RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN
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.
申请公布号
WO2017048732(A1)
申请公布日期
2017.03.23
申请号
WO2016US51552
申请日期
2016.09.13
申请人
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL