发明名称 Adeno-associated virus-mediated delivery of GDNF to skeletal muscles
摘要 Compositions and methods for delivering GDNF to skeletal muscles to result in a therapeutic effect are disclosed. The compositions and methods use adeno-associated virus (AAV)-based gene delivery systems. The methods are useful for treating motoneuron diseases, such as amyotrophic lateral sclerosis (ALS).
申请公布号 US7112321(B2) 申请公布日期 2006.09.26
申请号 US20020327620 申请日期 2002.12.19
申请人 GENZYME CORPORATION 发明人 WANG LIJUN;MURAMATSU SHIN-ICHI;NAKANO IMAHARU;MIZUKAMI HIROAKI;OZAWA KEIYA
分类号 A61K48/00;A61K35/76;A61P25/14;C07K14/475;C12N15/864 主分类号 A61K48/00
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