Molecular Therapy - Methods & Clinical Development
Volume 5, 16 June 2017, Pages 106-115
Original ArticleImproved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
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open access
Graphical Abstract
Keywords
Rett syndrome
MeCP2
cisterna magna
intrathecal
microRNA
AAV
viral vector
Mecp2−/y
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© 2017 The Authors.