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Treatment Strategies


Developing Therapies to Fix the Faulty MECP2 Gene In Rett Syndrome

Dr. Watts and colleagues are pursuing two kinds of approaches to repair mutations in the MECP2 gene—either in the DNA itself, or in the messenger RNA produced from the MECP2 gene. The DNA-targeted approaches are being explored in collaboration with Drs. Sontheimer, Wolfe and Khvorova. Dr. Watts is also collaborating with Dr. Jacobson on an alternative approach to attempt to “convince” the protein synthesis apparatus of the cell to read through the mutation in MECP2 and produce a correct protein.


Rett Syndrome

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Rett Syndrome Investigators

Guangping Gao, Ph.D.

Guangping Gao, PhD

Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of microbiology and physiological systems, Penelope Booth Rockwell Professor in biomedical Research

Gao Lab

Michael Green

Michael Green, MD, PhD

Chair and professor of molecular, cell & cancer biology, Lambi and Sarah Adams Chair of Genetic Research, Co-director of the Li Weibo Rare Diseases Research Institute, Vice Provost for Strategic Research Initiatives

Green Lab

Jonathan Watts, PhD

Associate professor, RNA Therapeutics Institute

Watts 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.


Research That Gives Hope