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 at the University of Florida College of Medicine, in Gainesville, Florida, USA.
He has worked with AAV and AAV vectors for nearly 43 years.
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 over four decades, he has mentored 52 Postdoctoral and Clinical Fellows. Four students have graduated with MS degrees, and 10 students have received their PhD degrees from his laboratory.
He has received uninterrupted research funding for 38 years from the National Institutes of Health (NIH), and his current research is supported by grants from the NIH, Pfizer, Children’s Miracle Network, and the Kitzman Foundation. He has also been awarded 21 US Patents with 21 additional US patent applications that have been filed on his research on AAV 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 Molecular Therapy, Human Gene Therapy, Journal of Virology, Journal of Gene Regulation, International Journal of Molecular Sciences, and Frontiers in Immunology, and Journal of Integrative Medicine. He also serves as Executive Editor of Journal of Genetic Syndromes & Gene Therapy, and Associate Editor of Vaccines & Molecular Therapeutics.
He has published 214 peer-reviewed research articles, book chapters, reviews, and miscellaneous articles, and 250 abstracts.
He was the founding scientist of the very first AAV gene therapy company, Avigen, which was launched in 1992. He is also a co-founder of an additional AAV gene therapy company, Lacerta Therapeutics, which was launched in 2017.
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. 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/II Clinical Trial for Leber’s Hereditary Optic Neuropathy (LHON). The NextGen and the GenX vectors have been combined in his laboratory to develop the optimized (“OptX”) AAV serotype vectors that are more efficient at further reduced doses. Some of these AAV vectors have been licensed to various gene therapy companies.
Recently, his laboratory has developed generation Y (”GenY”) and “OptY” AAV vectors that are transcriptionally more efficient.. More recently, his laboratory has also developed generation Z (”GenZ”) AAV vectors that overcome the major rate-limiting step of viral second-strand DNA synthesis, and lead to robust transgene expression from single-stranded AAV serotype vectors.
His laboratory has also reported the development of AAV vectors that are capable of dampening the host humoral immune response as well. Efforts are currently to develop the ultimate (“Ult”) AAV vectors that may prove to be more efficient, less immunogenic, and capable of repeat-dosing.
His laboratory has also identified the remarkable tropism of two AAV vectors for primary human cells: AAV3 for liver, and AAV6 for hematopoietic stem cells. The current emphasis of his research is on gene therapy of genetic diseases such as hemophilia and muscular dystrophies; gene therapy of malignant disorders such as hepatoblastoma and hepatocellular carcinoma; and gene therapy and “nuclease-free” genome editing for 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
Teaching Profile
Courses Taught
2014,2016-2018,2018-2021,2020-2024
GMS6252 Molecular Therapy II ? Disease Targets and Applications
2016-2018,2020-2023
GMS6064 Tumor Biology
2018
GMS7980 Research for Doctoral Dissertation
2018
GMS7979 Advanced Research
2012
GMS5905 Special Topics in Biomedical Sciences
2009
IDH4917 Undergrad Research
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.
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. His laboratory has also modified the AAV genome to develop generation X (“GenX”) generation Y (“GenY”), and generation Z (“GenZ”) AAV vectors with which significantly 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 capsids and the GenX and GenY genomes have been combined in Dr. Srivastava’s laboratory to develop the optimized [“OptX” and “OptY”] AAV serotype vectors, vectors that are more efficient at further reduced doses. Some of these AAV vectors have been licensed to various gene therapy companies.
His laboratory has also reported the development of AAV vectors that are capable of dampening the host humoral immune response as well. Efforts are currently to develop the ultimate (“Ult”) AAV vectors that may prove to be more efficient, less immunogenic, and capable of repeat-dosing.
His laboratory has also identified the remarkable tropism of two AAV vectors for primary human cells: AAV3 for liver, and AAV6 for hematopoietic stem cells. The current emphasis of his research is on gene therapy of genetic diseases such as hemophilia and muscular dystrophies; gene therapy of malignant disorders such as hepatoblastoma and hepatocellular carcinoma; and gene therapy and “nuclease-free” genome editing for β-thalassemia and sickle cell disease.
Areas of Interest
- Adeno-Associated Viral Gene Therapy
- Hematology
- Liver Disease
Open Researcher and Contributor ID (ORCID)
0000-0003-1168-8034
Publications
2023
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
Gene therapy.
30(9)
[DOI] 10.1038/s41434-023-00412-2.
[PMID] 37495717.
2023
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy
Molecular Therapy – Methods & Clinical Development.
31
[DOI] 10.1016/j.omtm.2023.101147.
2023
Nicholas Muzyczka, PhD [1947-2023].
Human gene therapy.
34(3-4):81-82
[DOI] 10.1089/hum.2023.29235.nmu.
[PMID] 36794979.
2023
Nicholas Muzyczka, PhD.
Molecular therapy : the journal of the American Society of Gene Therapy.
31(3)
[DOI] 10.1016/j.ymthe.2023.01.027.
[PMID] 36764298.
2023
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
Molecular Therapy – Nucleic Acids.
32:949-959
[DOI] 10.1016/j.omtn.2023.05.014.
[PMID] 37293185.
2022
Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children.
Frontiers in microbiology.
13
[DOI] 10.3389/fmicb.2022.1033615.
[PMID] 36274690.
2021
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette.
Molecular therapy. Methods & clinical development.
23:98-107
[DOI] 10.1016/j.omtm.2021.08.001.
[PMID] 34631930.
2021
Prevalence of Adeno-Associated Virus 3 Capsid Binding and Neutralizing Antibodies in Healthy and Hemophilia B Individuals from India.
Human gene therapy.
32(9-10):451-457
[DOI] 10.1089/hum.2020.258.
[PMID] 33207962.
2021
Reply to “D” matters in recombinant AAV packaging.
Molecular therapy : the journal of the American Society of Gene Therapy.
29(9):2628-2630
[DOI] 10.1016/j.ymthe.2021.08.012.
[PMID] 34437839.
2021
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
Gene therapy.
28(7-8):447-455
[DOI] 10.1038/s41434-020-00206-w.
[PMID] 33244179.
2020
A Tribute to Barrie J. Carter.
Human gene therapy.
31(9-10):491-493
[DOI] 10.1089/hum.2020.29118.bca.
[PMID] 32239977.
2020
AAV Vectors: Are They Safe?
Human gene therapy.
31(13-14):697-699
[DOI] 10.1089/hum.2020.187.
[PMID] 32611206.
2020
Adeno-Associated Virus D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response.
Human gene therapy.
31(9-10):565-574
[DOI] 10.1089/hum.2020.018.
[PMID] 32220217.
2020
Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B.
Human gene therapy.
31(19-20):1114-1123
[DOI] 10.1089/hum.2020.099.
[PMID] 32657150.
2020
Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing.
Molecular therapy. Nucleic acids.
20:451-458
[DOI] 10.1016/j.omtn.2020.03.009.
[PMID] 32276210.
2020
Role of Essential Metal Ions in AAV Vector-Mediated Transduction.
Molecular therapy. Methods & clinical development.
18:159-166
[DOI] 10.1016/j.omtm.2020.05.019.
[PMID] 32637447.
2020
Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors.
Molecular therapy. Methods & clinical development.
17:545-555
[DOI] 10.1016/j.omtm.2020.03.007.
[PMID] 32258217.
2020
When the Capsid Meets the Promoter: A New Insight into Transgene Expression from Adeno-Associated Virus Vectors.
Human gene therapy.
31(21-22):1144-1145
[DOI] 10.1089/hum.2020.29138.asr.
[PMID] 33200964.
2019
Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors.
Molecular therapy. Methods & clinical development.
12:248-265
[DOI] 10.1016/j.omtm.2019.01.008.
[PMID] 30815511.
2019
Chinese Medicine Protein and Peptide in Gene and Cell Therapy.
Current protein & peptide science.
20(3):251-264
[DOI] 10.2174/1389203719666180612082432.
[PMID] 29895243.
2019
Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases.
Gastroenterology clinics of North America.
48(2):319-330
[DOI] 10.1016/j.gtc.2019.02.005.
[PMID] 31046978.
2019
XVIIth International Parvovirus Workshop.
Human gene therapy.
30(3):252-256
[DOI] 10.1089/hum.2019.29084.mam.
[PMID] 30807259.
2017
AAV Infection: Protection from Cancer.
Human gene therapy.
28(4):323-327
[DOI] 10.1089/hum.2016.147.
[PMID] 27832705.
2017
Authors’ Response to Jesse D. Riordan, Hum Gene Ther 2017;28:375-376; DOI: 10.1089/hum.2017.045.
Human gene therapy.
28(5):376-377
[DOI] 10.1089/hum.2017.050.
[PMID] 28498777.
2017
Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors.
Human gene therapy methods.
28(1):49-59
[DOI] 10.1089/hgtb.2016.089.
[PMID] 28125909.
2017
Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.
Journal of translational medicine.
15(1)
[DOI] 10.1186/s12967-017-1200-1.
[PMID] 28460646.
2017
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells.
Blood.
129(24):3184-3195
[DOI] 10.1182/blood-2016-11-751040.
[PMID] 28468798.
2017
The hepatocyte-specific HNF4α/miR-122 pathway contributes to iron overload-mediated hepatic inflammation.
Blood.
130(8):1041-1051
[DOI] 10.1182/blood-2016-12-755967.
[PMID] 28655781.
2016
Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector.
Human gene therapy.
27(1):1-6
[DOI] 10.1089/hum.2015.29017.asr.
[PMID] 26784640.
2016
Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses.
Human gene therapy methods.
27(4):143-9
[DOI] 10.1089/hgtb.2016.054.
[PMID] 27431826.
2016
High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing.
Scientific reports.
6
[DOI] 10.1038/srep35495.
[PMID] 27759036.
2016
In vivo tissue-tropism of adeno-associated viral vectors.
Current opinion in virology.
21:75-80
[DOI] 10.1016/j.coviro.2016.08.003.
[PMID] 27596608.
2016
Microglia-specific targeting by novel capsid-modified AAV6 vectors.
Molecular therapy. Methods & clinical development.
3
[DOI] 10.1038/mtm.2016.26.
[PMID] 27308302.
2016
Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors.
Molecular therapy. Methods & clinical development.
3
[DOI] 10.1038/mtm.2016.29.
[PMID] 27200382.
2016
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.
Molecular therapy : the journal of the American Society of Gene Therapy.
24(6):1042-1049
[DOI] 10.1038/mt.2016.61.
[PMID] 27019999.
2015
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?
Human gene therapy.
26(12):779-81
[DOI] 10.1089/hum.2015.29014.kib.
[PMID] 26690810.
2015
Enhanced Transgene Expression From Recombinant Single-Stranded D-Sequence-Substituted Adeno-Associated Virus Vectors in Human Cell Lines in Vitro and in Murine Hepatocytes in Vivo
Journal Of Virology.
89(2):952-961
[DOI] 10.1128/JVI02581-14.
2015
Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.
Journal of virology.
89(2):952-61
[DOI] 10.1128/JVI.02581-14.
[PMID] 25355884.
2015
Prevalence of neutralizing antibodies against liver-tropic adeno-associated virus serotype vectors in 100 healthy Chinese and its potential relation to body constitutions.
Journal of integrative medicine.
13(5):341-6
[DOI] 10.1016/S2095-4964(15)60200-X.
[PMID] 26343106.
2015
Productive life cycle of adeno-associated virus serotype 2 in the complete absence of a conventional polyadenylation signal.
The Journal of general virology.
96(9):2780-2787
[DOI] 10.1099/jgv.0.000229.
[PMID] 26297494.
2015
Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads to Improved Transduction by AAV2, But Not AAV8, Vectors in Murine Hepatocytes In Vivo.
Human gene therapy methods.
26(6):211-20
[DOI] 10.1089/hgtb.2015.115.
[PMID] 26421998.
2015
The Adeno-Associated Virus Genome Packaging Puzzle.
Journal of molecular and genetic medicine : an international journal of biomedical research.
9(3)
[PMID] 26949410.
2014
Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo.
Journal of integrative medicine.
12(6):483-94
[DOI] 10.1016/S2095-4964(14)60057-1.
[PMID] 25412666.
2014
Efficient lysis of epithelial ovarian cancer cells by MAGE-A3-induced cytotoxic T lymphocytes using rAAV-6 capsid mutant vector.
Vaccine.
32(8):938-43
[DOI] 10.1016/j.vaccine.2013.12.049.
[PMID] 24406390.
2014
Lignin nanotubes as vehicles for gene delivery into human cells.
Biomacromolecules.
15(1):327-38
[DOI] 10.1021/bm401555p.
[PMID] 24308459.
2014
Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo.
Journal of integrative medicine.
12(1):20-34
[DOI] 10.1016/S2095-4964(14)60003-0.
[PMID] 24461592.
2014
Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector.
Human gene therapy methods.
25(1):72-82
[DOI] 10.1089/hgtb.2013.011.
[PMID] 24191859.
2014
Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model.
Human gene therapy.
25(12):1023-34
[DOI] 10.1089/hum.2014.099.
[PMID] 25296041.
2014
Template-Synthesized Lignin Nanotubes as Vehicles for Smart Delivery of Dna Into Human Cells
.
247
2013
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.
Blood.
121(12):2224-33
[DOI] 10.1182/blood-2012-10-460733.
[PMID] 23325831.
2013
Functional Roles of the Aav2 Inverted Terminal Repeat in Messenger Rna Transport and Transgene Expression
Molecular Therapy.
21:S30-S31
2013
High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo.
PloS one.
8(3)
[DOI] 10.1371/journal.pone.0058757.
[PMID] 23516552.
2013
High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression By Optimized Scaav6 Serotype Vectors in Vitro and in a Murine Xenograft Model in Vivo
Molecular Therapy.
21:S126-S127
2013
High-Efficiency Transduction of Primary Human Monocyte-Derived Dendritic Cells By Recombinant Aav6 Vectors Containing Mutations in Surface-Exposed Serine and Threonine Residues
Molecular Therapy.
21
2013
Molecular cloning, overexpression, and an efficient one-step purification of α5β1 integrin.
Protein expression and purification.
92(1):21-8
[DOI] 10.1016/j.pep.2013.08.013.
[PMID] 23993979.
2013
Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?
PloS one.
8(3)
[DOI] 10.1371/journal.pone.0059142.
[PMID] 23527116.
2013
Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 Vectors: the Final Threshold?
Molecular Therapy.
21
2013
Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo.
Cytotherapy.
15(8):986-98
[DOI] 10.1016/j.jcyt.2013.04.003.
[PMID] 23830234.
2013
Pristimerin Enhances Recombinant Adeno-Associated Virus Serotype 2 Vector-Mediated Transgene Expression Both in Vitro and in Vivo
Molecular Therapy.
21
2013
Productive Life Cycle of the Aav2 in the Complete Absence of a Conventional Polyadenylation Signal
Molecular Therapy.
21
2013
Regulation of Host Cellular Dna Binding Proteins Augment the Gene Transfer Efficiency of Adeno-Associated Virus (Aav) Vectors
Molecular Therapy.
21
2013
Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads To Improved Transduction By Recombinant Aav2 and Aav8 Vectors in Murine Hepatocytes in Vivo
Molecular Therapy.
21:S208-S209
2013
Systemic Gene Transfer in Neonatal Dogs Using Tyrosine Mutant Aav-1,-6 And-9
Molecular Therapy.
21
2012
A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs.
Human gene therapy.
23(2):202-9
[DOI] 10.1089/hum.2011.147.
[PMID] 21967249.
2012
Activation of the Cellular Unfolded Protein Response By Recombinant Adeno-Associated Virus Vectors: Implications in Gene Therapy
Molecular Therapy.
20
2012
Bio-Engineering of Adeno-Associated Virus Serotype (Aav)-2 Capsid At Serine/Threonine Residues Improves Its Transduction Efficiency Both in Vitro and in Vivo
Molecular Therapy.
20:S47-S48
2012
Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia.
Journal of genetic syndromes & gene therapy.
S1
[PMID] 23264889.
2012
Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells.
Gene therapy.
19(4):375-84
[DOI] 10.1038/gt.2011.105.
[PMID] 21776025.
2012
High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors.
Vaccine.
30(26):3908-17
[DOI] 10.1016/j.vaccine.2012.03.079.
[PMID] 22497875.
2012
High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells By Optimized Aav6 Serotype Vectors and Erythroid Lineage-Restricted Transcriptional Targeting Using the Human Parvovirus B19 Promoter
Molecular Therapy.
20:S48-S49
2012
High-Efficiency Transduction of Primary Human Monocyte-Derived Dendritic Cells By Recombinant Adeno-Associated Virus 2 Vectors Containing Mutations in Surface-Exposed Serine and Threonine Residues
Molecular Therapy.
20:S144-S145
2012
High-Efficiency Transduction of Primary Human Monocyte-Derived Dendritic Cells By Recombinant Adeno-Associated Virus 2 Vectors Containing Mutations in Surface-Exposed Serine and Threonine Residues
Molecular Therapy.
20:S144-S145
2012
High-Efficiency Transgene Expression in Monocyte-Derived Dendritic (Modcs) Mediated By Recombinant Adeno-Associated Virus Vectors Containing Short Subsets of Human Cd11C Promoter
Molecular Therapy.
20
2012
Limitations of Encapsidation of Recombinant Scaav2 Genomes in Different Serotype Capsids and Their Quantitation
Molecular Therapy.
20
2012
Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation.
Human gene therapy methods.
23(4):225-33
[DOI] 10.1089/hgtb.2012.090.
[PMID] 22966785.
2012
Mechanism of Transduction of Human Hematopoietic Cells By Recombinant Aav6 Vectors
Molecular Therapy.
20:S229-S230
2012
Mechanism of Transduction of Human Hematopoietic Cells By Recombinant Aav6 Vectors
Molecular Therapy.
20:S229-S230
2012
Role of molecular genetics in hemophilia: from diagnosis to therapy.
Seminars in thrombosis and hemostasis.
38(1):64-78
[DOI] 10.1055/s-0031-1300953.
[PMID] 22314605.
2012
Site-Directed Mutagenesis of Surface-Exposed Tyrosine, Serine, and Threonine Residues Leads To High-Efficiency Transduction By Recombinant Adeno-Associated Virus 2 Vectors
Molecular Therapy.
20
2011
A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo.
Human gene therapy.
22(5):633-40
[DOI] 10.1089/hum.2010.243.
[PMID] 21219084.
2011
Cellular fusion for gene delivery to SCA1 affected Purkinje neurons.
Molecular and cellular neurosciences.
47(1):61-70
[DOI] 10.1016/j.mcn.2011.03.003.
[PMID] 21420496.
2011
High-efficiency transduction of liver cancer cells by recombinant adeno-associated virus serotype 3 vectors.
Journal of visualized experiments : JoVE.
(49)
[DOI] 10.3791/2538.
[PMID] 21445055.
2011
Innate Immune Responses to AAV Vectors.
Frontiers in microbiology.
2
[DOI] 10.3389/fmicb.2011.00194.
[PMID] 21954398.
2011
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina.
Molecular therapy : the journal of the American Society of Gene Therapy.
19(2):293-301
[DOI] 10.1038/mt.2010.234.
[PMID] 21045809.
2011
Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency.
Human gene therapy.
22(8):935-49
[DOI] 10.1089/hum.2010.121.
[PMID] 21142972.
2010
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle.
Human gene therapy.
21(10):1343-8
[DOI] 10.1089/hum.2010.003.
[PMID] 20497037.
2010
Adeno-Associated Virus-Mediated Knockdown of Pyruvate Dehydrogenase E1 Alpha in Specific Regions of the Rat Brain
Mitochondrion.
10
[DOI] 10.1016/j.mito.2009.12.01.
2010
An animal model of PDH deficiency using AAV8-siRNA vector-mediated knockdown of pyruvate dehydrogenase E1α.
Molecular genetics and metabolism.
101(2-3):183-91
[DOI] 10.1016/j.ymgme.2010.07.008.
[PMID] 20685142.
2010
Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2.
Human gene therapy.
21(9):1129-36
[DOI] 10.1089/hum.2010.016.
[PMID] 20486772.
2010
High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.
Molecular therapy : the journal of the American Society of Gene Therapy.
18(12):2048-56
[DOI] 10.1038/mt.2010.172.
[PMID] 20736929.
2010
High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy.
Human gene therapy.
21(11):1527-43
[DOI] 10.1089/hum.2010.005.
[PMID] 20507237.
2010
Human hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus serotype 3.
Human gene therapy.
21(12):1741-7
[DOI] 10.1089/hum.2010.075.
[PMID] 20545554.
2010
Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses.
Human gene therapy.
21(3):271-83
[DOI] 10.1089/hum.2009.100.
[PMID] 19788390.
2010
Two decades of clinical gene therapy–success is finally mounting.
Discovery medicine.
9(45):105-11
[PMID] 20193635.
2009
AAV3-mediated transfer and expression of the pyruvate dehydrogenase E1 alpha subunit gene causes metabolic remodeling and apoptosis of human liver cancer cells.
Molecular genetics and metabolism.
98(3):289-99
[DOI] 10.1016/j.ymgme.2009.05.010.
[PMID] 19586787.
2009
Adeno-Associated Virus-Mediated Knockdown of Pyruvate Dehydrogenase E1F(N)Over-Tilde At in in Specific Regions of the Rat Brain
Molecular Genetics and Metabolism.
98
2009
Calpeptin increases the activity of upstream stimulatory factor and induces high level globin gene expression in erythroid cells.
The Journal of biological chemistry.
284(30):20130-5
[DOI] 10.1074/jbc.M109.001461.
[PMID] 19491096.
2009
Comparison of Transduction Efficiency of Tyrosine-Mutant Aav Vectors in Muscle
Molecular Therapy.
17:S175-S176
2009
Critical Requirement of a Cellular Chaperone Protein, Fkbp52, for Intracellular Trafficking and Nuclear Transport of Tyrosine-Mutant Aav2 Vectors for High-Efficiency Transduction of Murine Embryo Fibroblasts
Molecular Therapy.
17
2009
High-Efficiency Transduction of Human Cells in Vitro and Murine Hepatocytes in Vivo At Further Reduced Doses of Aav2 Vectors Containing Multiple-Mutations in Surface-Exposed Tyrosine Residues in the Viral Capsid
Molecular Therapy.
17
2009
High-Efficiency Transduction of the Mouse Retina By Tyrosine-Mutant Aav Serotype Vectors
Molecular Therapy.
17:463-471
[DOI] 10.1038/mt.2008.269.
[PMID] 19066593.
2009
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.
Molecular therapy : the journal of the American Society of Gene Therapy.
17(3):463-71
[DOI] 10.1038/mt.2008.269.
[PMID] 19066593.
2009
Human Hematopoietic Stem Cell Transduction By Aav Vectors: Identification of Aav6 as the Most Efficient Serotype, and Further Augmentation in Transduction Efficiency With Point-Mutations At Tyrosine Residues 705 and 731 in the Viral Capsid
Molecular Therapy.
17
2009
Human Hematopoietic Stem Cell Transduction By Aav Vectors: Identification of Aav6 as the Most Efficient Serotype, and Further Augmentation in Transduction Efficiency With Point-Mutations At Tyrosine Residues 705 and 731 in the Viral Capsid
Molecular Therapy.
17
2009
Novel Aav2 Tyrosine Mutant Capsids Provide Long-Term Therapeutic Factor Ix Expression in a Difficult To Tolerize Murine Hemophilia Model
Molecular Therapy.
17
2009
Novel Tyrosine Mutant Vectors Expand the Utilities of Aav-Mediated Muscle Gene Therapy
Molecular Therapy.
17
2009
Tyrosine-Modified Raav2 Vectors Display Enhanced Transduction of Human Hematopoietic Stem Cells
Molecular Therapy.
17
2008
A combined therapeutic approach for pyruvate dehydrogenase deficiency using self-complementary adeno-associated virus serotype-specific vectors and dichloroacetate.
Molecular genetics and metabolism.
93(4):381-7
[DOI] 10.1016/j.ymgme.2007.10.131.
[PMID] 18206410.
2008
Adeno-associated virus-mediated gene transfer.
Journal of cellular biochemistry.
105(1):17-24
[DOI] 10.1002/jcb.21819.
[PMID] 18500727.
2008
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses.
Proceedings of the National Academy of Sciences of the United States of America.
105(22):7827-32
[DOI] 10.1073/pnas.0802866105.
[PMID] 18511559.
2008
Optimization of recombinant adeno-associated viral vectors for human beta-globin gene transfer and transgene expression.
Human gene therapy.
19(4):365-75
[DOI] 10.1089/hum.2007.173.
[PMID] 18399730.
2008
Pyruvate dehydrogenase complex deficiency caused by ubiquitination and proteasome-mediated degradation of the E1 subunit.
The Journal of biological chemistry.
283(1):237-243
[DOI] 10.1074/jbc.M704748200.
[PMID] 17923481.
2008
Recombinant self-complementary adeno-associated virus serotype vector-mediated hematopoietic stem cell transduction and lineage-restricted, long-term transgene expression in a murine serial bone marrow transplantation model.
Human gene therapy.
19(4):376-83
[DOI] 10.1089/hum.2007.143.
[PMID] 18370591.
2008
Single-polarity recombinant adeno-associated virus 2 vector-mediated transgene expression in vitro and in vivo: mechanism of transduction.
Molecular therapy : the journal of the American Society of Gene Therapy.
16(2):290-5
[PMID] 18087261.
2008
Stable integration of recombinant adeno-associated virus vector genomes after transduction of murine hematopoietic stem cells.
Human gene therapy.
19(3):267-78
[DOI] 10.1089/hum.2007.161.
[PMID] 18303957.
2008
Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo.
Gene therapy.
15(18):1287-93
[DOI] 10.1038/gt.2008.89.
[PMID] 18496572.
2008
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression.
Virology.
381(2):194-202
[DOI] 10.1016/j.virol.2008.08.027.
[PMID] 18834608.
2008
Working group report: the roles of glycans in hemostasis, inflammation and vascular biology.
Glycobiology.
18(10):747-9
[DOI] 10.1093/glycob/cwn065.
[PMID] 18621991.
2007
A dual role of EGFR protein tyrosine kinase signaling in ubiquitination of AAV2 capsids and viral second-strand DNA synthesis.
Molecular therapy : the journal of the American Society of Gene Therapy.
15(7):1323-30
[PMID] 17440440.
2007
Down-regulation of expression of rat pyruvate dehydrogenase E1alpha gene by self-complementary adeno-associated virus-mediated small interfering RNA delivery.
Mitochondrion.
7(4):253-9
[PMID] 17392036.
2007
Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity.
Human gene therapy.
18(2):171-82
[PMID] 17328683.
2006
Adeno-associated virus-mediated gene transfer in hematopoietic stem/ progenitor cells as a therapeutic tool.
Current gene therapy.
6(6):683-98
[PMID] 17168699.
2006
Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5.
Human gene therapy.
17(3):321-33
[PMID] 16544981.
2006
Role of cellular FKBP52 protein in intracellular trafficking of recombinant adeno-associated virus 2 vectors.
Virology.
353(2):283-93
[PMID] 16828834.
2006
Role of integrin cross-regulation in parvovirus B19 targeting.
Human gene therapy.
17(9):909-20
[PMID] 16972759.
2005
Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions.
Human gene therapy.
16(7):792-8
[PMID] 16000061.
Grants
Jan 2023
ACTIVE
Development of optimized AAVrh74 vectors for gene therapy of muscular dystrophies
Role: Principal Investigator
Funding: NATL INST OF HLTH NIAMS
Aug 2021
– Aug 2023
Synthetic AAV Vectors for Gene Therapy of Hemophilia in Children
Role: Principal Investigator
Funding: PFIZER INC
Mar 2021
– Feb 2022
Task Order # 13 GSD1a Research Support
Role: Principal Investigator
Funding: *AAVANTIBIO
Aug 2020
ACTIVE
Development of NextGen, GenX, GenY, and GenZ recombinant AAVrh74 vectors
Role: Principal Investigator
Funding: SAREPTA THERAPEUTICS
Feb 2020
– Jul 2021
Regulation of the pro-fibrotic connective tissue growth factor in alcoholic liver disease: mechanisms and targeting approaches
Role: Co-Investigator
Funding: NATL INST OF HLTH NIAAA
Sep 2019
– Sep 2023
Recombinant Adeno associated virus as a long-acting passive antibody vaccine against foreign and transboundary animal diseases
Role: Co-Investigator
Funding: US DEPT OF HOMELAND SECURITY
Sep 2019
– Aug 2021
STTR – Development of a Novel Therapeutic for Hyperthyroidism
Role: Co-Investigator
Funding: MEDOSOME BIOTEC
via NATL INST OF HLTH NIDDK
Aug 2018
– Nov 2019
Development of Clinical Candidate NextGen Optimized AAV3B serotype vectors for gene therapy of hemophilia B and hemophilia A
Role: Principal Investigator
Funding: *AAVECTIVE
Jul 2017
– Jun 2020
AAV2 and hepatocellular carcinoma
Role: Principal Investigator
Funding: NATL INST OF HLTH NIAID
Jan 2017
– Dec 2022
Mechanism of high-efficiency transduction of hepatocytes by optimized AAV vectors
Role: Principal Investigator
Funding: NATL INST OF HLTH NIGMS
Aug 2016
– Jul 2018
Development of Optimized AAV Serotype Vectors for Human Gene Therapy
Role: Principal Investigator
Funding: MEDOSOME BIOTEC
via NATL INST OF HLTH NIAID
May 2015
– Apr 2020
NEXT GENERATION OF RECOMBINANT AAV SEROTYPE VECTORS FOR GENE THERAPY
Role: Principal Investigator
Funding: NATL INST OF HLTH NHLBI
Jan 2015
ACTIVE
UF Health Cancer Center Bridge Seed Grant funded through the Florida Consortium of National Cancer Institute Centers Program
Role: Project Manager
Funding: UF HEALTH SHANDS HOSPITAL
Jul 2014
– Jun 2017
Cystic Fibrosis Research
Role: Principal Investigator
Funding: UF FOUNDATION
Jul 2005
ACTIVE
Kitzman Genetic Research
Role: Principal Investigator
Funding: UF FOUNDATION
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
Addresses:
- Business Mailing:
-
ROOM 492-A
CANCER AND GENETICS RESEARCH COMPLEX
2033 MOWRY ROAD
GAINESVILLE FL 326110001 - Business Street:
-
2033 MOWRY ROAD, ROOM 492-A
2033 MOWRY RD RM 492-A
CANCER AND GENETICS RESEARCH COMPLEX
GAINESVILLE FL 326112079