Arun Srivastava, PhD

Dr. Arun Srivastava is George H. Kitzman Professor of Genetics and Chief of Division of Cellular & Molecular Therapy in the Departments of Pediatrics, and Molecular Genetics & Microbiology, and Powell Gene Therapy Center. He received his PhD degree from the Indian Institute of Science in Bangalore, India. After completing his postdoctoral training at the Memorial Sloan-Kettering Cancer Center in New York, he worked as a Research Associate in the laboratory of Dr. Kenneth I. Berns at the University of Florida. For nearly two decades, he was on the faculty at Indiana University School of Medicine in Indianapolis, where he rose to the rank of Professor. He was recruited back to the University of Florida in 2004. In the past four decades, he has mentored 10 Clinical Fellows and 35 Postdoctoral Fellows. Four students have graduated with MS degrees, and 13 students have received their PhD degrees from his laboratory. His research activities are currently supported by grants from the National Institutes of Health and the Kitzman Foundation. He has also been awarded 20 US Patents on his research on human parvoviruses and their potential use as vectors in human gene therapy. He currently serves on an NIH Study Section as well as on the Editorial Boards of Journal of Virology, Human Gene Therapy, Recent Patents on DNA and Gene Sequences, Gene Therapy and Molecular Biology, Journal of Integrative Medicine, and Journal of Molecular Cytotherapy. He also serves as Executive Editor of Journal of Genetic Syndromes and Gene Therapy. He has published 140 peer-reviewed research articles, 27 book chapters, and 14 reviews, 14 miscellaneous articles, and 230 abstracts, mostly on human parvoviruses.

Dr. Srivastava’s research has been focused on the following two parvoviruses, the non-pathogenic adeno-associated virus (AAV), and a common human pathogen, the parvovirus B19, and the development of recombinant parvovirus vectors in human gene therapy. His laboratory has made seminal contributions to the field of parvoviruses, which include: identification of cellular co-receptors for AAV2 and AAV3 as well as parvovirus B19; elucidation of various steps involved in parvovirus trafficking in the cell and nuclear transport; identification of cellular proteins involved in the regulation of AAV DNA replication and encapsidation; development of recombinant AAV and parvovirus B19 vectors; and transgenic and knockout mouse models to study parvovirus-induced pathogenicity, and the use of parvovirus vectors for gene transfer and gene therapy. Recently, his laboratory has developed the next generation (“NextGen”) AAV vectors in which the viral capsid has been modified to achieve high-efficiency transduction at significantly reduced vector doses. More recently, his laboratory has also modified the AAV genome to develop the generation X (“GenX”) AAV vectors with which increased transgene expression can be achieved. The NextGen AAV vectors have been used by other investigators in a Phase I Clinical Trial for Leber’s Hereditary Optic Neuropathy (LHON). The NextGen and the GenX vectors have been combined in Dr. Srivastava’s laboratory to develop the optimized [“Opt”] AAV serotype vectors, and the current emphasis is on gene therapy of malignant disorders such as hepatoblastoma and hepatocellular carcinoma, and gene therapy/genome editing of genetic diseases such as hemophilia, β-thalassemia and sickle cell disease.

Dr. Srivastava’s research has been focused on the following two parvoviruses, the non-pathogenic adeno-associated virus (AAV), and a common human pathogen, the parvovirus B19, and the development of recombinant parvovirus vectors in human gene therapy. His laboratory has made seminal contributions to the field of parvoviruses, which include: identification of cellular co-receptors for AAV2 and AAV3 as well as parvovirus B19; elucidation of various steps involved in parvovirus trafficking in the cell and nuclear transport; identification of cellular proteins involved in the regulation of AAV DNA replication and encapsidation; development of recombinant AAV and parvovirus B19 vectors; and transgenic and knockout mouse models to study parvovirus-induced pathogenicity, and the use of parvovirus vectors for gene transfer and gene therapy. Recently, his laboratory has developed the next generation (“NextGen”) AAV vectors in which the viral capsid has been modified to achieve high-efficiency transduction at significantly reduced vector doses. More recently, his laboratory has also modified the AAV genome to develop the generation X (“GenX”) AAV vectors with which increased transgene expression can be achieved. The NextGen AAV vectors have been used by other investigators in a Phase I Clinical Trial for Leber’s Hereditary Optic Neuropathy (LHON). The NextGen and the GenX vectors have been combined in Dr. Srivastava’s laboratory to develop the optimized [“Opt”] AAV serotype vectors, and the current emphasis is on the potential gene therapy of genetic diseases such as β-thalassemia and sickle cell disease, and malignant disorders such as hepatoblastoma and hepatocellular carcinoma.

• Adeno-Associated Viral Gene Therapy
• Hematology
• Liver Disease

0000-0003-1168-8034