
                                SEQUENCE LISTING

<110> Sarepta Therapeutics, Inc.
      The United States of America, as represented by the Secretary, Dept. of
      Health and Human Services
      University of Maryland
      Erdos, Michael R.
      Collins, Francis S.
      Cao, Kan
      Kole, Ryszard
      Bestwick, Richard Keith

<120> OLIGONUCLEOTIDE ANALOGUES TARGETING
  HUMAN LMNA

<130> 120178.503WO

<140> PCT                 
<141> 2017-04-28  

<150> US 62/330,027       
<151> 2016-04-29  

<160> 25

<170> FastSEQ for Windows Version 4.0

<210> 1
<211> 270
<212> DNA
<213> Homo sapiens

<400> 1
ggctcccact gcagcagctc gggggacccc gctgagtaca acctgcgctc gcgcaccgtg      60
ctgtgcggga cctgcgggca gcctgccgac aaggcatctg ccagcggctc aggagcccag     120
gtgggcggac ccatctcctc tggctcttct gcctccagtg tcacggtcac tcgcagctac     180
cgcagtgtgg ggggcagtgg gggtggcagc ttcggggaca atctggtcac ccgctcctac     240
ctcctgggca actccagccc ccgaacccag                                      270

<210> 2
<211> 270
<212> DNA
<213> Homo sapiens

<400> 2
ggctcccact gcagcagctc gggggacccc gctgagtaca acctgcgctc gcgcaccgtg      60
ctgtgcggga cctgcgggca gcctgccgac aaggcatctg ccagcggctc aggagcccag     120
gtgggtggac ccatctcctc tggctcttct gcctccagtg tcacggtcac tcgcagctac     180
cgcagtgtgg ggggcagtgg gggtggcagc ttcggggaca atctggtcac ccgctcctac     240
ctcctgggca actccagccc ccgaacccag                                      270

<210> 3
<211> 25
<212> DNA
<213> Artificial Sequence

<220> 
<223> antisense oligonucleotide

<400> 3
ctgagccgct ggcagatgcc ttgtc                                            25

<210> 4
<211> 25
<212> DNA
<213> Artificial Sequence

<220> 
<223> antisense oligonucleotide

<400> 4
gaggagatgg gtccacccac ctggg                                            25

<210> 5
<211> 9
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 5
Arg Arg Arg Gln Arg Arg Lys Lys Arg
 1               5

<210> 6
<211> 9
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 6
Arg Lys Lys Arg Arg Gln Arg Arg Arg
 1               5

<210> 7
<211> 11
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 7
Arg Arg Arg Arg Arg Arg Arg Arg Arg Phe Phe
 1               5                  10

<210> 8
<211> 11
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 8
Arg Arg Arg Arg Arg Phe Phe Arg Arg Arg Arg
 1               5                  10

<210> 9
<211> 4
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 9
Arg Arg Arg Arg
 1

<210> 10
<211> 5
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 10
Arg Arg Arg Arg Arg
 1               5

<210> 11
<211> 6
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 11
Arg Arg Arg Arg Arg Arg
 1               5

<210> 12
<211> 7
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 12
Arg Arg Arg Arg Arg Arg Arg
 1               5

<210> 13
<211> 8
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 13
Arg Arg Arg Arg Arg Arg Arg Arg
 1               5

<210> 14
<211> 9
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 14
Arg Arg Arg Arg Arg Arg Arg Arg Arg
 1               5

<210> 15
<211> 16
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 2,4,6,8,10,12,14,16
<223> Xaa = 6-aminohexanoic acid


<400> 15
Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa
 1               5                  10                  15

<210> 16
<211> 12
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 2,5,8,11
<223> Xaa = 6-aminohexanoic acid

<400> 16
Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg
 1               5                  10

<210> 17
<211> 15
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 2,5,8,11,14
<223> Xaa = 6-aminohexanoic acid

<400> 17
Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg
 1               5                  10                  15

<210> 18
<211> 12
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 2
<223> Xaa = 6-aminohexanoic acid

<220>
<221> VARIANT        
<222> 5
<223> Xaa = beta-alanine

<220>
<221> VARIANT        
<222> 8
<223> Xaa = 6-aminohexanoic acid

<220>
<221> VARIANT        
<222> 11
<223> Xaa = beta-alanine

<400> 18
Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg
 1               5                  10

<210> 19
<211> 14
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 19
Arg Ala Arg Arg Ala Arg Arg Ala Arg Arg Ala Arg Phe Phe
 1               5                  10

<210> 20
<211> 14
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 20
Arg Gly Arg Arg Gly Arg Arg Gly Arg Arg Gly Arg Phe Phe
 1               5                  10

<210> 21
<211> 10
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 21
Arg Phe Phe Arg Phe Phe Arg Phe Phe Arg
 1               5                  10

<210> 22
<211> 14
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 2,5,8,11,13
<223> Xaa = 6-aminohexanoic acid

<220>
<221> VARIANT        
<222> 14
<223> Xaa = beta-alanine

<400> 22
Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Xaa Xaa
 1               5                  10

<210> 23
<211> 12
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<220>
<221> VARIANT        
<222> 11
<223> Xaa = 6-aminohexanoic acid

<220>
<221> VARIANT        
<222> 12
<223> Xaa = beta-alanine

<400> 23
Arg Phe Phe Arg Phe Phe Arg Phe Phe Arg Xaa Xaa
 1               5                  10

<210> 24
<211> 11
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 24
Arg Phe Phe Arg Phe Phe Arg Phe Phe Arg Gly
 1               5                  10

<210> 25
<211> 7
<212> PRT
<213> Artificial Sequence

<220> 
<223> cell penetrating peptide

<400> 25
Arg Arg Arg Arg Arg Arg Gly
 1               5
