Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

Title
Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Authors
Keywords
Rett syndrome, MeCP2, cisterna magna, intrathecal, microRNA, AAV, viral vector, Mecp2
Journal
Publisher
Elsevier BV
Online
2017-04-19
DOI
10.1016/j.omtm.2017.04.006

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