                         SEQUENCE LISTING

<110>  ALEXION PHARMACEUTICALS, INC.
       Hutchinson, Andrew
       Reynders, John V.W.
       del Angel, Guillermo
       Jain, Nina
       Forbes, Christen D.
       Xiao-Qin, Ren
       Burton, Barbara
 
<120>  LYSOSOMAL ACID LIPASE DEFICIENCY COMPOSITIONS AND METHODS

<130>  535.00010201

<150>  62/351631
<151>  2016-06-17

<150>  62/358951
<151>  2016-07-06

<150>  62/457356
<151>  2017-02-10

<160>  3     

<170>  PatentIn version 3.5

<210>  1
<211>  1200
<212>  DNA
<213>  Human

<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>  Human

<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>  21
<212>  PRT
<213>  Human

<400>  3

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 
            20      


