagaagatc     4620

ctttgatctt ttctacgggg tctgacgctc agtggaacga aaactcacgt taagggattt     4680

tggtcatgaa caataaaact gtctgcttac ataaacagta atacaagggg tgttatgagc     4740

catattcaac gggaaacgtc ttgctctagg ccgcgattaa attccaacat ggatgctgat     4800

ttatatgggt ataaatgggc tcgcgataat gtcgggcaat caggtgcgac aatctatcga     4860

ttgtatggga agcccgatgc gccagagttg tttctgaaac atggcaaagg tagcgttgcc     4920

aatgatgtta cagatgagat ggtcagacta aactggctga cggaatttat gcctcttccg     4980

accatcaagc attttatccg tactcctgat gatgcatggt tactcaccac tgcgatcccc     5040

gggaaaacag cattccaggt attagaagaa tatcctgatt caggtgaaaa tattgttgat     5100

gcgctggcag tgttcctgcg ccggttgcat tcgattcctg tttgtaattg tccttttaac     5160

agcgatcgcg tatttcgtct cgctcaggcg caatcacgaa tgaataacgg tttggttgat     5220

gcgagtgatt ttgatgacga gcgtaatggc tggcctgttg aacaagtctg gaaagaaatg     5280

cataaacttt tgccattctc accggattca gtcgtcactc atggtgattt ctcacttgat     5340

aaccttattt ttgacgaggg gaaattaata ggttgtattg atgttggacg agtcggaatc     5400

gcagaccgat accaggatct tgccatccta tggaactgcc tcggtgagtt ttctccttca     5460

ttacagaaac ggctttttca aaaatatggt attgataatc ctgatatgaa taaattgcag     5520

tttcatttga tgctcgatga gtttttctaa gaattcgttc ttcggggcga aaactctcaa     5580

ggatcttacc gctgttgaga tccagttcga tgtaacccac tcgtgc                    5626


<210>  5
<211>  192
<212>  DNA
<213>  Homo sapiens

<400>  5
cagacatgat aagatacatt gatgagtttg gacaaaccac aactagaatg cagtgaaaaa       60

aatgctttat ttgtgaaatt tgtgatgcta ttgctttatt tgtaaccatt ataagctgca      120

ataaacaagt taacaacaac aattgcattc attttatgtt tcaggttcag ggggaggtgt      180

gggaggtttt tt                                                          192


<210>  6
<211>  130
<212>  DNA
<213>  Homo sapiens

<400>  6
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct                                                             130


<210>  7
<211>  228
<212>  DNA
<213>  Homo sapiens

<400>  7
aaccccactc cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc       60

cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag ctgcattaat      120

gaatcggcca acgcgcgggg agaggcggtt tgcgtattgg gcgctcttcc gcttcctcgc      180

tcactgactc gctgcgctcg gtcgttcggc tgcggcgagc ggtatcag                   228


