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

Developmental and epileptic encephalopathy (DEE) associated with de novo variants in the gene encoding dynamin-1 (DNM1) is a severe debilitating disease with no pharmacological remedy. Like most genetic DEEs, the majority of DNM1 patients suffer from therapy-resistant seizures and comorbidities such...

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Detalles Bibliográficos
Autores principales: Aimiuwu, Osasumwen V., Fowler, Allison M., Sah, Megha, Teoh, Jia Jie, Kanber, Ayla, Pyne, Nettie K., Petri, Sabrina, Rosenthal-Weiss, Chana, Yang, Mu, Harper, Scott Q., Frankel, Wayne N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7335739/
https://www.ncbi.nlm.nih.gov/pubmed/32353324
http://dx.doi.org/10.1016/j.ymthe.2020.04.007

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