Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
申请人:Sarepta Therapeutics, Inc.
公开号:US10202602B2
公开(公告)日:2019-02-12
Oligonucleotide analogs 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.
Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
Alkylation of benzo- and dibenzocrown ethers by various alcohols
作者:A. K. Tashmukhamedova、I. A. Stempnevskaya、N. Zh. Saifullina、M. G. Levkovich
DOI:10.1007/bf00471797
日期:1986.11
METHODS FOR TREATING PROGEROID LAMINOPATHIES USING OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
申请人:Sarepta Therapeutics, Inc.
公开号:US20170051278A1
公开(公告)日:2017-02-23
Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.