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.
引用
收藏
页码：21 / 25
页数：5
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