                         SEQUENCE LISTING

<110>  The Regents of the University of California
 
<120>  METHODS FOR THE TREATMENT OF DANON DISEASE AND OTHER DISORDERS OF
        AUTOPHAGY

<130>  1959169-00006

<150>  US62/280,269
<151>  2016-01-19

<160>  12    

<170>  PatentIn version 3.5

<210>  1
<211>  1233
<212>  DNA
<213>  Homo sapiens

<400>  1
atggtgtgct tccgcctctt cccggttccg ggctcagggc tcgttctggt ctgcctagtc       60

ctgggagctg tgcggtctta tgcattggaa cttaatttga cagattcaga aaatgccact      120

tgcctttatg caaaatggca gatgaatttc acagttcgct atgaaactac aaataaaact      180

tataaaactg taaccatttc agaccatggc actgtgacat ataatggaag catttgtggg      240

gatgatcaga atggtcccaa aatagcagtg cagttcggac ctggcttttc ctggattgcg      300

aattttacca aggcagcatc tacttattca attgacagcg tctcattttc ctacaacact      360

ggtgataaca caacatttcc tgatgctgaa gataaaggaa ttcttactgt tgatgaactt      420

ttggccatca gaattccatt gaatgacctt tttagatgca atagtttatc aactttggaa      480

aagaatgatg ttgtccaaca ctactgggat gttcttgtac aagcttttgt ccaaaatggc      540

acagtgagca caaatgagtt cctgtgtgat aaagacaaaa cttcaacagt ggcacccacc      600

atacacacca ctgtgccatc tcctactaca acacctactc caaaggaaaa accagaagct      660

ggaacctatt cagttaataa tggcaatgat acttgtctgc tggctaccat ggggctgcag      720

ctgaacatca ctcaggataa ggttgcttca gttattaaca tcaaccccaa tacaactcac      780

tccacaggca gctgccgttc tcacactgct ctacttagac tcaatagcag caccattaag      840

tatctagact ttgtctttgc tgtgaaaaat gaaaaccgat tttatctgaa ggaagtgaac      900

atcagcatgt atttggttaa tggctccgtt ttcagcattg caaataacaa tctcagctac      960

tgggatgccc ccctgggaag ttcttatatg tgcaacaaag agcagactgt ttcagtgtct     1020

ggagcatttc agataaatac ctttgatcta agggttcagc ctttcaatgt gacacaagga     1080

aagtattcta cagctcaaga ctgcagtgca gatgacgaca acttcctagt gcccatagcg     1140

gtgggagctg ccttggcagg agtacttatt ctagtgttgc tggcttattt tattggtctc     1200

aagcaccatc atgctggata tgagcaattt tag                                  1233


<210>  2
<211>  1233
<212>  DNA
<213>  Homo sapiens

<400>  2
atggtgtgct tccgcctctt cccggttccg ggctcagggc tcgttctggt ctgcctagtc       60

ctgggagctg tgcggtctta tgcattggaa cttaatttga cagattcaga aaatgccact      120

tgcctttatg caaaatggca gatgaatttc acagttcgct atgaaactac aaataaaact      180

tataaaactg taaccatttc agaccatggc actgtgacat ataatggaag catttgtggg      240

gatgatcaga atggtcccaa aatagcagtg cagttcggac ctggcttttc ctggattgcg      300

aattttacca aggcagcatc tacttattca attgacagcg tctcattttc ctacaacact      360

ggtgataaca caacatttcc tgatgctgaa gataaaggaa ttcttactgt tgatgaactt      420

ttggccatca gaattccatt gaatgacctt tttagatgca atagtttatc aactttggaa      480

aagaatgatg ttgtccaaca ctactgggat gttcttgtac aagcttttgt ccaaaatggc      540

acagtgagca caaatgagtt cctgtgtgat aaagacaaaa cttcaacagt ggcacccacc      600

atacacacca ctgtgccatc tcctactaca acacctactc caaaggaaaa accagaagct      660

ggaacctatt cagttaataa tggcaatgat acttgtctgc tggctaccat ggggctgcag      720

ctgaacatca ctcaggataa ggttgcttca gttattaaca tcaaccccaa tacaactcac      780

tccacaggca gctgccgttc tcacactgct ctacttagac tcaatagcag caccattaag      840

tatctagact ttgtctttgc tgtgaaaaat gaaaaccgat tttatctgaa ggaagtgaac      900

atcagcatgt atttggttaa tggctccgtt ttcagcattg caaataacaa tctcagctac      960

tgggatgccc ccctgggaag ttcttatatg tgcaacaaag agcagactgt ttcagtgtct     1020

ggagcatttc agataaatac ctttgatcta agggttcagc ctttcaatgt gacacaagga     1080

aagtattcta cagcccaaga gtgttcgctg gatgatgaca ccattctaat cccaattata     1140

gttggtgctg gtctttcagg cttgattatc gttatagtga ttgcttacgt aattggcaga     1200

agaaaaagtt atgctggata tcagactctg taa                                  1233


<210>  3
<211>  1236
<212>  DNA
<213>  Homo sapiens

<400>  3
atggtgtgct tccgcctctt cccggttccg ggctcagggc tcgttctggt ctgcctagtc       60

ctgggagctg tgcggtctta tgcattggaa cttaatttga cagattcaga aaatgccact      120

tgcctttatg caaaatggca gatgaatttc acagttcgct atgaaactac aaataaaact      180

tataaaactg taaccatttc agaccatggc actgtgacat ataatggaag catttgtggg      240

gatgatcaga atggtctcaa aatagcagtg cagttcggac ctggcttttc ctggattgcg      300

aattttacca aggcagcatc tacttattca attgacagcg tctcattttc ctacaacact      360

ggtgataaca caacatttcc tgatgctgaa gataaaggaa ttcttactgt tgatgaactt      420

ttggccatca gaattccatt gaatgacctt tttagatgca atagtttatc aactttggaa      480

aagaatgatg ttgtccaaca ctactgggat gttcttgtac aagcttttgt ccaaaatggc      540

acagtgagca caaatgagtt cctgtgtgat aaagacaaaa cttcaacagt ggcacccacc      600

atacacacca ctgtgccatc tcctactaca acacctactc caaaggaaaa accagaagct      660

ggaacctatt cagttaataa tggcaatgat acttgtctgc tggctaccat ggggctgcag      720

ctgaacatca ctcaggataa ggttgcttca gttattaaca tcaaccccaa tacaactcac      780

tccacaggca gctgccgttc tcacactgct ctacttagac tcaatagcag caccattaag      840

tatctagact ttgtctttgc tgtgaaaaat gaaaaccgat tttatctgaa ggaagtgaac      900

atcagcatgt atttggttaa tggctccgtt ttcagcattg caaataacaa tctcagctac      960

tgggatgccc ccctgggaag ttcttatatg tgcaacaaag agcagactgt ttcagtgtct     1020

ggagcatttc agataaatac ctttgatcta agggttcagc ctttcaatgt gacacaagga     1080

aagtattcta cagctgaaga atgttctgct gactctgacc tcaactttct tattcctgtt     1140

gcagtgggtg tggccttggg cttccttata attgttgtct ttatctctta tatgattgga     1200

agaaggaaaa gtcgtactgg ttatcagtct gtgtaa                               1236


<210>  4
<211>  90
<212>  PRT
<213>  Homo sapiens

<400>  4

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
1               5                   10                  15      


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Gln Asp Cys 
        35                  40                  45              


Ser Ala Asp Asp Asp Asn Phe Leu Val Pro Ile Ala Val Gly Ala Ala 
    50                  55                  60                  


Leu Ala Gly Val Leu Ile Leu Val Leu Leu Ala Tyr Phe Ile Gly Leu 
65                  70                  75                  80  


Lys His His His Ala Gly Tyr Glu Gln Phe 
                85                  90  


<210>  5
<211>  90
<212>  PRT
<213>  Mus musculus

<400>  5

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Val 
1               5                   10                  15      


Leu Ser Val Ser Arg Ala Phe Gln Ile Asn Thr Phe Asn Leu Lys Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Lys Gly Gln Tyr Ser Thr Ala Gln Asp Cys 
        35                  40                  45              


Ser Ala Asp Glu Asp Asn Phe Leu Val Pro Ile Ala Val Gly Ala Ala 
    50                  55                  60                  


Leu Gly Gly Val Leu Ile Leu Val Leu Leu Ala Tyr Phe Ile Gly Leu 
65                  70                  75                  80  


Lys Arg His His Thr Gly Tyr Glu Gln Phe 
                85                  90  


<210>  6
<211>  90
<212>  PRT
<213>  Homo sapiens

<400>  6

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
1               5                   10                  15      


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Gln Glu Cys 
        35                  40                  45              


Ser Leu Asp Asp Asp Thr Ile Leu Ile Pro Ile Ile Val Gly Ala Gly 
    50                  55                  60                  


Leu Ser Gly Leu Ile Ile Val Ile Val Ile Ala Tyr Val Ile Gly Arg 
65                  70                  75                  80  


Arg Lys Ser Tyr Ala Gly Tyr Gln Thr Leu 
                85                  90  


<210>  7
<211>  90
<212>  PRT
<213>  Mus musculus

<400>  7

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Val 
1               5                   10                  15      


Leu Ser Val Ser Arg Ala Phe Gln Ile Asn Thr Phe Asn Leu Lys Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Lys Gly Gln Tyr Ser Thr Ala Gln Glu Cys 
        35                  40                  45              


Ser Leu Asp Asp Asp Thr Ile Leu Ile Pro Ile Ile Val Gly Ala Gly 
    50                  55                  60                  


Leu Ser Gly Leu Ile Ile Val Ile Val Ile Ala Tyr Leu Ile Gly Arg 
65                  70                  75                  80  


Arg Lys Thr Tyr Ala Gly Tyr Gln Thr Leu 
                85                  90  


<210>  8
<211>  91
<212>  PRT
<213>  Homo sapiens

<400>  8

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
1               5                   10                  15      


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Glu Glu Cys 
        35                  40                  45              


Ser Ala Asp Ser Asp Leu Asn Phe Leu Ile Pro Val Ala Val Gly Val 
    50                  55                  60                  


Ala Leu Gly Phe Leu Ile Ile Val Val Phe Ile Ser Tyr Met Ile Gly 
65                  70                  75                  80  


Arg Arg Lys Ser Arg Thr Gly Tyr Gln Ser Val 
                85                  90      


<210>  9
<211>  91
<212>  PRT
<213>  Mus musculus

<400>  9

Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Val 
1               5                   10                  15      


Leu Ser Val Ser Arg Ala Phe Gln Ile Asn Thr Phe Asn Leu Lys Val 
            20                  25                  30          


Gln Pro Phe Asn Val Thr Lys Gly Gln Tyr Ser Thr Ala Glu Glu Cys 
        35                  40                  45              


Ala Ala Asp Ser Asp Leu Asn Phe Leu Ile Pro Val Ala Val Gly Val 
    50                  55                  60                  


Ala Leu Gly Phe Leu Ile Ile Ala Val Phe Ile Ser Tyr Met Ile Gly 
65                  70                  75                  80  


Arg Arg Lys Ser Arg Thr Gly Tyr Gln Ser Val 
                85                  90      


<210>  10
<211>  410
<212>  PRT
<213>  Homo sapiens

<400>  10

Met Val Cys Phe Arg Leu Phe Pro Val Pro Gly Ser Gly Leu Val Leu 
1               5                   10                  15      


Val Cys Leu Val Leu Gly Ala Val Arg Ser Tyr Ala Leu Glu Leu Asn 
            20                  25                  30          


Leu Thr Asp Ser Glu Asn Ala Thr Cys Leu Tyr Ala Lys Trp Gln Met 
        35                  40                  45              


Asn Phe Thr Val Arg Tyr Glu Thr Thr Asn Lys Thr Tyr Lys Thr Val 
    50                  55                  60                  


Thr Ile Ser Asp His Gly Thr Val Thr Tyr Asn Gly Ser Ile Cys Gly 
65                  70                  75                  80  


Asp Asp Gln Asn Gly Pro Lys Ile Ala Val Gln Phe Gly Pro Gly Phe 
                85                  90                  95      


Ser Trp Ile Ala Asn Phe Thr Lys Ala Ala Ser Thr Tyr Ser Ile Asp 
            100                 105                 110         


Ser Val Ser Phe Ser Tyr Asn Thr Gly Asp Asn Thr Thr Phe Pro Asp 
        115                 120                 125             


Ala Glu Asp Lys Gly Ile Leu Thr Val Asp Glu Leu Leu Ala Ile Arg 
    130                 135                 140                 


Ile Pro Leu Asn Asp Leu Phe Arg Cys Asn Ser Leu Ser Thr Leu Glu 
145                 150                 155                 160 


Lys Asn Asp Val Val Gln His Tyr Trp Asp Val Leu Val Gln Ala Phe 
                165                 170                 175     


Val Gln Asn Gly Thr Val Ser Thr Asn Glu Phe Leu Cys Asp Lys Asp 
            180                 185                 190         


Lys Thr Ser Thr Val Ala Pro Thr Ile His Thr Thr Val Pro Ser Pro 
        195                 200                 205             


Thr Thr Thr Pro Thr Pro Lys Glu Lys Pro Glu Ala Gly Thr Tyr Ser 
    210                 215                 220                 


Val Asn Asn Gly Asn Asp Thr Cys Leu Leu Ala Thr Met Gly Leu Gln 
225                 230                 235                 240 


Leu Asn Ile Thr Gln Asp Lys Val Ala Ser Val Ile Asn Ile Asn Pro 
                245                 250                 255     


Asn Thr Thr His Ser Thr Gly Ser Cys Arg Ser His Thr Ala Leu Leu 
            260                 265                 270         


Arg Leu Asn Ser Ser Thr Ile Lys Tyr Leu Asp Phe Val Phe Ala Val 
        275                 280                 285             


Lys Asn Glu Asn Arg Phe Tyr Leu Lys Glu Val Asn Ile Ser Met Tyr 
    290                 295                 300                 


Leu Val Asn Gly Ser Val Phe Ser Ile Ala Asn Asn Asn Leu Ser Tyr 
305                 310                 315                 320 


Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
                325                 330                 335     


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            340                 345                 350         


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Gln Asp Cys 
        355                 360                 365             


Ser Ala Asp Asp Asp Asn Phe Leu Val Pro Ile Ala Val Gly Ala Ala 
    370                 375                 380                 


Leu Ala Gly Val Leu Ile Leu Val Leu Leu Ala Tyr Phe Ile Gly Leu 
385                 390                 395                 400 


Lys His His His Ala Gly Tyr Glu Gln Phe 
                405                 410 


<210>  11
<211>  410
<212>  PRT
<213>  Homo sapiens

<400>  11

Met Val Cys Phe Arg Leu Phe Pro Val Pro Gly Ser Gly Leu Val Leu 
1               5                   10                  15      


Val Cys Leu Val Leu Gly Ala Val Arg Ser Tyr Ala Leu Glu Leu Asn 
            20                  25                  30          


Leu Thr Asp Ser Glu Asn Ala Thr Cys Leu Tyr Ala Lys Trp Gln Met 
        35                  40                  45              


Asn Phe Thr Val Arg Tyr Glu Thr Thr Asn Lys Thr Tyr Lys Thr Val 
    50                  55                  60                  


Thr Ile Ser Asp His Gly Thr Val Thr Tyr Asn Gly Ser Ile Cys Gly 
65                  70                  75                  80  


Asp Asp Gln Asn Gly Pro Lys Ile Ala Val Gln Phe Gly Pro Gly Phe 
                85                  90                  95      


Ser Trp Ile Ala Asn Phe Thr Lys Ala Ala Ser Thr Tyr Ser Ile Asp 
            100                 105                 110         


Ser Val Ser Phe Ser Tyr Asn Thr Gly Asp Asn Thr Thr Phe Pro Asp 
        115                 120                 125             


Ala Glu Asp Lys Gly Ile Leu Thr Val Asp Glu Leu Leu Ala Ile Arg 
    130                 135                 140                 


Ile Pro Leu Asn Asp Leu Phe Arg Cys Asn Ser Leu Ser Thr Leu Glu 
145                 150                 155                 160 


Lys Asn Asp Val Val Gln His Tyr Trp Asp Val Leu Val Gln Ala Phe 
                165                 170                 175     


Val Gln Asn Gly Thr Val Ser Thr Asn Glu Phe Leu Cys Asp Lys Asp 
            180                 185                 190         


Lys Thr Ser Thr Val Ala Pro Thr Ile His Thr Thr Val Pro Ser Pro 
        195                 200                 205             


Thr Thr Thr Pro Thr Pro Lys Glu Lys Pro Glu Ala Gly Thr Tyr Ser 
    210                 215                 220                 


Val Asn Asn Gly Asn Asp Thr Cys Leu Leu Ala Thr Met Gly Leu Gln 
225                 230                 235                 240 


Leu Asn Ile Thr Gln Asp Lys Val Ala Ser Val Ile Asn Ile Asn Pro 
                245                 250                 255     


Asn Thr Thr His Ser Thr Gly Ser Cys Arg Ser His Thr Ala Leu Leu 
            260                 265                 270         


Arg Leu Asn Ser Ser Thr Ile Lys Tyr Leu Asp Phe Val Phe Ala Val 
        275                 280                 285             


Lys Asn Glu Asn Arg Phe Tyr Leu Lys Glu Val Asn Ile Ser Met Tyr 
    290                 295                 300                 


Leu Val Asn Gly Ser Val Phe Ser Ile Ala Asn Asn Asn Leu Ser Tyr 
305                 310                 315                 320 


Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
                325                 330                 335     


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            340                 345                 350         


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Gln Glu Cys 
        355                 360                 365             


Ser Leu Asp Asp Asp Thr Ile Leu Ile Pro Ile Ile Val Gly Ala Gly 
    370                 375                 380                 


Leu Ser Gly Leu Ile Ile Val Ile Val Ile Ala Tyr Val Ile Gly Arg 
385                 390                 395                 400 


Arg Lys Ser Tyr Ala Gly Tyr Gln Thr Leu 
                405                 410 


<210>  12
<211>  411
<212>  PRT
<213>  Homo sapiens

<400>  12

Met Val Cys Phe Arg Leu Phe Pro Val Pro Gly Ser Gly Leu Val Leu 
1               5                   10                  15      


Val Cys Leu Val Leu Gly Ala Val Arg Ser Tyr Ala Leu Glu Leu Asn 
            20                  25                  30          


Leu Thr Asp Ser Glu Asn Ala Thr Cys Leu Tyr Ala Lys Trp Gln Met 
        35                  40                  45              


Asn Phe Thr Val Arg Tyr Glu Thr Thr Asn Lys Thr Tyr Lys Thr Val 
    50                  55                  60                  


Thr Ile Ser Asp His Gly Thr Val Thr Tyr Asn Gly Ser Ile Cys Gly 
65                  70                  75                  80  


Asp Asp Gln Asn Gly Pro Lys Ile Ala Val Gln Phe Gly Pro Gly Phe 
                85                  90                  95      


Ser Trp Ile Ala Asn Phe Thr Lys Ala Ala Ser Thr Tyr Ser Ile Asp 
            100                 105                 110         


Ser Val Ser Phe Ser Tyr Asn Thr Gly Asp Asn Thr Thr Phe Pro Asp 
        115                 120                 125             


Ala Glu Asp Lys Gly Ile Leu Thr Val Asp Glu Leu Leu Ala Ile Arg 
    130                 135                 140                 


Ile Pro Leu Asn Asp Leu Phe Arg Cys Asn Ser Leu Ser Thr Leu Glu 
145                 150                 155                 160 


Lys Asn Asp Val Val Gln His Tyr Trp Asp Val Leu Val Gln Ala Phe 
                165                 170                 175     


Val Gln Asn Gly Thr Val Ser Thr Asn Glu Phe Leu Cys Asp Lys Asp 
            180                 185                 190         


Lys Thr Ser Thr Val Ala Pro Thr Ile His Thr Thr Val Pro Ser Pro 
        195                 200                 205             


Thr Thr Thr Pro Thr Pro Lys Glu Lys Pro Glu Ala Gly Thr Tyr Ser 
    210                 215                 220                 


Val Asn Asn Gly Asn Asp Thr Cys Leu Leu Ala Thr Met Gly Leu Gln 
225                 230                 235                 240 


Leu Asn Ile Thr Gln Asp Lys Val Ala Ser Val Ile Asn Ile Asn Pro 
                245                 250                 255     


Asn Thr Thr His Ser Thr Gly Ser Cys Arg Ser His Thr Ala Leu Leu 
            260                 265                 270         


Arg Leu Asn Ser Ser Thr Ile Lys Tyr Leu Asp Phe Val Phe Ala Val 
        275                 280                 285             


Lys Asn Glu Asn Arg Phe Tyr Leu Lys Glu Val Asn Ile Ser Met Tyr 
    290                 295                 300                 


Leu Val Asn Gly Ser Val Phe Ser Ile Ala Asn Asn Asn Leu Ser Tyr 
305                 310                 315                 320 


Trp Asp Ala Pro Leu Gly Ser Ser Tyr Met Cys Asn Lys Glu Gln Thr 
                325                 330                 335     


Val Ser Val Ser Gly Ala Phe Gln Ile Asn Thr Phe Asp Leu Arg Val 
            340                 345                 350         


Gln Pro Phe Asn Val Thr Gln Gly Lys Tyr Ser Thr Ala Glu Glu Cys 
        355                 360                 365             


Ser Ala Asp Ser Asp Leu Asn Phe Leu Ile Pro Val Ala Val Gly Val 
    370                 375                 380                 


Ala Leu Gly Phe Leu Ile Ile Val Val Phe Ile Ser Tyr Met Ile Gly 
385                 390                 395                 400 


Arg Arg Lys Ser Arg Thr Gly Tyr Gln Ser Val 
                405                 410     


