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What is Canavan Disease?

Canavan disease is an inherited disorder caused by mutations in the aspartoacylase (AspA) gene. The disease disrupts the growth or maintenance of the myelin sheath, which covers nerves and promotes efficient transmission of nerve impulses, resulting in damage to brain cells. While this condition occurs in all ethnic backgrounds, it is most common in people of Ashkenazi Jewish heritage.

Therapeutic Strategies

Developing Adeno-Associated Virus (AAV) Mediated Gene Therapy

Dr. Gao’s lab is undertaking two strategies to develop treatments for Canavan disease. First, the lab is actively involved in developing adeno-associated virus (AAV) vector-mediated gene therapy strategies to deliver the AspA gene. Second, they are aiming to understand how aspartoacylase malfunction, or the resulting increase in a compound called N-acetyl-L-aspartic acid, cause extensive brain destruction.

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Canavan Disease Investigator

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

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

What is Canavan Disease?

Therapeutic Strategies

Developing Adeno-Associated Virus (AAV) Mediated Gene Therapy

Canavan Disease Investigator

Guangping Gao, PhD

Clinical Trials

Rare Diseases & Conditions

Technologies

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