Xiao Xiao, Ph.D.

Fred Eshelman Distinguished Professor of Gene Therapy

Xiao received a B.S. in pharmaceutical chemistry from the Shanghai Medical University School of Pharmacy in 1982. He received a M.S. in biochemistry in 1985 from Wuhan University in China and a Ph.D. in biology in 1992 from the University of Pittsburgh.

Xiao’s work on muscular dystrophy, especially the Duchenne muscular dystrophy, has received international recognition. His paper published in 2000 on gene therapy for DMD was broadly covered by the media, and his groundbreaking work has been developed into the first gene therapy phase I clinical trial for DMD in the United States. In 2005, his paper on systemic gene delivery for muscular dystrophy was recognized by the National Institutes for Health as a major scientific advancement, and was selected as one of the 100 top scientific stories in 2005 by Discover magazine.

As an independent investigator, Xiao has published 58 peer-reviewed papers from 1998 to 2006. He is the inventor or co-inventor of twelve issued or pending U.S. patents and is also a member on a number of study sections at the National Institutes of Health.

Research

The Xiao lab is interested in molecular medicine, specifically, gene delivery and therapy for various genetic and acquired diseases. The lab genetically engineers a non-pathogenic and defective DNA virus, named adeno-associate virus. The engineered AAV has all of its own genes gutted and replaced by our own genes of interests. As a result, the twenty-two nanometer AAV particles now serve as tiny FedEx/UPS trucks to deliver therapeutic genes to a variety of cells, tissues and even the whole body. Besides its superb efficiency, AAV also offers an excellent safety profile. For example, Xiao lab has developed AAV vectors to treat diseases like muscular dystrophies, heart failure, diabetes, arthritis, hepatitis and cancer, etc. A first of its kind gene therapy for Duchenne muscular dystrophy, a lethal childhood genetic disease, is in a phase I clinical trial.

In addition to gene delivery for therapeutic purposes, AAV can also be used as a powerful tool to study basic biology such as molecular genetics, signal transduction, apoptosis, mechanisms of pathogenesis and even the engineering of animal models. For example, AAV vectors can be used to deliver protein-encoding genes, antisense RNA, small interference RNA (siRNA) or microRNA to tissues like the muscle, heart, liver, pancreas, kidney, lung, brain and spinal cord, etc., to over-express, up-regulate or knockdown a gene or multiple genes for the purposes of dissecting particular molecular pathways, biological functions and immunology consequences and even creating disease models.

Education, Certification and Licensure

  • Ph.D. in Biology, University of Pittsburgh
  • M.S. in Biochemistry, Wuhan University, China
  • B.S. in Pharmaceutical Chemistry, Shanghai Medical University School of Pharmacy, China