AAV-mediated BDNF and GAS6 muscle delivery delays disease onset in SOD1^G93A ALS mice.

ABSTRACT: Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease, with limited treatments. Gene therapy offers an alternative strategy for treating a large portion of ALS patients, however, the disparate genetic alterations in ALS complicate the development of gene therapies. Tyrosine receptor kinase B (TRKB) and Tyro3 receptors are highly expressed in mouse spinal cord motor neurons, suggesting that their ligands, brain-derived neurotrophic factor (BDNF) and growth arrest-specific 6 (GAS6), respectively, are crucial for neuronal survival. In this study, we tested whether genetically induced and muscle tissue-specific expression of such survival-enhancing ligands would ameliorate symptom development in the SOD1^G93A ALS mouse model. The therapeutic vectors (AAV-P_mus<

SUBMITTER: Le Y 

PROVIDER: S-EPMC13472854 | biostudies-literature | 2026 Jul

REPOSITORIES: biostudies-literature