RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

Published 2020 View Full Article

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Title
RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model
Authors
Keywords
epilepsy, developmental and epileptic encephalopathy, DEE, dynamin-1, DNM1, gene therapy, RNA interferance, adeno-associated virus 9, AAV9, infantile spasms, Lennox-Gastaut syndrome
Journal
MOLECULAR THERAPY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-04-16
DOI
10.1016/j.ymthe.2020.04.007

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