                               SEQUENCE LISTING

<110> SANGAMO THERAPEUTICS, INC.
      SHIRE HUMAN GENETIC THERAPIES, INC.
 
<120> HTT REPRESSORS AND USES THEREOF

<130> 8327-018740

<140>
<141>

<150> 62/792,701
<151> 2019-01-15

<160> 23    

<170> PatentIn version 3.5

<210> 1
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 1
Gln Ser Gly Asp Leu Thr Arg 
1               5           


<210> 2
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 2
Lys His Gly Asn Leu Ser Glu 
1               5           


<210> 3
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 3
Lys Arg Cys Asn Leu Arg Cys 
1               5           


<210> 4
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 4
Cys Pro Ser His Leu Thr Arg 
1               5           


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

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 5
Arg Gln Phe Asn Arg His Gln 
1               5           


<210> 6
<211> 28
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      Human or mouse HTT sequence

<400> 6
agcagcagca gcagcagcag cagcagca                                          28


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

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 7
Ser Pro Glu Gln Leu Ser Arg 
1               5           


<210> 8
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 8
Gln Trp Ser Thr Arg Lys Arg 
1               5           


<210> 9
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 9
Lys Gln Gly Asn Leu Val Glu 
1               5           


<210> 10
<211> 7
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      peptide

<400> 10
Arg Ser Asp Asn Leu Ser Glu 
1               5           


<210> 11
<211> 28
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      Human or mouse HTT sequence

<400> 11
cagcagcagc agcagcagca gcagcagc                                          28


<210> 12
<211> 21
<212> DNA
<213> Unknown

<220>
<223> Description of Unknown: 
      Human or mouse HTT sequence

<400> 12
cagcagcagc agcagcagca g                                                 21


<210> 13
<211> 825
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 13
atggccccca agaaaaagcg gaaagtgggc atccacgggg tacccgccgc tatggctgag       60

aggcccttcc agtgtcgaat ctgcatgcgt aagtttgccc agtccggcga cctgacccgc      120

cataccaaga tacacacggg cgagaagccc ttccagtgtc gaatctgcat gcgtaacttc      180

agtcagtccg gcgacctgac ccgccacatc cgcacccaca ccggcgagaa gccttttgcc      240

tgtgacattt gtgggaggaa atttgcccag tccggcgacc tgacccgcca taccaagata      300

cacacgccga acccgcaccg ccgcaccgac ccgtcccaca agcccttcca gtgtcgaatc      360

tgcatgcgta acttcagtaa gcacggcaac ctgtccgagc acatccgcac ccacaccggc      420

gagaagcctt ttgcctgtga catttgtggg aggaaatttg ccaagcgctg taacctgcgc      480

tgtcatacca agatacacct gcgccaaaaa gatgcggccc ggggatccgg catggatgct      540

aagtcactaa ctgcctggtc ccggacactg gtgaccttca aggatgtatt tgtggacttc      600

accagggagg agtggaagct gctggacact gctcagcaga tcgtgtacag aaatgtgatg      660

ctggagaact ataagaacct ggtttccttg ggttatcagc ttactaagcc agatgtgatc      720

ctccggttgg agaagggaga agagccctgg ctggtggaga gagaaattca ccaagagacc      780

catcctgatt cagagactgc atttgaaatc aaatcatcag tttaa                      825


<210> 14
<211> 827
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 14
atggccccca agaaaaagcg gaaagtgggc atccacgggg tacccgccgc tatggctgag       60

aggcccttcc agtgtcgaat ctgcatgcgt aacttcagtt gtccgtccca cctgacccgc      120

cacatccgca cccacaccgg cgagaagcct tttgcctgtg acatttgtgg gaggaaattt      180

gcccagtccg gcgacctgac ccgccatacc aagatacaca cgcctaatcc tcatcgccgc      240

actgatccca gccataagcc cttccagtgt cgaatctgca tgcgtaactt cagtaagcac      300

ggcaacctgt ccgagcacat ccgcacccac accggcgaga agccttttgc ctgtgacatt      360

tgtgggagga aatttgccaa gcgctgtaac ctgcgctgtc ataccaagat acacacgggc      420

tcccaatccc ccttccagtg tcgaatctgc atgcgtaagt ttgcccgcca gttcaaccgc      480

caccagcata ccaagataca cctgcgccaa aaagatgcgg cccggggatc cggcatggat      540

gctaagtcac taactgcctg gtcccggaca ctggtgacct tcaaggatgt atttgtggac      600

ttcaccaggg aggagtggaa gctgctggac actgctcagc agatcgtgta cagaaatgtg      660

atgctggaga actataagaa cctggtttcc ttgggttatc agcttactaa gccagatgtg      720

atcctccggt tggagaaggg agaagagccc tggctggtgg agagagaaat tcaccaagag      780

acccatcctg attcagagac tgcatttgaa atcaaatcat cagttta                    827


<210> 15
<211> 800
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 15
atggccccca agaaaaagcg gaaagtgggc atccacgggg tacccgccgc tatggctgag       60

aggcccttcc agtgtcgaat ctgcatgcgt aacttcagtt gtccgtccca cctgacccgc      120

cacatccgca cccacaccgg cgagaagcct tttgcctgtg acatttgtgg gaggaaattt      180

gcccagtccg gcgacctgac ccgccatacc aagatacaca cgggcgagaa gcccttccag      240

tgtcgaatct gcatgcgtaa cttcagttgt ccgtcccacc tgacccgcca catccgcacc      300

cacaccggcg agaagccttt tgcctgtgac atttgtggga ggaaatttgc ccagtccggc      360

gacctgaccc gccataccaa gatacacacg ggatctcaga agcccttcca gtgtcgaatc      420

tgcatgcgta agtttgccca gtccggcgac ctgacccgcc ataccaagat acacctgcgc      480

caaaaagatg cggcccgggg atccggcatg gatgctaagt cactaactgc ctggtcccgg      540

acactggtga ccttcaagga tgtatttgtg gacttcacca gggaggagtg gaagctgctg      600

gacactgctc agcagatcgt gtacagaaat gtgatgctgg agaactataa gaacctggtt      660

tccttgggtt atcagcttac taagccagat gtgatcctcc ggttggagaa gggagaagag      720

ccctggctgg tggagagaga aattcaccaa gagacccatc ctgattcaga gactgcattt      780

gaaatcaaat catcagttta                                                  800


<210> 16
<211> 740
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 16
atggccccca agaaaaagcg gaaagtgggc atccacgggg tacccgccgc tatggctgag       60

aggcccttcc agtgtcgaat ctgcatgcgt aacttcagtt ccccggagca gctgtcccgc      120

cacatccgca cccacaccgg cgagaagcct tttgcctgtg acatttgtgg gaggaaattt      180

gcccagtggt ccacccgcaa gcgccatacc aagatacaca cgccgaaccc gcaccgccgc      240

accgacccgt cccacaagcc cttccagtgt cgaatctgca tgcgtaactt cagtaagcag      300

ggcaacctgg tggagcacat ccgcacccac accggcgaga agccttttgc ctgtgacatt      360

tgtgggagga aatttgccaa gcgctgtaac ctgcgctgtc ataccaagat acacctgcgc      420

caaaaagatg cggcccgggg atccggcatg gatgctaagt cactaactgc ctggtcccgg      480

acactggtga ccttcaagga tgtatttgtg gacttcacca gggaggagtg gaagctgctg      540

gacactgctc agcagatcgt gtacagaaat gtgatgctgg agaactataa gaacctggtt      600

tccttgggtt atcagcttac taagccagat gtgatcctcc ggttggagaa gggagaagag      660

ccctggctgg tggagagaga aattcaccaa gagacccatc ctgattcaga gactgcattt      720

gaaatcaaat catcagttta                                                  740


<210> 17
<211> 922
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 17
atgcccccaa gaaaaagcgg aaagtgggca tccacggggt acccgccgct atggctgaga       60

ggcccttcca gtgtcgaatc tgcatgcgta acttcagtcg ctccgacaac ctgtccgagc      120

acatccgcac ccacaccggc gagaagcctt ttgcctgtga catttgtggg aggaaatttg      180

ccaagcgctg taacctgcgc tgtcatacca agatacacac gcatcccagg gcacctattc      240

ccaagccctt ccagtgtcga atctgcatgc gtaacttcag tcagtccggc gacctgaccc      300

gccacatccg cacccacacc ggcgagaagc cttttgcctg tgacatttgt gggaggaaat      360

ttgcccagtc cggcgacctg acccgccata ccaagataca cacgccgaac ccgcaccgcc      420

gcaccgaccc gtcccacaag cccttccagt gtcgaatctg catgcgtaac ttcagtcgct      480

ccgacaacct gtccgagcac atccgcaccc acaccggcga gaagcctttt gcctgtgaca      540

tttgtgggag gaaatttgcc aagcgctgta acctgcgctg tcataccaag atacacctgc      600

gccaaaaaga tgcggcccgg ggatccggca tggatgctaa gtcactaact gcctggtccc      660

ggacactggt gaccttcaag gatgtatttg tggacttcac cagggaggag tggaagctgc      720

tggacactgc tcagcagatc gtgtacagaa atgtgatgct ggagaactat aagaacctgg      780

tttccttggg ttatcagctt actaagccag atgtgatcct ccggttggag aagggagaag      840

agccctggct ggtggagaga gaaattcacc aagagaccca tcctgattca gagactgcat      900

ttgaaatcaa atcatcagtt ta                                               922


<210> 18
<211> 274
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 18
Met Ala Pro Lys Lys Lys Arg Lys Val Gly Ile His Gly Val Pro Ala 
1               5                   10                  15      


Ala Met Ala Glu Arg Pro Phe Gln Cys Arg Ile Cys Met Arg Lys Phe 
            20                  25                  30          


Ala Gln Ser Gly Asp Leu Thr Arg His Thr Lys Ile His Thr Gly Glu 
        35                  40                  45              


Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe Ser Gln Ser Gly 
    50                  55                  60                  


Asp Leu Thr Arg His Ile Arg Thr His Thr Gly Glu Lys Pro Phe Ala 
65                  70                  75                  80  


Cys Asp Ile Cys Gly Arg Lys Phe Ala Gln Ser Gly Asp Leu Thr Arg 
                85                  90                  95      


His Thr Lys Ile His Thr Pro Asn Pro His Arg Arg Thr Asp Pro Ser 
            100                 105                 110         


His Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe Ser Lys His 
        115                 120                 125             


Gly Asn Leu Ser Glu His Ile Arg Thr His Thr Gly Glu Lys Pro Phe 
    130                 135                 140                 


Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Lys Arg Cys Asn Leu Arg 
145                 150                 155                 160 


Cys His Thr Lys Ile His Leu Arg Gln Lys Asp Ala Ala Arg Gly Ser 
                165                 170                 175     


Gly Met Asp Ala Lys Ser Leu Thr Ala Trp Ser Arg Thr Leu Val Thr 
            180                 185                 190         


Phe Lys Asp Val Phe Val Asp Phe Thr Arg Glu Glu Trp Lys Leu Leu 
        195                 200                 205             


Asp Thr Ala Gln Gln Ile Val Tyr Arg Asn Val Met Leu Glu Asn Tyr 
    210                 215                 220                 


Lys Asn Leu Val Ser Leu Gly Tyr Gln Leu Thr Lys Pro Asp Val Ile 
225                 230                 235                 240 


Leu Arg Leu Glu Lys Gly Glu Glu Pro Trp Leu Val Glu Arg Glu Ile 
                245                 250                 255     


His Gln Glu Thr His Pro Asp Ser Glu Thr Ala Phe Glu Ile Lys Ser 
            260                 265                 270         


Ser Val 
        


<210> 19
<211> 275
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 19
Met Ala Pro Lys Lys Lys Arg Lys Val Gly Ile His Gly Val Pro Ala 
1               5                   10                  15      


Ala Met Ala Glu Arg Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe 
            20                  25                  30          


Ser Cys Pro Ser His Leu Thr Arg His Ile Arg Thr His Thr Gly Glu 
        35                  40                  45              


Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Gln Ser Gly 
    50                  55                  60                  


Asp Leu Thr Arg His Thr Lys Ile His Thr Pro Asn Pro His Arg Arg 
65                  70                  75                  80  


Thr Asp Pro Ser His Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn 
                85                  90                  95      


Phe Ser Lys His Gly Asn Leu Ser Glu His Ile Arg Thr His Thr Gly 
            100                 105                 110         


Glu Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Lys Arg 
        115                 120                 125             


Cys Asn Leu Arg Cys His Thr Lys Ile His Thr Gly Ser Gln Ser Pro 
    130                 135                 140                 


Phe Gln Cys Arg Ile Cys Met Arg Lys Phe Ala Arg Gln Phe Asn Arg 
145                 150                 155                 160 


His Gln His Thr Lys Ile His Leu Arg Gln Lys Asp Ala Ala Arg Gly 
                165                 170                 175     


Ser Gly Met Asp Ala Lys Ser Leu Thr Ala Trp Ser Arg Thr Leu Val 
            180                 185                 190         


Thr Phe Lys Asp Val Phe Val Asp Phe Thr Arg Glu Glu Trp Lys Leu 
        195                 200                 205             


Leu Asp Thr Ala Gln Gln Ile Val Tyr Arg Asn Val Met Leu Glu Asn 
    210                 215                 220                 


Tyr Lys Asn Leu Val Ser Leu Gly Tyr Gln Leu Thr Lys Pro Asp Val 
225                 230                 235                 240 


Ile Leu Arg Leu Glu Lys Gly Glu Glu Pro Trp Leu Val Glu Arg Glu 
                245                 250                 255     


Ile His Gln Glu Thr His Pro Asp Ser Glu Thr Ala Phe Glu Ile Lys 
            260                 265                 270         


Ser Ser Val 
        275 


<210> 20
<211> 266
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 20
Met Ala Pro Lys Lys Lys Arg Lys Val Gly Ile His Gly Val Pro Ala 
1               5                   10                  15      


Ala Met Ala Glu Arg Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe 
            20                  25                  30          


Ser Cys Pro Ser His Leu Thr Arg His Ile Arg Thr His Thr Gly Glu 
        35                  40                  45              


Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Gln Ser Gly 
    50                  55                  60                  


Asp Leu Thr Arg His Thr Lys Ile His Thr Gly Glu Lys Pro Phe Gln 
65                  70                  75                  80  


Cys Arg Ile Cys Met Arg Asn Phe Ser Cys Pro Ser His Leu Thr Arg 
                85                  90                  95      


His Ile Arg Thr His Thr Gly Glu Lys Pro Phe Ala Cys Asp Ile Cys 
            100                 105                 110         


Gly Arg Lys Phe Ala Gln Ser Gly Asp Leu Thr Arg His Thr Lys Ile 
        115                 120                 125             


His Thr Gly Ser Gln Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Lys 
    130                 135                 140                 


Phe Ala Gln Ser Gly Asp Leu Thr Arg His Thr Lys Ile His Leu Arg 
145                 150                 155                 160 


Gln Lys Asp Ala Ala Arg Gly Ser Gly Met Asp Ala Lys Ser Leu Thr 
                165                 170                 175     


Ala Trp Ser Arg Thr Leu Val Thr Phe Lys Asp Val Phe Val Asp Phe 
            180                 185                 190         


Thr Arg Glu Glu Trp Lys Leu Leu Asp Thr Ala Gln Gln Ile Val Tyr 
        195                 200                 205             


Arg Asn Val Met Leu Glu Asn Tyr Lys Asn Leu Val Ser Leu Gly Tyr 
    210                 215                 220                 


Gln Leu Thr Lys Pro Asp Val Ile Leu Arg Leu Glu Lys Gly Glu Glu 
225                 230                 235                 240 


Pro Trp Leu Val Glu Arg Glu Ile His Gln Glu Thr His Pro Asp Ser 
                245                 250                 255     


Glu Thr Ala Phe Glu Ile Lys Ser Ser Val 
            260                 265     


<210> 21
<211> 246
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 21
Met Ala Pro Lys Lys Lys Arg Lys Val Gly Ile His Gly Val Pro Ala 
1               5                   10                  15      


Ala Met Ala Glu Arg Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe 
            20                  25                  30          


Ser Ser Pro Glu Gln Leu Ser Arg His Ile Arg Thr His Thr Gly Glu 
        35                  40                  45              


Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Gln Trp Ser 
    50                  55                  60                  


Thr Arg Lys Arg His Thr Lys Ile His Thr Pro Asn Pro His Arg Arg 
65                  70                  75                  80  


Thr Asp Pro Ser His Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn 
                85                  90                  95      


Phe Ser Lys Gln Gly Asn Leu Val Glu His Ile Arg Thr His Thr Gly 
            100                 105                 110         


Glu Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Lys Arg 
        115                 120                 125             


Cys Asn Leu Arg Cys His Thr Lys Ile His Leu Arg Gln Lys Asp Ala 
    130                 135                 140                 


Ala Arg Gly Ser Gly Met Asp Ala Lys Ser Leu Thr Ala Trp Ser Arg 
145                 150                 155                 160 


Thr Leu Val Thr Phe Lys Asp Val Phe Val Asp Phe Thr Arg Glu Glu 
                165                 170                 175     


Trp Lys Leu Leu Asp Thr Ala Gln Gln Ile Val Tyr Arg Asn Val Met 
            180                 185                 190         


Leu Glu Asn Tyr Lys Asn Leu Val Ser Leu Gly Tyr Gln Leu Thr Lys 
        195                 200                 205             


Pro Asp Val Ile Leu Arg Leu Glu Lys Gly Glu Glu Pro Trp Leu Val 
    210                 215                 220                 


Glu Arg Glu Ile His Gln Glu Thr His Pro Asp Ser Glu Thr Ala Phe 
225                 230                 235                 240 


Glu Ile Lys Ser Ser Val 
                245     


<210> 22
<211> 307
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 22
Met Ala Pro Lys Lys Lys Arg Lys Val Gly Ile His Gly Val Pro Ala 
1               5                   10                  15      


Ala Met Ala Glu Arg Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe 
            20                  25                  30          


Ser Arg Ser Asp Asn Leu Ser Glu His Ile Arg Thr His Thr Gly Glu 
        35                  40                  45              


Lys Pro Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Lys Arg Cys 
    50                  55                  60                  


Asn Leu Arg Cys His Thr Lys Ile His Thr His Pro Arg Ala Pro Ile 
65                  70                  75                  80  


Pro Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe Ser Gln Ser 
                85                  90                  95      


Gly Asp Leu Thr Arg His Ile Arg Thr His Thr Gly Glu Lys Pro Phe 
            100                 105                 110         


Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Gln Ser Gly Asp Leu Thr 
        115                 120                 125             


Arg His Thr Lys Ile His Thr Pro Asn Pro His Arg Arg Thr Asp Pro 
    130                 135                 140                 


Ser His Lys Pro Phe Gln Cys Arg Ile Cys Met Arg Asn Phe Ser Arg 
145                 150                 155                 160 


Ser Asp Asn Leu Ser Glu His Ile Arg Thr His Thr Gly Glu Lys Pro 
                165                 170                 175     


Phe Ala Cys Asp Ile Cys Gly Arg Lys Phe Ala Lys Arg Cys Asn Leu 
            180                 185                 190         


Arg Cys His Thr Lys Ile His Leu Arg Gln Lys Asp Ala Ala Arg Gly 
        195                 200                 205             


Ser Gly Met Asp Ala Lys Ser Leu Thr Ala Trp Ser Arg Thr Leu Val 
    210                 215                 220                 


Thr Phe Lys Asp Val Phe Val Asp Phe Thr Arg Glu Glu Trp Lys Leu 
225                 230                 235                 240 


Leu Asp Thr Ala Gln Gln Ile Val Tyr Arg Asn Val Met Leu Glu Asn 
                245                 250                 255     


Tyr Lys Asn Leu Val Ser Leu Gly Tyr Gln Leu Thr Lys Pro Asp Val 
            260                 265                 270         


Ile Leu Arg Leu Glu Lys Gly Glu Glu Pro Trp Leu Val Glu Arg Glu 
        275                 280                 285             


Ile His Gln Glu Thr His Pro Asp Ser Glu Thr Ala Phe Glu Ile Lys 
    290                 295                 300                 


Ser Ser Val 
305         


<210> 23
<211> 201
<212> DNA
<213> Homo sapiens


<220>
<221> misc_feature
<222> (1)..(201)
<223> This sequence may encompass 15-67 "cag" repeating units

<220> 
<223> See specification as filed for detailed description of
      substitutions and preferred embodiments

<400> 23
cagcagcagc agcagcagca gcagcagcag cagcagcagc agcagcagca gcagcagcag       60

cagcagcagc agcagcagca gcagcagcag cagcagcagc agcagcagca gcagcagcag      120

cagcagcagc agcagcagca gcagcagcag cagcagcagc agcagcagca gcagcagcag      180

cagcagcagc agcagcagca g                                                201



