Improved MECP2 gene therapy extends the survival of MeCP2-null mice without apparent toxicity after intracisternal delivery

Sarah E. Sinnett, Ralph D. Hector, Kamal K.E. Gadalla, Clifford Heindel, Daphne Chen, Violeta Zaric, Mark E.S. Bailey, Stuart R. Cobb, Steven J. Gray

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Medicine & Life Sciences