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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Principais autores:	J. Hsiao, T.Y. Yuan, M.S. Tsai, C.Y. Lu, Y.C. Lin, M.L. Lee, S.W. Lin, F.C. Chang, H. Liu Pimentel, C. Olive, C. Coito, G. Shen, M. Young, T. Thorne, M. Lawrence, M. Magistri, M.A. Faghihi, O. Khorkova, C. Wahlestedt
Formato:	Artigo
Idioma:	Inglês
Publicado:	Elsevier 2016-07-01
Series:	EBioMedicine
Assuntos:	Dravet syndrome SCN1A Long non-coding RNA Natural antisense transcript AntagoNAT Oligonucleotide-based compound
Acceso en liña:	http://www.sciencedirect.com/science/article/pii/S2352396416301943
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