In vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy.

ABSTRACT: Epilepsy is a major health burden, calling for new mechanistic insights and therapies. CRISPR-mediated gene editing shows promise to cure genetic pathologies, although hitherto it has mostly been applied ex vivo. Its translational potential for treating non-genetic pathologies is still unexplored. Furthermore, neurological diseases represent an important challenge for the application of CRISPR, because of the need in many cases to manipulate gene function of neurons in situ. A variant of CRISPR, CRISPRa, offers the possibility to modulate the expression of endogenous genes by directly targeting their promoters. We asked if this strategy can effectively treat acquired focal epilepsy, focusing on ion channels because their manipulation is known be effective in changing network hyperactivity

SUBMITTER: Colasante G 

PROVIDER: S-EPMC7089667 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature