EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
申请人:Sarepta Therapeutics, Inc.
公开号:US20210169919A1
公开(公告)日:2021-06-10
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
申请人:Sarepta Therapeutics, Inc.
公开号:US20220193246A1
公开(公告)日:2022-06-23
Methods for treating Duchenne muscular dystrophy using conjugates of cell penetrating peptides to antisense oligonucleotide that induce exon skipping in the human dystrophin gene. In one embodiment, the cell penetrating peptide is an arginine-rich peptide comprising six contiguous arginine residues and the antisense oligonucleotide is eteplirsen, golodirsen or casimersen.
[EN] EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY<br/>[FR] OLIGOMÈRES À SAUTS D'EXONS ASSOCIÉS À LA DYSTROPHIE MUSCULAIRE
申请人:SAREPTA THERAPEUTICS INC
公开号:WO2020023688A1
公开(公告)日:2020-01-30
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 2 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): Formula (I) or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R100 are defined herein.