Colasante et al. exploit an activatory CRISPR-targeting Scn1a gene promoter as a therapeutic strategy to rescue Scn1a haploinsufficiency in a mouse model of Dravet syndrome and restore physiological levels of its gene product, the Nav1.1 voltage-gated sodium channel.
dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice / 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. - In: MOLECULAR THERAPY. - ISSN 1525-0016. - 28:1(2020), pp. 235-253. [10.1016/j.ymthe.2019.08.018]
dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice
Simone Brusco;Simone Bido;Valerio Castoldi;Silvia Marenna;Letizia Leocani;
2020-01-01
Abstract
Colasante et al. exploit an activatory CRISPR-targeting Scn1a gene promoter as a therapeutic strategy to rescue Scn1a haploinsufficiency in a mouse model of Dravet syndrome and restore physiological levels of its gene product, the Nav1.1 voltage-gated sodium channel.| File | Dimensione | Formato | |
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