Gene Therapy Extends Lifespan and Reduces Symptoms in Sandhoff Disease Cat Model
Intravenous gene therapy extended lifespan, reduced neurological symptoms, and improved disease markers in a Sandhoff disease cat model, supporting further investigation of systemic AAV gene therapy for this disorder. The post Gene Therapy Extends Lifespan and Reduces Symptoms in Sandhoff Disease Cat Model appeared first on GEN - Genetic Engineering and Biotechnology News .
Gene therapy has shown promise in extending lifespan and reducing symptoms in a cat model of Sandhoff disease, a fatal neurodegenerative condition caused by the absence of a specific enzyme. The study, published in Science Translational Medicine, delivered a bicistronic AAV vector-based gene therapy intravenously to the cats before symptoms appeared.
Although the treatment was safe, the cats lived significantly longer lives - two times longer with a low dose and three times longer with a high dose, compared to untreated cats. The therapy also alleviated tremors, reduced central nervous system damage, and improved liver damage in the high-dose group. These findings suggest the potential for IV-delivered gene therapy to restore Hex enzyme activity in Sandhoff disease patients.
Written by urgent.news from GEN Biotechnology's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
