Induced exon inclusion in spinal muscle atrophy

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专利权人:

Sarepta Therapeutics; Inc.

发明人:

Linsley, Peter,Leppert, Brian James

申请号：

AU2012345638

公开号：

AU2012345638B2

申请日:

2012.11.30

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

来源网站：

中国工程科技知识中心

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http://www.ckcest.cn/home/