Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.

Abstract

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… (More)
DOI: 10.1038/mt.2012.200

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Cite this paper

@article{Gadalla2013ImprovedSA, title={Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.}, author={Kamal K. E. Gadalla and Mark E. S. Bailey and Rosemary C Spike and Paul D S Ross and Kenton T Woodard and Sahana Nagabhushan Kalburgi and Lavanya Bachaboina and Jie Deng and Anne E West and Richard Jude Samulski and Steven J. Gray and Stuart Cobb}, journal={Molecular therapy : the journal of the American Society of Gene Therapy}, year={2013}, volume={21 1}, pages={18-30} }