公开(公告)号：US20140011977A1

公开(公告)日：2014-01-09

申请号：US13901476

申请日：2013-05-23

Applicant: Cold Spring Harbor Laboratory

Inventor： Adrian R. Krainer ,  Luca Cartegni

IPC: C12N15/113

CPC classification number: C12N15/1135 ,  A61K38/00 ,  A61K48/00 ,  C07K2319/00 ,  C12N15/113 ,  C12N2310/15 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2310/52 ,  C12N2310/3521

Abstract: The present invention provides a chimeric molecule including a base-pairing segment that binds specifically to a single-stranded nucleic acid molecule; and a moiety that modulates splicing or translation. The invention also provides a chimeric molecule including a base-pairing segment that binds specifically to a double-stranded nucleic acid molecule; and a peptide that modulates transcription, wherein the peptide comprises up to about one hundred amino acid residues.

Abstract translation: 本发明提供了包含与单链核酸分子特异性结合的碱基配对区段的嵌合分子; 以及调节拼接或翻译的部分。 本发明还提供了嵌合分子，其包括与双链核酸分子特异性结合的碱基配对区段; 和调节转录的肽，其中肽包含多至约一百个氨基酸残基。

公开(公告)号：US20130109091A1

公开(公告)日：2013-05-02

申请号：US13720474

申请日：2012-12-19

Applicant: Isis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/33 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.