CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy-Reference-Cited by-同舟云学术

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Published:2010-04-01 Issue:4 Volume:120 Page:1253-1264
ISSN:0021-9738
Container-title:Journal of Clinical Investigation
language:en
Short-container-title:J. Clin. Invest.

Author:

Passini Marco A.,Bu Jie,Roskelley Eric M.,Richards Amy M.,Sardi S. Pablo,O’Riordan Catherine R.,Klinger Katherine W.,Shihabuddin Lamya S.,Cheng Seng H.

Publisher

American Society for Clinical Investigation

Subject

General Medicine

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1. Identification and characterization of a spinal muscular atrophy-determining gene

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4. Modern Management of Spinal Muscular Atrophy

5. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2

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