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Treatment Strategies


Gene Therapy Strategy for Cockayne Syndrome

There is no cure for Cockayne syndrome at this time, and treatment of the syndrome is focused on managing symptoms and complications. However, there are several UMass Chan labs, including the Sena-Esteves Lab and the Flotte Lab, that are studying gene therapy treatment for genetic diseases that affect children. In particular, UMass Chan researchers Miguel Sena-Esteves, PhD, and Ana Rita Batista, PhD, are currently conducting preclinical research on a CSA gene replacement therapy for Cockayne syndrome. Their work focuses on identifying an adeno-associated virus (AAV) vector candidate and testing its effectiveness and safety in an animal model, with the ultimate goal of proceeding to an AAV gene therapy clinical trial to treat CSA gene mutations in human patients with Cockayne syndrome. Learn more about this gene therapy research here.


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Cockayne Syndrome Investigators

Miguel Sena-Esteves, Ph.D.

Associate Professor, Department of Neurology


terence flotte md

Terence Flotte, MD

Professor of pediatrics, Celia and Isaac Haidak Professor of Medical Education

Flotte Lab

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.