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Treatment Strategies


Gene Therapy Strategy for Tay-Sachs Disease

The Sena-Esteves Lab is working on devising new ways to deliver therapeutic levels of the missing enzyme HexA to the entire brain by injection of adeno-associated virus (AAV) vectors into specific structures in the central nervous system. Another research area of the lab focuses on developing new AAV vectors capable of crossing the blood-brain barrier for effective gene delivery to the brain after intravascular infusion.


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Tay-Sachs Investigators

Miguel Sena-Esteves, Ph.D.

Associate Professor, Department of Neurology


Heather Gray-Edwards DVM, PhD

Assistant professor of radiology


Clinical Trials

At the Li Weibo Institute for Rare Diseases Research, our researchers and clinicians are dedicated to finding new treatment options for patients. Our clinical trials program is another way we continue to advance treatments and make new discoveries. If you or someone you know may be interested in a clinical trial, please subscribe for updates and you will be notified when we open up a new trial.