Nicholas Muzyczka

Nicholas Muzyczka

University of Florida College of Medicine


Dr. Muzyczka received his PhD in biochemistry from Johns Hopkins University in 1974. He then began working on the development of gene therapy vectors. In 1984, he published the first general purpose AAV vector for long term expression in mammalian cells and subsequently secured the first patent involving non-AAV genes inserted into AAV. His laboratory discovered the functions of the AAV Rep protein and studied the mechanisms of AAV DNA replication, integration and transcriptional control. This included mapping the AAV terminal repeat, reconstitution of AAV DNA replication in vitro, identification of transcription control elements, and characterization of capsid assembly and trafficking. His laboratory has also collaborated on work that lead to potential therapies for neurodegenerative, pulmonary, cardiovascular and eye diseases, and he has used AAV vectors to create animal models of learning and memory defects and Parkinson disease. In 1994, he returned to the UF College of Medicine, where he became the founding Director of the UF Powell Gene Therapy Center and oversaw the development of many of the standard techniques that are currently used to manufacture AAV vectors. In 2001, Dr. Muzyczka and his colleagues founded AGTC, a company designed to commercialize gene therapy applications. He was the founding CEO and Chairman of the Board of AGTC, and continues to serve as a scientific consultant. He is an inventor on over 20 issued or pending patents related to AAV vector technology and has served on the Board of Directors of the American Society for Gene Therapy and the NIH Recombinant Advisory Committee.


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