Arun Srivastava

Arun Srivastava, PhD

Division Chief, George H. Kitzman Professor

Department: MD-PEDS-CELL / MOLEC THERP DIV
Business Phone: (352) 273-8259
Business Email: aruns@peds.ufl.edu

About Arun Srivastava

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.

Accomplishments

ILBS-TATA Professor of Eminence
2019 · Institute of Liver and Biliary Sciences, New Delhi, India
University of Florida Term Professor
2017 · University of Florida
Children’s Miracle Network Scholar
2013 · Department of Pediatrics, University of Florida
University of Florida Research Foundation Professor
2013 · University of Florida
Honorary Professor and Advisor
2010 · Shenzhen Institute of Xiangya Biomedicine, Central South University, Shenzhen, PR China
Member, Program Project Review Committee
2007 · National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
Member, Scientific Review Board, Gene Therapy Resource Program
2007 · National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
Member, Medical Biochemistry Study Section
1999 · National Institutes of Health, Bethesda, MD
Excellence in Scientific Publication Award
1997 · Sigma Xi Scientific Society
Member, Steering Committee, National Gene Vector Laboratories
1997 · National Gene Vector Laboratories, National Institutes of Health, Bethesda, MD
Member, Scientific Advisory Board
1996 · Immusol, Inc.
Founder and Member, Scientific Advisory Board
1992 · Avigen, Inc.
Member, Medical Advisory Board
1992 · Cancer Research Foundation of America
Established Investigator
1990 · American Heart Association
Overseas Conference Fund Travel Award
1989 · Indiana University School of Medicine, Indianapolis, IN
Medical Research Endowment Award
1984 · University of Arkansas For Medical Sciences, Little Rock, AR
Sreenivasaya Memorial Medal
1979 · Indian Institute of Science, Bangalore, India
Senior Research Fellowship
1975 · University Grants Commission, Government of India
Junior Research Fellowship
1974 · Department of Atomic Energy, Government of India
Best All-Round Student Medal
1971 · Ewing Christian College, University of Allahabad, Allahabad, India
Coondoo Memorial Medal for Academic Excellence
1970 · Ewing Christian College, University of Allahabad, Allahabad, India
Dudgeon Memorial Medal for Academic Excellence
1970 · Ewing Christian College, University of Allahabad, Allahabad, India
Merit Scholar
1967 · UP State Government, India

Research Profile

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.

Open Researcher and Contributor ID (ORCID)

0000-0003-1168-8034

Areas of Interest
  • Adeno-Associated Viral Gene Therapy
  • Hematology
  • Liver Disease

Publications

2020 Human gene therapy
2020 Molecular therapy. Methods & clinical development
2020 Human gene therapy
2019 Molecular Therapy-Methods & Clinical Development
2017 Human gene therapy
2016 Molecular therapy. Methods & clinical development
2016 Molecular therapy. Methods & clinical development
2015 Journal of molecular and genetic medicine : an international journal of biomedical research
2012 Seminars in thrombosis and hemostasis
2011 Molecular therapy : the journal of the American Society of Gene Therapy
2011 Frontiers in microbiology
2009 Molecular therapy : the journal of the American Society of Gene Therapy
2008 Journal of cellular biochemistry

Grants

Aug 2020 ACTIVE
Development of NextGen, GenX, GenY, and GenZ recombinant AAVrh74 vectors
SAREPTA THERAPEUTICS · Principal Investigator
Sep 2019 ACTIVE
Recombinant Adeno associated virus as a long-acting passive antibody vaccine against foreign and transboundary animal diseases
US DEPT OF HOMELAND SECURITY ·
Sep 2019 ACTIVE
STTR – Development of a Novel Therapeutic for Hyperthyroidism
MEDOSOME BIOTEC ·
Aug 2018 – Nov 2019
Development of Clinical Candidate NextGen Optimized AAV3B serotype vectors for gene therapy of hemophilia B and hemophilia A
*AAVECTIVE · Principal Investigator
Jul 2017 – Jun 2020
AAV2 and hepatocellular carcinoma
NATL INST OF HLTH NIAID · Principal Investigator
Jan 2017 ACTIVE
Mechanism of high-efficiency transduction of hepatocytes by optimized AAV vectors
NATL INST OF HLTH NIGMS · Principal Investigator
Aug 2016 – Jul 2018
Development of Optimized AAV Serotype Vectors for Human Gene Therapy
MEDOSOME BIOTEC · Principal Investigator
May 2015 – Apr 2020
NEXT GENERATION OF RECOMBINANT AAV SEROTYPE VECTORS FOR GENE THERAPY
NATL INST OF HLTH NHLBI · Principal Investigator
Jan 2015 ACTIVE
UF Health Cancer Center Bridge Seed Grant funded through the Florida Consortium of National Cancer Institute Centers Program
UF HEALTH SHANDS HOSPITAL · Project Manager
Jul 2014 – Jun 2017
F013607, Cystic Fibrosis Research
UF FOU · Principal Investigator
Jul 2005 ACTIVE
Kitzman Genetic Research
UF FOU · Principal Investigator

Patents

Issued May 2013
Tyrosine-Modified Recombinant AAV Vectors for Human Gene Therapy
#8,445,267
Issued July 2013
PDK Inhibitor Compounds and Methods of Use Thereof
#8,492,344
Published May 2014
Capsid-Modified rAAV3 Vector Compositions and Uses in Gene Therapy of Human Liver Cancer (CIP)
#US2016/0333372
Issued August 2014
Therapeutic Methods Based on Tyrosine-Substituted, Capsid-Modified RAAV Vectors (DIV)
#8,802,440
Published March 2015
Improved rAAV Vectors and Methods for Transduction of Photoreceptors and RPE Cells
#US2016/0369299
Issued October 2015
Improved Transduction Efficiency, Capsid-Modified RAAV Vectors and Methods of Use (CON)
#9,157,098
Published November 2015
Development of Novel Recombinant AAV Vectors Containing Glucocorticoid Receptor-Binding Elements for High-Efficiency Transduction
#US17/0356009
Published February 2016
Recombinant AAV1, AAV5, and AAV6 Capsid Mutants and Uses Thereof
#US2018/0030096
Published February 2016
Recombinant AAV-Parvovirus B19 Hybrid Vectors for Gene Therapy of Human Hemoglobinopathies
#US2018/0135074
Published August 2016
Capsid-Mutated rAAV Vectors and Methods of Use (CON of 15258)
#US2016/0361439
Published February 2017
High-Transduction-Efficiency rAAV Vectors, Compositions, and Methods of Use (DIV)
#US2016/0275337
Issued April 2017
Development of Novel Recombinant AAV Vectors containing Lysine-Mutations for Human Gene Therapy
#9,611,302
Published August 2017
AAV Vectors With High Transduction Efficiency and Uses Thereof For Gene Therapy (CON)
#US2018-0105559
Issued August 2017
Development of Novel Recombinant AAV Vectors containing Lysine-Mutations for Human Gene Therapy
#9,725,485
Published August 2017
Capsid-Modified rAAV Vectors and Methods of Use (CON)
#US2018/0036428
Issued October 2017
Capsid-Modified rAAV Vectors and Methods of Use (CON)
#9,775,918
Published February 2018
Capsid-Modified rAAV Vector Compositions Having Improved Transduction Efficiencies, and Methods of Use (CON)
#US-2018-0244727
Issued March 2018
Activation of the NF-kB Pathway by AAV Vectors and its Implications in Immune Response and Gene Therapy-CIP
#9,920,097
Issued June 2018
A Novel Hairpin mRNA Element for Regulation of Protein Translation
#10,006,049
Issued July 2018
Capsid-Modified rAAV Vector Compositions and Methods Therefor
#10,011,640

Education

PhD
1979 · Indian Institute of Science
MSc
1973 · University of Allahabad
BSc
1971 · University of Allahabad

Contact Details

Phones:
Business:
(352) 273-8259
Emails:
Business:
aruns@peds.ufl.edu