Antisense-based therapy for the treatment of spinal muscular atrophy-Reference-Cited by-同舟云学术

Antisense-based therapy for the treatment of spinal muscular atrophy

Published:2012-10-01 Issue:1 Volume:199 Page:21-25
ISSN:1540-8140
Container-title:Journal of Cell Biology
language:en
Short-container-title:

Author:

Rigo Frank¹,Hua Yimin²,Krainer Adrian R.²,Bennett C. Frank¹

Affiliation:

1. Isis Pharmaceuticals, Carlsbad, CA 92010

2. Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724

Abstract

One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.

Publisher

Rockefeller University Press

Subject

Cell Biology

Link

http://rupress.org/jcb/article-pdf/199/1/21/1357614/jcb_201207087.pdf

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