Reviewing the Potential of Gene Therapies to Treat Aging – Fight Aging!

Here find a tour of some of the more high profile projects aimed at the production of gene therapies to treat aging. A lot more could be done than is being done, in large part because present gene therapy vectors have many limitations on the ability to effectively deliver payloads, despite ongoing improvements. They cannot deliver to the whole body in adults. They cannot deliver well to many specific organs without involving direct injections. Delivery is uneven from cell to cell in a tissue. And so forth. These problems are well understood, and many groups are attempting to produce fixes, but for now gene therapies perform well in only some circumstances. For example: permanently increasing circulating amounts of a given signal protein, since the therapy only has to affect a small number of cells in a fat pad following a subcutaneous injection in order to turn them into a factory for that protein.

Gene therapy technology offers transformative potential by enabling precise genetic modifications and targeted delivery to aged tissues. Advances in gene editing tools have revolutionized the modulation of genetic and epigenetic factors associated with aging. Concurrently, optimized delivery systems, including adeno-associated virus (AAV) and lipid nanoparticles (LNPs), enhance targeting efficiency. These advances offer innovative and robust approaches for targeting and modulating aging-related regulatory pathways, promoting a transformative shift in aging intervention from “symptom relief” to “mechanism addressing”, while simultaneously accelerating the research and development process.

The development of gene therapy technologies has provided new avenues for aging intervention, demonstrating unique advantages. Compared to traditional approaches such as drug treatments and lifestyle interventions, gene therapy utilizes delivery vectors to achieve in vivo inhibition or activation of key regulatory genes or pathways involved in aging, offering greater potential for delaying aging and extending healthy lifespan. Here, we systematically elaborate on current research progress in gene therapy for aging intervention from aspects of enhancing genomic and epigenetic stability, maintaining energy metabolism homeostasis, modulating immune functions, and promoting cellular rejuvenation.

Link: https://doi.org/10.1016/j.cellin.2025.100254