RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN

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专利权人:

发明人:

SCOTT QUENTON HARPER,JIAN LIU

申请号：

US14853517

公开号：

US20160310548A1

申请日:

2015.09.14

申请国别(地区):

US

年份:

2016

代理人:

摘要：

The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

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