Rewriting SCN1A: Genome Editing for Genetic Epilepsies
The use of an adenine base editor (ABE) to directly correct SCN1AR613X, a recurrent variant found in patients with DS, suggests the therapeutic potential of prime editing for the treatment of patients with SCN1A-associated GEFS+.
Samantha A Dow, Tracy A. Bedrosian
· Epilepsy Currents · 0 citations