Guangping Gao, Ph.D.


Guangping Gao, Ph.D. is currently the Professor and Director of Horae Gene Therapy Center at University of Massachusetts Medical School. During his 30-plus years of scientific research, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy of human diseases, encompassing disease gene cloning, causative mutation identification, pathomechanisms, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, viral vector manufacturing for preclinical and clinical gene therapy applications, and technology platform development as novel approaches for gene therapy.

More specifically, Dr. Gao’s current research areas include molecular mechanisms of AAV evolution and diversity; AAV capsid structure-tissue tropism-host response correlations; AAV capsid discovery; modifications and vector genome engineering for novel vector development; next generation recombinant AAV (rAAV) manufacturing and quality control testing pipeline development; gene silencing and gene editing for in vivo somatic gene therapy; molecular and immunological interactions between endogenous AAV, rAAV and host, rAAV vectored antibody and other therapeutic biologics production in vivo and minimization of anti-transgene responses; microRNA functional genomics and therapeutic development; nonhuman primate animal modeling for human genetic diseases; preclinical, translational, and clinical development of rAAV gene therapy for CNS disorders such as Canavan disease, an inherited lethal white matter degenerative disorder. 

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ASGCT Policy Summit

September 23-25, 2020