The present disclosure provides alternative sugar moieties and polynucleotides comprising such sugar moieties, and methods of use thereof.
本公开提供了替代糖基和包含这些糖基的多核苷酸及其使用方法。
ASYMMETRIC AUXILIARY GROUP
申请人:Chiralgen, LTD.
公开号:US20150197540A1
公开(公告)日:2015-07-16
To provide a chiral reagent or a salt thereof. The chiral reagent has following chemical formula (I). In the formula (I), G
1
and G
2
are independently a hydrogen atom, a nitro group (—NO
2
), a halogen atom, a cyano group (—CN), a group of formula (II) or (III), or both G
1
and G
2
taken together to form a group of formula (IV).
US4016267A
申请人:——
公开号:US4016267A
公开(公告)日:1977-04-05
US9598458B2
申请人:——
公开号:US9598458B2
公开(公告)日:2017-03-21
[EN] COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY<br/>[FR] COMPOSITIONS POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE
申请人:SAREPTA THERAPEUTICS INC
公开号:WO2020214763A1
公开(公告)日:2020-10-22
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.