EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
申请人:SAREPTA THERAPEUTICS, INC.
公开号:US20160040162A1
公开(公告)日:2016-02-11
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
抗义RNA分子能够结合到人类肌营养不良蛋白基因中的特定靶位点,诱导外显子53跳跃。
[EN] EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY<br/>[FR] CONJUGUÉS OLIGOMÈRES DE SAUTS D'EXONS POUR LA DYSTROPHIE MUSCULAIRE
申请人:SAREPTA THERAPEUTICS INC
公开号:WO2018118662A1
公开(公告)日:2018-06-28
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
与人类肌营养不良基因中选择的靶位点互补的反义寡核苷酸共轭物用于诱导外显子53跳跃。
Antisense-induced exon exclusion in type VII collagen
申请人:Sarepta Therapeutics, Inc.
公开号:US10849917B2
公开(公告)日:2020-12-01
The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
FUNCTIONALLY-MODIFIED OLIGONUCLEOTIDES AND SUBUNITS THEREOF
申请人:Sarepta Therapeutics, Inc.
公开号:US20140330006A1
公开(公告)日:2014-11-06
Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
[EN] PROCESSES FOR PREPARING OLIGOMERS<br/>[FR] PROCÉDÉS DE PRÉPARATION D'OLIGOMÈRES
申请人:SAREPTA THERAPEUTICS INC
公开号:WO2017205496A1
公开(公告)日:2017-11-30
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomersynthesis while maintaining overall yield and purity of a synthesized oligomer.