                         SEQUENCE LISTING

<110>  THE BROAD INSTITUTE, INC.
       THE BRIGHAM & WOMEN'S HOSPITAL, INC.
       DANA-FARBER CANCER INSTITUTE, INC.
 
<120>  AGENTS FOR REVERSING TOXIC PROTEINOPATHIES

<130>  52199-532001WO

<150>  62/865,096
<151>  2019-06-21

<150>  62/878,304
<151>  2019-07-24

<160>  13    

<170>  PatentIn version 3.5

<210>  1
<211>  3607
<212>  DNA
<213>  Homo sapiens

<400>  1
atgacaccgg gcacccagtc tcctttcttc ctgctgctgc tcctcacagt gcttacagtt       60

gttacgggtt ctggtcatgc aagctctacc ccaggtggag aaaaggagac ttcggctacc      120

cagagaagtt cagtgcccag ctctactgag aagaatgctg tgagtatgac cagcagcgta      180

ctctccagcc acagccccgg ttcaggctcc tccaccactc agggacagga tgtcactctg      240

gccccggcca cggaaccagc ttcaggttca gctgccacct ggggacagga tgtcacctcg      300

gtcccagtca ccaggccagc cctgggctcc accaccccac cagcccacga tgtcacctca      360

gccccggaca acaagccagc cccgggctcc accgcccccc ccagcccacg gtgtcacctc      420

ggccccggac accaggccgg ccccgggctc caccgccccc caagcccacg gtgtcacctc      480

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc      540

ggccccggac accaggcccg ccccgggctc caccgccccc ccagcccacg gtgtcacctc      600

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc      660

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc      720

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc      780

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc      840

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc      900

ggccccggag agcaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc      960

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1020

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1080

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1140

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1200

ggccccggag agcaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1260

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc     1320

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc     1380

ggccccggag agcaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1440

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1500

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1560

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1620

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1680

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1740

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1800

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     1860

ggccccggac accaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc     1920

ggccccggag agcaggccgg ccccgggctc caccgcgccc gcagcccacg gtgtcacctc     1980

ggccccggag agcaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2040

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2100

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2160

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2220

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2280

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2340

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2400

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2460

ggccccggac accaggccgg ccccgggctc caccgccccc ccagcccacg gtgtcacctc     2520

ggccccggac accaggcggg ccccgggctc caccccggcc ccgggctcca ccgccccccc     2580

agcccacggt gtcaccttgg ccccggacac caggccggcc ccgggctcca ccgccccccc     2640

agcccatggt gtcacctcgg ccccggacaa caggcccgcc ttgggctcca ccgcccctcc     2700

agtccacaat gtcacctcgg cctcaggctc tgcatcaggc tcagcttcta ctctggtgca     2760

caacggcacc tctgccaggg ctaccacaac cccagccagc aagagcactc cattctcaat     2820

tcccagccac cactctgata ctcctaccac ccttgccagc catagcacca agactgatgc     2880

cagtagcact caccatagct cggtacctcc tctcacctcc tccaatcaca gcacttctcc     2940

ccagttgtct actggggtct ctttcttttt cctgtctttt cacatttcaa acctccagtt     3000

taattcctct ctggaagatc ccagcaccga ctactaccaa gagctgcaga gagacatttc     3060

tgaaatgttt ttgcagattt ataaacaagg gggttttctg ggcctctcca atattaagtt     3120

caggccagga tctgtggtgg tacaattgac tctggccttc cgagaaggta ccatcaatgt     3180

gcacgacgtg gagacacagt tcaatcagta taaaacggaa gcagcctctc gatataacct     3240

gacgatctca gacgtcagcg tgagtgatgt gccatttcct ttctctgccc agtctggggc     3300

tggggtgcca ggctggggca tcgcgctgct ggtgctggtc tgtgttctgg ttgcgctggc     3360

cattgtctat ctcattgcct tggctgtctg tcagtgccgc cgaaagaact acgggcagct     3420

ggacatcttt ccagcccggg atacctacca tcctatgagc gagtacccca cctaccacac     3480

ccatgggcgc tatgtgcccc ctagcagtac cgatcgtagc ccctatgaga aggtttctgc     3540

aggtaacggt ggcagcagcc tctcttacac aaacccagca gtggcagcca cttctgccaa     3600

cttgtag                                                               3607


<210>  2
<211>  1923
<212>  DNA
<213>  Homo sapiens

<400>  2
atggggcagc catctctgac ttggatgctg atggtggtgg tggcctcttg gttcatcaca       60

actgcagcca ctgacacctc agaagcaaga tggtgctctg aatgtcacag caatgccacc      120

tgcacggagg atgaggccgt tacgacgtgc acctgtcagg agggcttcac cggcgatggc      180

ctgacctgcg tggacctgga tgagtgcgcc attcctggag ctcacaactg ctccgccaac      240

agcagctgcg taaacacgcc aggctccttc tcctgcgtct gccccgaagg cttccgcctg      300

tcgcccggtc tcggctgcac agacgtggat gagtgcgctg agcctgggct tagccactgc      360

cacgccctgg ccacacgtgt caatgtggtg ggcagctact tgtgcgtatg ccccgcgggc      420

taccgggggg atggatggca ctgtgagtgc tccccgggct cctgcgggcc ggggttggac      480

tgcgtgcccg agggcgacgc gctcgtgtgc gcggatccgt gccaggcgca ccgcaccctg      540

gacgagtact ggcgcagcac cgagtacggg gagggctacg cctgcgacac ggacctgcgc      600

ggctggtacc gcttcgtggg ccagggcggt gcgcgcatgg ccgagacctg cgtgccagtc      660

ctgcgctgca acacggccgc ccccatgtgg ctcaatggca cgcatccgtc cagcgacgag      720

ggcatcgtga gccgcaaggc ctgcgcgcac tggagcggcc actgctgcct gtgggatgcg      780

tccgtccagg tgaaggcctg tgccggcggc tactacgtct acaacctgac agcgcccccc      840

gagtgtcacc tggcgtactg cacagacccc agctccgtgg aggggacgtg tgaggagtgc      900

agtatagacg aggactgcaa atcgaataat ggcagatggc actgccagtg caaacaggac      960

ttcaacatca ctgatatctc cctcctggag cacaggctgg aatgtggggc caatgacatg     1020

aaggtgtcgc tgggcaagtg ccagctgaag agtctgggct tcgacaaggt cttcatgtac     1080

ctgagtgaca gccggtgctc gggcttcaat gacagagaca accgggactg ggtgtctgta     1140

gtgaccccag cccgggatgg cccctgtggg acagtgttga cgaggaatga aacccatgcc     1200

acttacagca acaccctcta cctggcagat gagatcatca tccgtgacct caacatcaaa     1260

atcaactttg catgctccta ccccctggac atgaaagtca gcctgaagac cgccctacag     1320

ccaatggtca gtgctctaaa catcagagtg ggcgggaccg gcatgttcac cgtgcggatg     1380

gcgctcttcc agaccccttc ctacacgcag ccctaccaag gctcctccgt gacactgtcc     1440

actgaggctt ttctctacgt gggcaccatg ttggatgggg gcgacctgtc ccgatttgca     1500

ctgctcatga ccaactgcta tgccacaccc agtagcaatg ccacggaccc cctgaagtac     1560

ttcatcatcc aggacagatg cccacacact agagactcaa ctatccaagt ggtggagaat     1620

ggggagtcct cccagggccg attttccgtc cagatgttcc ggtttgctgg aaactatgac     1680

ctagtctacc tgcactgtga agtctatctc tgtgacacca tgaatgaaaa gtgcaagcct     1740

acctgctctg ggaccagatt ccgaagtggg agtgtcatag atcaatcccg tgtcctgaac     1800

ttgggtccca tcacacggaa aggtgtccag gccacagtct caagggcttt tagcagcttg     1860

gggctcctga aagtctggct gcctctgctt ctctcggcca ccttgaccct gacttttcag     1920

tga                                                                   1923


<210>  3
<211>  2768
<212>  DNA
<213>  Homo sapiens

<400>  3
agagtcatcc agctggagcc ctgagtggct gagctcaggc cttcgcagca ttcttgggtg       60

ggagcagcca cgggtcagcc acaagggcca cagccatgaa tggcacagaa ggccctaact      120

tctacgtgcc cttctccaat gcgacgggtg tggtacgcag ccacttcgag tacccacagt      180

actacctggc tgagccatgg cagttctcca tgctggccgc ctacatgttt ctgctgatcg      240

tgctgggctt ccccatcaac ttcctcacgc tctacgtcac cgtccagcac aagaagctgc      300

gcacgcctct caactacatc ctgctcaacc tagccgtggc tgacctcttc atggtcctag      360

gtggcttcac cagcaccctc tacacctctc tgcatggata cttcgtcttc gggcccacag      420

gatgcaattt ggagggcttc tttgccaccc tgggcggtga aattgccctg tggtccttgg      480

tggtcctggc catcgagcgg tacgtggtgg tgtgtaagcc catgagcaac ttccgcttcg      540

gggagaacca tgccatcatg ggcgttgcct tcacctgggt catggcgctg gcctgcgccg      600

cacccccact cgccggctgg tccaggtaca tccccgaggg cctgcagtgc tcgtgtggaa      660

tcgactacta cacgctcaag ccggaggtca acaacgagtc ttttgtcatc tacatgttcg      720

tggtccactt caccatcccc atgattatca tctttttctg ctatgggcag ctcgtcttca      780

ccgtcaagga ggccgctgcc cagcagcagg agtcagccac cacacagaag gcagagaagg      840

aggtcacccg catggtcatc atcatggtca tcgctttcct gatctgctgg gtgccctacg      900

ccagcgtggc attctacatc ttcacccacc agggctccaa cttcggtccc atcttcatga      960

ccatcccagc gttctttgcc aagagcgccg ccatctacaa ccctgtcatc tatatcatga     1020

tgaacaagca gttccggaac tgcatgctca ccaccatctg ctgcggcaag aacccactgg     1080

gtgacgatga ggcctctgct accgtgtcca agacggagac gagccaggtg gccccggcct     1140

aagacctgcc taggactctg tggccgacta taggcgtctc ccatccccta caccttcccc     1200

cagccacagc catcccacca ggagcagcgc ctgtgcagaa tgaacgaagt cacataggct     1260

ccttaatttt tttttttttt ttaagaaata attaatgagg ctcctcactc acctgggaca     1320

gcctgagaag ggacatccac caagacctac tgatctggag tcccacgttc cccaaggcca     1380

gcgggatgtg tgcccctcct cctcccaact catctttcag gaacacgagg attcttgctt     1440

tctggaaaag tgtcccagct tagggataag tgtctagcac agaatggggc acacagtagg     1500

tgcttaataa atgctggatg gatgcaggaa ggaatggagg aatgaatggg aagggagaac     1560

atatctatcc tctcagaccc tcgcagcagc agcaactcat acttggctaa tgatatggag     1620

cagttgtttt tccctccctg ggcctcactt tcttctccta taaaatggaa atcccagatc     1680

cctggtcctg ccgacacgca gctactgaga agaccaaaag aggtgtgtgt gtgtctatgt     1740

gtgtgtttca gcactttgta aatagcaaga agctgtacag attctagtta atgttgtgaa     1800

taacatcaat taatgtaact agttaattac tatgattatc acctcctgat agtgaacatt     1860

ttgagattgg gcattcagat gatggggttt cacccaacct tggggcaggt ttttaaaaat     1920

tagctaggca tcaaggccag accagggctg ggggttgggc tgtaggcagg gacagtcaca     1980

ggaatgcaga atgcagtcat cagacctgaa aaaacaacac tgggggaggg ggacggtgaa     2040

ggccaagttc ccaatgaggg tgagattggg cctggggtct cacccctagt gtggggcccc     2100

aggtcccgtg cctccccttc ccaatgtggc ctatggagag acaggccttt ctctcagcct     2160

ctggaagcca cctgctcttt tgctctagca cctgggtccc agcatctaga gcatggagcc     2220

tctagaagcc atgctcaccc gcccacattt aattaacagc tgagtccctg atgtcatcct     2280

tatctcgaag agcttagaaa caaagagtgg gaaattccac tgggcctacc ttccttgggg     2340

atgttcatgg gccccagttt ccagtttccc ttgccagaca agcccatctt cagcagttgc     2400

tagtccattc tccattctgg agaatctgct ccaaaaagct ggccacatct ctgaggtgtc     2460

agaattaagc tgcctcagta actgctcccc cttctccata taagcaaagc cagaagctct     2520

agctttaccc agctctgcct ggagactaag gcaaattggg ccattaaaag ctcagctcct     2580

atgttggtat taacggtggt gggttttgtt gctttcacac tctatccaca ggatagattg     2640

aaactgccag cttccacctg atccctgacc ctgggatggc tggattgagc aatgagcaga     2700

gccaagcagc acagagtccc ctggggctag aggtggagga ggcagtcctg ggaatgggaa     2760

aaacccca                                                              2768


<210>  4
<211>  2546
<212>  DNA
<213>  Homo sapiens

<400>  4
aggtggagca agatggctgt ggagctgggc gtgctgctcg tccggccccg gcccggaacc       60

gggctgggta gagtgatgcg gaccctcctg ctggtgctgt ggctggcgac gcgcggaagc      120

gcgctctact ttcacatcgg agagacggag aagaagtgct ttattgagga gatcccggac      180

gagaccatgg tcataggaaa ctaccggacg cagctgtatg acaagcagcg ggaggagtac      240

cagccggcca ccccggggct tggcatgttt gtggaggtga aggacccaga ggacaaggtc      300

atcctggccc ggcagtatgg ctccgagggc aggttcactt tcacttccca tacccctggt      360

gagcaccaga tctgtcttca ctccaattcc accaagttct ccctctttgc tggaggcatg      420

ctgagagttc acctggacat ccaggtaggt gaacatgcca atgactatgc agaaattgct      480

gctaaagaca agttgagtga gttgcagcta cgagtgcgac agctggtgga acaagtggag      540

cagatccaga aagagcagaa ctaccagcgg tggcgagagg agcgcttccg gcagaccagt      600

gagagcacca accagcgggt gctgtggtgg tccattctgc agaccctcat cctcgtggcc      660

atcggtgtct ggcagatgcg gcacctcaag agcttctttg aagccaagaa gcttgtgtag      720

ctgtcccagg cgtcacaacc catcctccca ggctggggga gaaaggacct cctggaactg      780

acttcttctg tcaggaggac tggtttccag ccatacctgt tctggaaggg agaggggctg      840

gaggcaccca caggcacaag ctgaaggcag cagcttggct aatactgagc aggtagtggg      900

gcaaattcct gccctctctc tctggcctct gggccgtttg gtagtaatca cccaagggct      960

ggtaaagccc ctcctcttgg cacctcagaa tcacagtgtt actgatcagg gatgtgaggc     1020

tgctgttggg ggtgggggga ggggaatggg caggcaagcc agtcttctgt cttcctttgc     1080

taacttaggg ttttgagcag gttggggtat ggtgcctgtc atacccacct gccaccctgg     1140

gaacctcact gttctctctt tcagcctaga cctgctgatc cagggtgtgt gtgagttgag     1200

ggtgggtgga ggggtttgca gtgtgggaat gtggccctgc agttgacctg agctgcttca     1260

catggttgtc cattctgggg cttaaagaac tgggaccaga ccaagtagag gccttggtgc     1320

tggttggggt ggggcctgca gagtcttagt tactgatttc attttcaata aatgtaggtt     1380

tgttacatga gtttcccaat aaaaaaaaaa atgacttctt gtccagtgca agtgactcag     1440

tcatcagtgg gcacacactg cagggtgcct cagggaatgc cagttcttcc aaagagcaaa     1500

gcacttcaca ttccaaagtg aattcccacc agtcagcttc attctttcct tcttctccag     1560

gccttcctgt ggcagggaat agtgggtttg tccaagatta tacaacaagt aaattgggct     1620

ggggctcaaa tttacaccct ttcctctgtg ccagctccct ggtgaagttc cctctttcta     1680

gagtcagtaa gcaggattgt catggatgct gccaggaagt gcctggtaag gaggtgcatt     1740

gagcagggga gtgctacagg acagccaccc tgggctggca gggacaagga tgttgatggg     1800

ctaaaccaac agcaagtgat ttcaaccagg accatgaagg agaggaagga ttctgctgga     1860

aggagatggc aggacagggg tggttggaga agtggaggca aacagctgga atggaggtgg     1920

gtgggtgttt aatttcagct gcagagggtg ttgtgaggaa gctggaaagg aaggttggat     1980

tagagaagcc tcgagctcca ggtaagcgat ttggacatgc ccacctttca agaggggctg     2040

caggcaccca caggcacaag ctgaaggcag cagcttggct ggcttaatac tgagcaggtg     2100

gtggggtaaa tgcctgcccc cctccctctg gcctctgggc cctttgcagt aatcacccag     2160

ggtctggtaa agccactgag agccctactg gcacctcaga atcacagtgt tattgatcag     2220

ggatgtgagg ctgctgttgg gggttggggg aggcaaatgg gcaggcagtt ttgagaagaa     2280

ccttctaata agaaatgtga gggaggttac agcagtgtgt gagaaagacc aggaagaagg     2340

agacaagttt gggggctgct tcccctaatg ggatgatgca atctgggctc atgctgccaa     2400

ctaattcttc cacatgaaaa aaaaaagttt tttggcgggc acggtggttc acacctgtaa     2460

tcccagcact ttgggaggct gaggtaggtg gatggcctga ggccaggagt ttgagaccag     2520

cctggccaac atggtgaaac ctcatc                                          2546


<210>  5
<211>  235
<212>  PRT
<213>  Homo sapiens

<400>  5

Met Ala Val Glu Leu Gly Val Leu Leu Val Arg Pro Arg Pro Gly Thr 
1               5                   10                  15      


Gly Leu Gly Arg Val Met Arg Thr Leu Leu Leu Val Leu Trp Leu Ala 
            20                  25                  30          


Thr Arg Gly Ser Ala Leu Tyr Phe His Ile Gly Glu Thr Glu Lys Lys 
        35                  40                  45              


Cys Phe Ile Glu Glu Ile Pro Asp Glu Thr Met Val Ile Gly Asn Tyr 
    50                  55                  60                  


Arg Thr Gln Leu Tyr Asp Lys Gln Arg Glu Glu Tyr Gln Pro Ala Thr 
65                  70                  75                  80  


Pro Gly Leu Gly Met Phe Val Glu Val Lys Asp Pro Glu Asp Lys Val 
                85                  90                  95      


Ile Leu Ala Arg Gln Tyr Gly Ser Glu Gly Arg Phe Thr Phe Thr Ser 
            100                 105                 110         


His Thr Pro Gly Glu His Gln Ile Cys Leu His Ser Asn Ser Thr Lys 
        115                 120                 125             


Phe Ser Leu Phe Ala Gly Gly Met Leu Arg Val His Leu Asp Ile Gln 
    130                 135                 140                 


Val Gly Glu His Ala Asn Asp Tyr Ala Glu Ile Ala Ala Lys Asp Lys 
145                 150                 155                 160 


Leu Ser Glu Leu Gln Leu Arg Val Arg Gln Leu Val Glu Gln Val Glu 
                165                 170                 175     


Gln Ile Gln Lys Glu Gln Asn Tyr Gln Arg Trp Arg Glu Glu Arg Phe 
            180                 185                 190         


Arg Gln Thr Ser Glu Ser Thr Asn Gln Arg Val Leu Trp Trp Ser Ile 
        195                 200                 205             


Leu Gln Thr Leu Ile Leu Val Ala Ile Gly Val Trp Gln Met Arg His 
    210                 215                 220                 


Leu Lys Ser Phe Phe Glu Ala Lys Lys Leu Val 
225                 230                 235 


<210>  6
<211>  27
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  6
ggcagagaaa ggaaatggca catcact                                           27


<210>  7
<211>  27
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  7
ctgctgctcc tcacagtgct tacaggt                                           27


<210>  8
<211>  20
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  8
gctaccacag cccctaaacc                                                   20


<210>  9
<211>  19
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  9
gctgtggctg gagagtacg                                                    19


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

<220>
<223>  synthetic

<400>  10
ccagccatag caccaagact                                                   20


<210>  11
<211>  20
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  11
ggaaggaaag gccgatactc                                                   20


<210>  12
<211>  22
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  12
ttacgagaga aaactcatgg cc                                                22


<210>  13
<211>  22
<212>  DNA
<213>  Artificial

<220>
<223>  synthetic

<400>  13
gggtccaagt tgtccagaat gc                                                22


