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
Michael Flanagan, PhD, chief scientific officer at Avidity
David Barrett, JD, the chief executive officer of ASGCT
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
David Barrett, JD, the chief executive officer of ASGCT
Alfred L. Garfall, MD, MS, associate professor of medicine (hematology-oncology) and director, Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program, Hospital of the University of Pennsylvania; and section chief, Multiple Myeloma, Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania,
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
© 2025 MJH Life Sciences

All rights reserved.