Welcome to the Gao Lab for Gene Therapy
Development and translation of viral gene therapy systems for the treatment of disorders of CNS and other organs
Our lab focuses on the development of recombinant Adeno-associated virus (rAAV) capsids and genome design to lay the foundation for translational research, such as development of treatment for different diseases with or without primary genetic cause. Although, Canavan disease (CD), Amyotrophic lateral sclerosis (ALS) and Rett syndrome are CNS disorders, we are also pursuing treatment development for diseases such as hypercholesterolemia.
Canavan disease (CD) is a fatal inherited disorder of the central nervous system caused by mutations in the aspartoacylase (ASPA) gene, leading to accumulation of N-acetylaspartate (NAA). First symptoms start shortly after birth or develop during the first years of life. Research in Gao lab focuses on two aspects of this disease: Development of rAAV based gene therapy (can we have subgroups for this?) and to understand the pathomechanism (can we have subgroups for this?) of this disease.
Partner with Us
The Gao Lab focus on platform development and treatment of different diseases offers great opportunities for partnerships with individuals, organizations and industry. For any interest in partnering with us, feel free to contact us at any time with questions and suggestions. Read more