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

Colasante, G.; Lignani, G.; Brusco, S.; Di Berardino, C.; Carpenter, J.; Giannelli, S.; Valassina, N.; Bido, S.; Ricci, R.; Castoldi, V.; Marenna, S.; Church, T.; Massimino, L.; Morabito, G.; Benfenati, F.; Schorge, S.; Leocani, L.; Kullmann, D. M.; Broccoli, V.

doi:10.1016/j.ymthe.2019.08.018

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 G.;Lignani G.;Brusco S.;Di Berardino C.;Carpenter J.;Giannelli S.;Valassina N.;Bido S.;Ricci R.;Castoldi V.;Marenna S.;Church T.;Massimino L.;Morabito G.;Benfenati F.;Schorge S.;Leocani L.;Kullmann D. M.;Broccoli V.

2019-01-01

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.