klotho gene therapy

Klotho gene therapy is an investigational genetic treatment designed to increase expression of the Klotho protein, a key regulator of aging and cognitive function. The Klotho gene encodes two main protein variants: full-length (primarily renal) and secreted (s-KL), with the latter being crucial for brain health. Therapeutic approaches include delivery via adeno-associated virus (AAV) vectors or plasmids to induce endogenous production of secreted Klotho in target tissues such as muscle or neurons. Preclinical and early clinical development focus on neurodegenerative diseases like amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, Huntington’s disease, spinal cord injury, and stroke[2][6][10]. Mechanistically, increased Klotho levels are associated with inhibition of Wnt/β-catenin signaling (reducing fibrosis and promoting tissue regeneration), suppression of insulin-like growth factor 1 signaling (reducing oxidative stress), modulation of glutamate transporters to prevent excitotoxicity in neurons, enhancement of synaptic plasticity and neurogenesis via FOXO transcription factors/Akt pathway regulation[4][5]. Multiple companies are developing proprietary versions using different delivery platforms.

Other names

secreted alpha Klotho gene therapys-KL gene therapyKlotho plasmid gene therapy