Gene therapy for ageing
Frontier · Evidence and recent research
Notify me when meaningful evidence changes.
Where things stand
Gene therapy changes gene activity or the properties of cells to treat a particular condition. Approved applications exist for specific diseases, but their success cannot be transferred automatically to experimental ageing targets. For longevity, the relevant questions are which tissue and pathway are changed, whether people gain meaningful health benefits, and what risks persist after treatment. Source ↗ Recently: No update dated within the past 30 days is recorded in our collection. This describes our coverage, not a conclusion that the wider research is unchanged.
AI-written overview · not expert-reviewed · Background sources checked Sep 23, 2026 · Recent updates cover the last 30 days of this collection. A new report does not automatically change the evidence assessment.
Background and coverage
FDA: cellular and gene therapies ↗
- Regulatory status
- Investigational
- Availability
- Not available
- Category
- Frontier
- Human results verified here
- 0
Evidence collection is needs review. 0 approved assessments; 18 assessments awaiting review; 2 published studies with verified results. Safety: not assessed.
Recorded evidence activity (experimental)
A numeric breakdown will be available after baseline review. Safety is not yet assessed.
What changed
Evidence events affecting this intervention
Evaluating the Safety and Efficacy of Injectable Combination Klotho and Follistatin Plasmid Gene Therapy in Humans -- An Interventional, Non-Placebo Controlled Pilot Phase Study
Trial registry update: COMPLETED. No results reported in the supplied registry record.
Evaluating the Safety and Efficacy of Injectable Klotho Plasmid Gene Therapy in Humans: An Interventional, Non-Placebo-Controlled Pilot Phase Study
Trial registry update: ACTIVE_NOT_RECRUITING. No results reported in the supplied registry record.
SIRT6 activator MDL-800 reduced osteoarthritis severity in aging mice given monthly injections.
Male mice aged 18 months received weekly intraperitoneal injections of MDL-800 (a SIRT6 activator) or vehicle for 6 months, then were assessed for osteoarthritis at 24 months. MDL-800-treated mice showed less cartilage damage, osteophyte formation, bone sclerosis, and meniscal ossification compared to controls. Mechanistic studies in cultured human chondrocytes showed MDL-800 suppressed catabolic signaling pathways and reduced DNA damage.
HMGA1-HP1β peptide therapy extended lifespan in a mouse model of Hutchinson-Gilbert progeria syndrome
Researchers studied the HMGA1-HP1β regulatory axis in a mouse model of Hutchinson-Gilford progeria syndrome (LmnaG609G/G609G mice). They developed a peptide called UHP based on the HMGA1-HP1β binding region and found it reduced cellular senescence markers and significantly extended lifespan in the progeria mice, reportedly by stabilizing HP1β and suppressing senescence-associated secretory factors.
Injectable klotho-follistatin plasmid gene therapy assessed for safety in healthy adults in uncontrolled pilot study
This pilot study investigated the safety and efficacy of a combination klotho and follistatin gene therapy delivered via nonviral plasmid injection in healthy adult volunteers. The study sought to evaluate both safety outcomes and potential cognitive and health benefits, though no specific results are reported in the abstract.
Assessment history
The 25 most recent recorded changes, including corrections and backfill
Human studies with verified results
No published human results verified in this collection yet
Animal and mechanistic evidence
Clinical trials
Claims
Testable statements and where the evidence stands