MyoAAV-desmin-sKL

MyoAAV-desmin-sKL is an experimental gene therapy candidate being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS). Developed by researchers at the Universitat Autònoma de Barcelona and the Vall d'Hebron Institut de Recerca, the construct employs a specialized myotropic AAV (MyoAAV) capsid designed for efficient muscle transduction following systemic administration. The transgene encodes the secreted form of alpha-Klotho (sKL), a protein known for its anti-aging, anti-inflammatory, and neuroprotective effects. By using the desmin promoter, the therapy restricts sKL expression primarily to skeletal muscle, which then acts as a biofactory to release the protein into circulation or the local neuromuscular environment. Preclinical studies in SOD1G93A mice have shown that this approach improves motor performance, preserves neuromuscular junction innervation, and maintains motor neuron counts, suggesting potential for slowing disease progression in ALS.

Other names

MyoAAV-desmin-Klotho