Correction: Vyas et al. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Int. J. Mol. Sci. 2023, 24, 14611

Puntos clave

• To assess the efficacy of adeno-associated virus serotype 9 (AAV9) gene therapy in treating AB-variant GM2 gangliosidosis.
• Utilization of AAV9 vector for gene delivery
• Evaluation of neurological function in affected models
• Analysis of biochemical markers post-treatment
• Significant improvements in neurological function observed
• Reduction in GM2 ganglioside levels indicates successful gene therapy
• Potential for improved quality of life after treatment