dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice

Dravet syndrome (DS) is a severe epileptic encephalopathy caused mainly by heterozygous loss-of-function mutations of the SCN1A gene, indicating haploinsufficiency as the pathogenic mechanism. Here we tested whether catalytically dead Cas9 (dCas9)-mediated Scn1a gene activation can rescue Scn1a hapl...

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Bibliografische gegevens
Gepubliceerd in:Mol Ther
Hoofdauteurs: Colasante, Gaia, Lignani, Gabriele, Brusco, Simone, Di Berardino, Claudia, Carpenter, Jenna, Giannelli, Serena, Valassina, Nicholas, Bido, Simone, Ricci, Raffaele, Castoldi, Valerio, Marenna, Silvia, Church, Timothy, Massimino, Luca, Morabito, Giuseppe, Benfenati, Fabio, Schorge, Stephanie, Leocani, Letizia, Kullmann, Dimitri M., Broccoli, Vania
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2020
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Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6952031/
https://ncbi.nlm.nih.gov/pubmed/31607539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.08.018
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