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Therapeutic Strategies


Gene Therapy to Silence HTT

The labs of Drs. Mueller and Aronin are partnering to develop adeno-associated virus (AAV) vector-based gene therapy approaches using a synthetic microRNA targeting HTT to silence (or turn off) the disease-causing gene.


huntington's disease

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Huntington’s Disease Investigators

Neil Aronin MD

Neil Aronin MD

Professor of medicine, co-director of the Neurotherapeutics Institute

Aronin Lab

John E. Landers, Ph.D.

John E. Landers, Ph.D.

Professor of Neurology

Landers Lab

Miguel Sena-Esteves, Ph.D.

Associate Professor, Department of Neurology


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.


Research That Gives Hope