Antisense-based therapy for the treatment of spinal muscular atrophy

被引：126

作者：

Rigo, Frank ^{[1
]}

Hua, Yimin ^{[2
]}

Krainer, Adrian R. ^{[2
]}

Bennett, C. Frank ^{[1
]}

机构：

[1] ISIS Pharmaceut, Carlsbad, CA 92010 USA

[2] Cold Spring Harbor Lab, Cold Spring Harbor, NY 11724 USA

来源：

JOURNAL OF CELL BIOLOGY | 2012年 / 199卷 / 01期

关键词：

EXONIC SPLICING ENHANCER; SURVIVAL-MOTOR-NEURON; CARDIAC DEFECTS; MOUSE MODEL; OLIGONUCLEOTIDE; MECHANISMS; DISEASE; THERAPEUTICS; NECROSIS; MASKING;

D O I：

10.1083/jcb.201207087

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

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.

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页码：21 / 25

页数：5

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