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1.发明申请RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF EXON 2-TARGETED U7SNRNA POLYNUCLEOTIDE CONSTRUCTS 有权重组腺病毒相关的外显子2对靶向U7SNRNA多核苷酸构建体的病毒公开(公告)号：US20160076028A1
公开(公告)日：2016-03-17
申请号：US14785769
申请日：2014-04-18
Inventor： Kevin Flanigan , Aneline Vulin-Chaffiol , Nicolas Wein
IPC: C12N15/113 , C12N7/00
CPC classification number: C12N15/113 , A61K48/005 , C07K14/4708 , C12N7/00 , C12N15/111 , C12N15/86 , C12N2310/11 , C12N2320/32 , C12N2320/33 , C12N2330/51 , C12N2750/14132 , C12N2750/14143 , C12N2750/14171 , C12N2750/14321 , C12N2750/14333
Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Abstract translation: 本发明涉及重组腺相关病毒（rAAV）递送多核苷酸，用于治疗由DMD外显子2复制而产生的Duchenne肌营养不良症。本发明提供rAAV产品和使用rAAV治疗杜氏肌营养不良症的方法。
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