Article
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jan 2013
Gadalla Kamal K E, Bailey Mark E S, Spike Rosemary C, Ross Paul D, Woodard Kenton T, Kalburgi Sahana Nagabhushan, Bachaboina Lavanya, Deng Jie V, West Anne E, Samulski R Jude, Gray Steven J, Cobb Stuart R
Abstract excerpt
Typical Rett syndrome (RTT) is a pediatric disorder caused by loss-of-function mutations in the methyl-CpG binding protein 2 (MECP2) gene. The demonstrated reversibility of RTT-like phenotypes in mice suggests that MECP2 gene replacement is a potential therapeutic option in patients. We report improvements in survival and phenotypic severity in Mecp2-null male mice after neonatal intracranial delivery of a...
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