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.

Published 2019 | Version v1

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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.

Contributors

Others:

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.

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.

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URL: http://hdl.handle.net/11567/999413
URN: urn:oai:iris.unige.it:11567/999413

Origin repository: UNIGE

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

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