Ronald Crystal, MD: The Possibilities of Gene Therapy in Neurodegenerative Disease


The chairman of the Department of Genetic Medicine at Weill Cornell Medicine detailed the ongoing research in developing gene therapy for patients with Alzheimer disease.

“What I want people to take away [from this] is to pay attention to this space. This is going to be something real, and there will be selective diseases where this will be a therapy that, over the next decade, will become real for you in terms of treating patients.”

At the recent Alzheimer’s Drug Discovery Foundation International Conference, Ronald G. Crystal, MD, gave a talk to the more than 800 attendees regarding the current state of gene therapy for APOE4 influenced Alzheimer disease and other neurodegenerative disorders. He detailed the ongoing development in the space and offered some insight into the current state of a small trial in patients with Alzheimer.

Crystal, who is the chairman of the Department of Genetic Medicine at Weill Cornell Medicine, urged clinicians to pay attention to this space of therapeutic development, citing the progress that gene therapy has made after decades of research. With regard to Alzheimer, the plan is to provide the APOE2 gene—which literature has suggested may offer a protective effect against Alzheimer disease—to individuals who are APOE4 heterozygotes.

In this interview with NeurologyLive, Crystal offered his takeaways for the clinical community and discussed the current state of that research. This includes detailing the challenges of developing these therapies, particularly as it relates to the differences in approach to intracellular versus extracellular targets.

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