Upregulation of Haploinsufficient Gene Expression in the Brain by Targeting a Long Non-coding RNA Improves Seizure Phenotype in a Model of Dravet Syndrome

Dravet syndrome is a devastating genetic brain disorder caused by heterozygous loss-of-function mutation in the voltage-gated sodium channel gene SCN1A. There are currently no treatments, but the upregulation of SCN1A healthy allele represents an appealing therapeutic strategy. In this study we iden...

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Detalles Bibliográficos
Autores principales: Hsiao, J., Yuan, T.Y., Tsai, M.S., Lu, C.Y., Lin, Y.C., Lee, M.L., Lin, S.W., Chang, F.C., Liu Pimentel, H., Olive, C., Coito, C., Shen, G., Young, M., Thorne, T., Lawrence, M., Magistri, M., Faghihi, M.A., Khorkova, O., Wahlestedt, C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4972487/
https://www.ncbi.nlm.nih.gov/pubmed/27333023
http://dx.doi.org/10.1016/j.ebiom.2016.05.011

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