Ronald Crystal, MD: Introducing Genetics into Alzheimer Disease

“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.