Patent ID: 11911403
Assignee: SAREPTA THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. An antisense oligomer compound of 12 to 40 subunits, comprising:
at least one subunit that is a nucleotide analog having (i) a modified internucleoside linkage, (ii) a modified sugar moiety, or (iii) a combination of the foregoing; and
a targeting sequence complementary to 12 or more contiguous nucleotides in a target region spanning an exon/intron junction of human type VII collagen pre-mRNA, wherein the contiguous nucleotides include the exon/intron junction, and wherein said exon/intron junction comprises the splice junction of exon 80/intron 80, wherein the targeting sequence is selected from SEQ ID NOS: 27, 28, 29, and 30.