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Therapeutics

TERT gene therapy

DETERT-Gentherapie

TERT (telomerase reverse transcriptase) is the business end of telomerase, the enzyme that rebuilds your chromosome caps (telomeres). In most adult human tissues, TERT is switched off, so telomeres shorten with each cell division. TERT gene therapy tries to switch it back on. Delivered by AAV, TERT transgenes have extended median lifespan, delayed frailty, and improved metabolic and neuromuscular health in middle-aged and old mice (work from the Blasco lab). Strikingly, a single dose had lasting effects without raising cancer rates in those mouse models. For humans, this is still preclinical. The central safety worry: switching on telomerase might fuel tumors, by giving cells with cancer-causing mutations unlimited ability to divide. That concern is not resolved in human data. And self-administered TERT gene therapy outside regulated trials (reported around BioViva/Andrews) has no peer-reviewed safety or efficacy data, and represents an extreme risk.

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Sources

  1. Bernardes de Jesus B, Vera E, Schneeberger K et al.. (2012). Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer. *EMBO Molecular Medicine*doi:10.1002/emmm.201200245