Gene Editing and Modulation: the Holy Grail for the Genetic Epilepsies?

ABSTRACT: Epilepsy is a complex neurological disorder for which there are a large number of monogenic subtypes. Monogenic epilepsies are often severe and disabling, featuring drug-resistant seizures and significant developmental comorbidities. These disorders are potentially amenable to a precision medicine approach, of which genome editing using CRISPR/Cas represents the holy grail. Here we consider mutations in some of the most 'common' rare epilepsy genes and discuss the different CRISPR/Cas approaches that could be taken to cure these disorders. We consider scenarios where CRISPR-mediated gene modulation could serve as an effective therapeutic strategy and discuss whether a single gene corrective approach could hold therapeutic potential in the context of homeostatic compensation in the developi

SUBMITTER: Carpenter JC 

PROVIDER: S-EPMC8608979 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature