Two gene‑therapy reports outlined translational progress in distinct neurological disorders. Researchers demonstrated that restoring SynGAP1 expression via gene therapy rescued epilepsy and behavioral deficits in preclinical models of SYNGAP1‑related disorders, suggesting a therapeutic route for a rare neurodevelopmental condition. The study reported functional improvements across seizure burden and behavior scales. Separately, a silence‑and‑replace SPAST‑AAV9 approach prevented hereditary spastic paraplegia (HSP) symptoms in animal models by addressing both toxic gain‑of‑function and loss‑of‑function disease components. Authors of both studies emphasized the need for long‑term safety data and scalable vector manufacturing before clinical translation, but described clear preclinical efficacy that supports IND‑enabling work.