Biochemical and biophysical research communicationsAnimalsDependovirusSeizuresRatsGenetic Therapy
AAV9-mediated NGLY1 gene replacement suppresses non-epileptic convulsions in Ngly1-/- rats.
Makoto Asahina, Reiko Fujinawa, Hiroto Hirayama, Hiroshi Yukitake, Tadashi Suzuki
Published: 202510.1016/j.bbrc.2025.152823
Abstract
N-glycanase 1 (NGLY1) deficiency is a rare autosomal-recessive neurological disorder characterized by neurological dysfunction and so far, has no effective therapy. A systemic Ngly1-/- rat model recapitulates many patient symptoms, including developm…
Preview only. Read the full abstract at the source