Isis Pharmaceuticals, Inc.

Cold Spring Harbor Laboratory

Ionis Pharmaceuticals, Inc.

Biogen Ma Inc.

Yimin Hua

Adrian R Krainer

C Frank Bennett

Frank Rigo

Seng H Cheng

Brenda F Baker

Gene Hung

Lamya S Shihabuddin

Marco A Passini

Katherine W Klinger

Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. 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.

Compositions and methods for modulation of SMN2 splicing in a subject

Provided herein are antisense compounds and methods for recruiting one or more non-cleaving protein to a target nucleic acid in a cell. In certain instances such recruitment of a non-cleaving protein alters the function or activity of the target nucleic acid. In certain such instances, the target nucleic acid a pre-mRNA and the recruitment of the non-cleaving protein results in a change in splicing of the pre-mRNA.

Compounds and methods for modulating interaction between proteins and target nucleic acids

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.

Compositions and methods for modulation of SMN2 splicing

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Jericho, NY, United States of America

Yimin Hua