As described in today’s front-page New Yorker article, entitled How an Ultra-Rare Disease Accelerates Aging, The Progeria Research Foundation is thrilled to announce the pioneering Progeria Gene Editing Program.
“I’ve been involved with PRF most of my life”, says 22-year-old Kaylee from Ohio, who is prominently featured in the New Yorker article. “I’m really excited about the gene therapy they’re working on – when it’s ready, so am I!”
Base editing therapy uses CRISPR technology to target and correct a specific DNA base (or “letter”) in the genetic code. Because Progeria is caused by a change to a single letter in the DNA, it is perfectly suited for this type of gene therapy.
The Progeria Gene Team
PRF has been collaborating with an extraordinary team of world-renowned scientists and innovators. The team’s tireless work and dedication is bringing this transformative therapy to reality:
Top Left to Bottom Right:
Dr. Francis Collins, doctor en medicina – Longtime Progeria research partner and former Director of the National Institutes of Health (NIH)
Doctor Michael Erdos – Associate Investigator, Molecular Genetics Section, National Human Genome Research Institute (NHGRI)
Dra. Leslie Gordon, doctora en medicina – Co-founder & Medical Director of PRF, and the world’s foremost expert on Progeria
David Liu, doctor – MIT’s Broad Institute and Harvard University scientist, founder of Base Editing Gene Therapy Technology
*Sammy Basso, MS, Co-Founder of The Associazione Italiana Progeria Sammy Basso and PRF Ambassador, was an integral part of this team until his passing in October 2024.
From Mouse Model to Clinical Trials
The Progeria Gene Team and their lab members first demonstrated that gene editing could dramatically increase the lifespan of the Progeria mouse model — by an astounding 140% — a discovery that was published in one of the world’s top scientific journals, Naturaleza. Building on this remarkable achievement, the team has refined the drug and is now focused on steps needed to start a clinical trial: drug manufacturing, engaging with the US FDA, and developing clinical trial protocols.
The journey ahead will be multi-stage, technically complicated, and expensive. PRF is determined to meet every challenge to bring gene therapy to children and young adults with Progeria.
A Community-Driven Mission
The Progeria Community has been with us every step of the way. PRF’s leadership team regularly hosts online meetings with Progeria families throughout the world, so they are our partners on this journey toward the cure.
Moreover, the implications of this work may extend far beyond Progeria. In addition to curing Progeria, we are forging a path that other rare diseases can follow. Thus, this could be a groundbreaking leap forward not only for Progeria, but for other conditions that have long been overlooked.
“The hope of our team is to use base editing to fix the root cause of progeria, providing a pathway to a potential cure for children with progeria, as well as advancing treatments for millions of other children with fatal genetic diseases.”
David Liu, 2025 Breakthrough Prize Laureate in the Life Sciences for the development of base editing and prime editing, with Sammy Basso at the Brilliant Minds Conference in Sweden in 2023. They received a standing ovation for their presentation on base editing and progeria.
Your Support is Vital
Gene therapy is the most promising and the most expensive type of drug to manufacture. The unwavering commitment of PRF’s passionate supporters like you will make Progeria gene editing a reality.
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