NON-HUMAN PRIMATE BIODISTRIBUTION AND SAFETY STUDY SUPPORTS TRANSLATION OF AAV9-MEDIATED GENE SILENCING THERAPY FOR CMT1A NEUROPATHY

被引：0

作者：

Stavrou, Marina ^{[1
]}

Wallace, Lindsay ^{[2
]}

Price, Brian ^{[3
]}

Thangaraj, Merlin ^{[2
]}

Harper, Scott ^{[2
,4
]}

Kleopa, Kleopas ^{[1
,5
]}

机构：

[1] Cyprus Inst Neurol & Genet, Neurosci Dept, Nicosia, Cyprus

[2] Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA

[3] Armatus Bio, Columbus, OH USA

[4] Ohio State Univ, Sch Med, Dept Pediat, Columbus, OH 43210 USA

[5] Cyprus Inst Neurol & Genet, Ctr Neuromuscular Disorders, Nicosia, Cyprus

来源：

关键词：

Non-human primates; AAV9; Lumbar intrathecal injection; Charcot Marie Tooth type 1A; gene therapy;

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

O 382

引用

页码：S163 / S164

页数：2