Ronald Crystal, MD: Introducing Genetics into Alzheimer Disease

Video

Could the introduction of gene therapies into the Alzheimer space be the beginning of a new era of treatment?

“We’re not genetically modifying the whole individual. We’re genetically modifying 1 organ, where the disease is.”

While gene therapies have traditionally been conceptualized and developed for rare, hereditary disorders, they have begun to trickle into other fields. One of those fields is Alzheimer disease.

Ronald Crystal, MD, The chairman of the Department of Genetic Medicine at Weill Cornell Medicine, is helping to champion a gene therapy there. The difference with Alzheimer, despite it having at least a partial foundation in genetic risk factors, is that it is not a rare condition. This is what excites Crystal about the potential of a gene therapy for the condition.

The advantages of gene therapy as opposed to small molecule or monoclonal antibody treatments, as he puts it, are that they can be specifically directed to a given organ—in this case, the brain.

At the Alzheimer’s Drug Discovery Foundation’s 19th Annual Conference in Jersey City, New Jersey, NeurologyLive sat with Crystal to speak about what is happening with gene therapy in Alzheimer and dementias, and the promise it holds.

Recent Videos
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
© 2025 MJH Life Sciences

All rights reserved.