Telomerase gene therapy (AAV-TERT)
Mechanism, regulatory status, and an honest, tiered evidence map.
What it is
Class: Adeno-associated-virus vector expressing TERT (preclinical)
Also known as: AAV9-Tert, AAV-TERT, telomerase gene therapy
Relationship to telomere biology: Telomerase gene therapy delivers the TERT gene via an AAV vector so that treated cells express telomerase and extend their telomeres. It is the intervention behind the striking mouse-lifespan result — but there is NO human evidence, so it sits at animal grade only.
Regulatory status
Preclinical/experimental. There is no approved telomerase gene therapy and no human trial for aging. Any human application would face the telomerase–cancer-risk question directly.
Mechanism
In mice, a single AAV9-TERT treatment in adult/old animals lengthened telomeres, delayed several aging phenotypes, and increased lifespan without a reported rise in cancer incidence (Bernardes de Jesus/Blasco 2012); a related mouse study showed benefit in a short-telomere pulmonary-fibrosis model (Povedano/Blasco 2018). See /telomerase-cancer-paradox.
Evidence — Animal (mouse lifespan / disease model)
| Species / population | Adult (1-year) and old (2-year) mice; and a separate bleomycin/short-telomere mouse model of pulmonary fibrosis. |
| Exposure, route, schedule | Single intravenous dose of an AAV9 vector expressing mouse TERT (AAV9-Tert). |
| Comparator / duration | AAV9 empty-vector / untreated controls. |
| Endpoint / numeric result | Median lifespan increased ~24% when treated at 1 year and ~13% when treated at 2 years, with delayed aging markers and NO reported increase in cancer incidence (2012); improved lung function and reduced fibrosis in the pulmonary-fibrosis model (2018). |
| What it did NOT establish | These are mouse results. Mouse telomere biology differs substantially from human; there is no human lifespan, disease-outcome, or safety evidence, and the cancer-risk question is unresolved in humans. |
Negative or null findings
- No human data of any kind — the entire evidence base is preclinical (mouse).
- Mouse telomeres are much longer than human telomeres and mouse cells regulate telomerase differently, so the lifespan result does not transfer to a human claim.