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.
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.
There are more than 7,000 diseases defined as “rare” by the National Institutes of Health, generally those that impact fewer than 200,000 people. Please select disease or condition from the list below for more information about the latest in research, clinical trials and therapy strategies.
Li Weibo Institute for Rare Diseases Research is committed to advancing the research for all the diseases our investigators study. We are continuously investing in the latest technologies, therapeutics, and facilities in order to provide our researchers with the most equipped infrastructure available.
