Aravind Asokan

Surgery, Surgical Sciences

Professor in Surgery

Aravind Asokan Profile Photo

Research Interests

Virology
RNA Biology
Viral Engineering
RNA Engineering
Gene Therapy
Gene Editing
Gene Regulation
Regenerative Medicine

Bio

Synthetic Virology & Gene Therapy

Education

Ph.D. University of North Carolina, Chapel Hill, 2004

Positions

Professor in Surgery
Director of Gene Therapy
Professor of Biomedical Engineering
Professor of Molecular Genetics and Microbiology
Affiliate of the Duke Regeneration Center

Courses Taught

MGM 593: Research Independent Study
BME 792: Continuation of Graduate Independent Study
BME 791: Graduate Independent Study
BME 494: Projects in Biomedical Engineering (GE)
BME 493: Projects in Biomedical Engineering (GE)

Publications

Gibson RA, Jeck WR, Koch RL, Mehta A, Choi SJ, Sriraman Y, et al. Progressive liver disease and dysregulated glycogen metabolism in murine GSD IX γ2 models human disease. Mol Genet Metab. 2024 Oct 28;143(4):108597.
Meganck RM, Ogurlu R, Liu J, Moller-Tank S, Tse V, Blondel LO, et al. Sub-genomic flaviviral RNA elements increase the stability and abundance of recombinant AAV vector transcripts. J Virol. 2024 Aug 20;98(8):e0009524.
Kurt E, Devlin G, Asokan A, Segura T. Gene Delivery From Granular Scaffolds for Tunable Biologics Manufacturing. Small. 2024 Aug;20(31):e2309911.
Yadav A, Liang R, Press K, Schmidt A, Shabani Z, Leng K, et al. Evaluation of AAV Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy. Transl Stroke Res. 2024 Jul 8;
Smith TJ, Elmore ZC, Fusco RM, Hull JA, Rosales A, Martinez M, et al. Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transfer. Mol Ther. 2024 Jul 3;32(7):2080–93.
Karpurapu A, Williams HA, DeBenedittis P, Baker CE, Ren S, Thomas MC, et al. Deep Learning Resolves Myovascular Dynamics in the Failing Human Heart. JACC Basic Transl Sci. 2024 May;9(5):674–86.
Gao Q, Kahan R, Gonzalez TJ, Zhang M, Alderete IS, DeLaura I, et al. Gene delivery followed by ex vivo lung perfusion using an adeno-associated viral vector in a rodent lung transplant model. J Thorac Cardiovasc Surg. 2024 May;167(5):e131–9.
Loeb EJ, Havlik PL, Elmore ZC, Rosales A, Fergione SM, Gonzalez TJ, et al. Capsid-mediated control of adeno-associated viral transcription determines host range. Cell Rep. 2024 Mar 26;43(3):113902.
Zhu X, Huang Q, Jiang L, Nguyen V-T, Vu T, Devlin G, et al. Longitudinal intravital imaging of mouse placenta. Sci Adv. 2024 Mar 22;10(12):eadk1278.
Fiflis DN, Rey NA, Venugopal-Lavanya H, Sewell B, Mitchell-Dick A, Clements KN, et al. Repurposing CRISPR-Cas13 systems for robust mRNA trans-splicing. Nat Commun. 2024 Mar 14;15(1):2325.
Barzi M, Chen T, Gonzalez TJ, Pankowicz FP, Oh SH, Streff HL, et al. A humanized mouse model for adeno-associated viral gene therapy. Nat Commun. 2024 Mar 4;15(1):1955.
Clements KN, Gonzalez TJ, Asokan A. Engineering Synthetic circRNAs for Efficient CNS Expression. Methods Mol Biol. 2024;2765:227–46.
Asokan A, Shen S. Redirecting AAV vectors to extrahepatic tissues. Mol Ther. 2023 Dec 6;31(12):3371–5.
Asokan A. AAV vector immunotoxicity: Stopping the domino effect. Mol Ther. 2023 Dec 6;31(12):3357–8.
Gonzalez TJ, Mitchell-Dick A, Blondel LO, Fanous MM, Hull JA, Oh DK, et al. Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery. Nat Protoc. 2023 Nov;18(11):3413–59.
Konkimalla A, Elmore Z, Konishi S, Macadlo L, Katsura H, Tata A, et al. Efficient Adeno-associated Virus-mediated Transgenesis in Alveolar Stem Cells and Associated Niches. Am J Respir Cell Mol Biol. 2023 Sep;69(3):255–65.
Cigliola V, Shoffner A, Lee N, Ou J, Gonzalez TJ, Hoque J, et al. Spinal cord repair is modulated by the neurogenic factor Hb-egf under direction of a regeneration-associated enhancer. Nat Commun. 2023 Aug 11;14(1):4857.
Grigsby D, Klingeborn M, Kelly U, Chew LA, Asokan A, Devlin G, et al. AAV Gene Augmentation of Truncated Complement Factor H Differentially Rescues Ocular Complement Dysregulation in a Mouse Model. Invest Ophthalmol Vis Sci. 2023 Jul 3;64(10):25.
Kesseli SJ, Krischak MK, Gao Q, Gonzalez T, Zhang M, Halpern SE, et al. Adeno-associated virus mediates gene transduction after static cold storage treatment in rodent lung transplantation. In: J Thorac Cardiovasc Surg. 2023. p. e38–49.
Smith TJ, Fusco RM, Elmore ZC, Asokan A. Interplay between Furin and Sialoglycans in Modulating Adeno-Associated Viral Cell Entry. J Virol. 2023 May 31;97(5):e0009323.
Barzi M, Johnson CG, Chen T, Rodriguiz RM, Hemmingsen M, Gonzalez TJ, et al. Rescue of glutaric aciduria type I in mice by liver-directed therapies. Sci Transl Med. 2023 Apr 19;15(692):eadf4086.
Barzi M, Johnson C, Chen T, Rodriguiz R, Hemmingsen M, Gonzalez TJ, et al. RESCUE OF GLUTARIC ACIDURIA TYPE I MICE BY LIVER DIRECTED THERAPIES. In: Molecular Genetics and Metabolism. Elsevier BV; 2023. p. 107377–107377.
Nyberg WA, Ark J, To A, Clouden S, Reeder G, Muldoon JJ, et al. An evolved AAV variant enables efficient genetic engineering of murine T cells. Cell. 2023 Jan 19;186(2):446-460.e19.
Yan R, Cigliola V, Oonk KA, Petrover Z, DeLuca S, Wolfson DW, et al. An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair. Cell Stem Cell. 2023 Jan 5;30(1):96-111.e6.
Smith T, Hull JA, Fusco RM, Blondel L, Elmore Z, Asokan A. Newly Engineered IgM and IgG Cleaving Enzymes for AAV Gene Therapy. In: MOLECULAR THERAPY. 2023. p. 113–113.
Tarantal AF, Martinez M, Sanz L, Lee C, O’Geen H, Asokan A, et al. AAV Serotype Tropism and Editing in Young Rhesus Monkeys. In: MOLECULAR THERAPY. 2023. p. 42–42.
Gao Q, Kesseli SJ, Gonzalez T, Zhang M, Kahan R, Krischak M, et al. AAV9-mediated gene delivery to liver grafts during static cold storage in a rat liver transplant model. In: Frontiers in transplantation. 2023.
Peek JL, Rosales A, Welch R, Beckermann T, Woodard LE, Gonzalez TJ, et al. Hybrid Dual scAAV Delivery of Transgenes to the Kidney. In: MOLECULAR THERAPY. 2023. p. 788–788.
Fusco RM, Walton E, Asokan A. A Platform for Interrogating Transgene Silencing and Epigenetic Signatures of AAV Genomes. In: MOLECULAR THERAPY. 2023. p. 239–239.
Liang R, Press K, Gonzalez T, Asokan A, Su H. Evaluating AAV vectors for HHT gene therapy. In: ANGIOGENESIS. 2023. p. S4–S4.
Nyberg WA, Ark J, To A, Clouden S, Reeder G, Muldoon JJ, et al. An Evolved AAV Variant Enables Efficient Genetic Engineering of Murine T Cells and the Modeling of Trac-CAR-T Cells in Immunocompetent Tumor Models. In: MOLECULAR THERAPY. 2023. p. 538–538.
Ogurlu R, Meganck RM, Blondel LO, Rosales A, Asokan A. Engineering mRNA Stability with Flaviviral Genomic Elements to Improve AAV Transduction. In: MOLECULAR THERAPY. 2023. p. 96–96.
Rosales A, Blondel LO, Gao Q, Barbas AS, Asokan A. Evolving Nephrotropic AAV Variants Using Ex Vivo NHP Kidney Perfusion and Human Kidney Organoids. In: MOLECULAR THERAPY. 2023. p. 180–180.
Barzi M, Johnson CG, Chen T, Rodriguiz RM, Hemmingsen M, Gonzalez TJ, et al. Rescue of Glutaric Aciduria Type I Mice by Liver Directed Therapies. In: MOLECULAR THERAPY. 2023. p. 104–104.
Kahan R, Gao Q, Zhang M, Abraham N, Gonzalez T, Song M, et al. AAV9 PD-L1 Mediated Immunodulation of Donor Graft in Rat Lung Allotransplantation. In: JOURNAL OF HEART AND LUNG TRANSPLANTATION. 2023. p. S178–S178.
Mitchell-Dick AM, Adoff M, Kang EJ, Clouden S, Southwell DG, Asokan A. Structure Function Correlates of New AAV Variants Evolved in Human Brain Explants. In: MOLECULAR THERAPY. 2023. p. 460–1.
Wolfson D, Rosales A, Ogurlu R, Dvergsten T, Asokan A, Poss K. Tissue Regeneration Enhancer Elements Enable Injury Responsive AAV Gene Expression. In: MOLECULAR THERAPY. 2023. p. 136–136.
Sheahan B, Kislovskiy O, Thiriveedi V, Khumukcham S, Rosales A, Gonzalez T, et al. TREATMENT OF CYSTIC FIBROSIS WITH IN VIVO BASE EDITING OF THE INTESTINAL EPITHELIUM. In: GASTROENTEROLOGY. 2023. p. S150–S150.
Fiflis DN, Rey N, Milo S, Asokan A. CRISPR Assisted Trans-Splicing of RNA Fragments. In: MOLECULAR THERAPY. 2023. p. 182–182.
Mitchell-Dick AM, Adoff M, Kang EJ, Southwell DG, Asokan A. Barcoded Mapping of Promoter Specificity and Efficiency in Human Brain Explants and the Mouse Brain. In: MOLECULAR THERAPY. 2023. p. 670–670.
Loeb E, Havlik LP, Asokan A. Capsid Mediated Control of Adeno-Associated Viral Genome Transcription. In: MOLECULAR THERAPY. 2023. p. 137–137.
Konkimalla A, Konishi S, Kobayashi Y, Kadur Lakshminarasimha Murthy P, Macadlo L, Mukherjee A, et al. Multi-apical polarity of alveolar stem cells and their dynamics during lung development and regeneration. iScience. 2022 Oct 21;25(10):105114.
Gonzalez TJ, Simon KE, Blondel LO, Fanous MM, Roger AL, Maysonet MS, et al. Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. Nat Commun. 2022 Oct 10;13(1):5947.
Das A, Vijayan M, Walton EM, Stafford VG, Fiflis DN, Asokan A. Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex. J Virol. 2022 Feb 23;96(4):e0203921.
Emmanuel SN, Smith JK, Hsi J, Tseng Y-S, Kaplan M, Mietzsch M, et al. Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants. J Virol. 2022 Feb 9;96(3):e0125121.
Gao Q, DeLaura IF, Anwar IJ, Kesseli SJ, Kahan R, Abraham N, et al. Gene Therapy: Will the Promise of Optimizing Lung Allografts Become Reality? Front Immunol. 2022;13:931524.
Ark JI, Nyberg W, Simon K, Rosales A, Clouden S, Eyquem J, et al. Targeted In Vivo Manipulation of Murine T-Cells Using a Newly Evolved AAV Capsid Mutant. In: MOLECULAR THERAPY. 2022. p. 425–6.
Ark JI, Nyberg W, Clouden S, Havlik PL, Eyquem J, Asokan A. Evolution of a New AAV Variant with Murine T Lymphocyte Tropism Using the MHC-Ib Molecule H2-Q7 as a Receptor. In: MOLECULAR THERAPY. 2022. p. 389–389.
Austin B, Gulledge T, Bandoski-Gralinski C, Asokan A, Rivera RC. Pre-Existing Humoral Immunity to AAV Capsids: Species and Age-Related Differences in Anti-Capsid ELISA and Neutralizing Antibody Assays. In: MOLECULAR THERAPY. 2022. p. 340–340.
Rosales A, Gonzalez T, Asokan A. A Newly Evolved AAV Variant Enables Potent Gene Transfer in Kidneys of Multiple Species. In: MOLECULAR THERAPY. 2022. p. 555–555.
Elmore Z, Oh D, Asokan A. AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress. In: MOLECULAR THERAPY. 2022. p. 71–71.
Vekstein AM, Wendell DC, DeLuca S, Yan R, Chen Y, Bishawi M, et al. Targeted Delivery for Cardiac Regeneration: Comparison of Intra-coronary Infusion and Intra-myocardial Injection in Porcine Hearts. Front Cardiovasc Med. 2022;9:833335.
Kesseli SJ, Krischak MK, Gao Q, Halpern SE, Zhang M, Song M, et al. Intra-Tracheal Adeno-Associated Virus Mediates Gene Transduction During Static Cold Storage in Rodent Lung Transplantation. In: JOURNAL OF HEART AND LUNG TRANSPLANTATION. 2022. p. S38–9.
Loeb EJ, Havlik LP, Asokan A. Unlocking Avian AAV Transduction in Mammalian Cells and Tissues for Immune Evasion and Redosing. In: MOLECULAR THERAPY. 2022. p. 204–204.
Gulledge T, Austin B, Bandoski-Gralinski C, Asokan A, Rivera RC. STRV5, a Liver Detargeted AAV Capsid Demonstrates Improved Safety Profile After Intravenous Administration of High Doses in Non-Human Primates. In: MOLECULAR THERAPY. 2022. p. 339–339.
Gibson RA, Lim J-A, Choi SJ, Bali D, Young S, Koch R, et al. SEVERE PROGRESSION OF LIVER DISEASE IN AN AGING PHKG2^-/- MOUSE MODEL RECAPITULATES GSD IX G2 PATIENT PHENOTYPE. In: MOLECULAR GENETICS AND METABOLISM. 2022. p. 272–272.
Barzi M, Johnson C, Chen T, Hemmingsen M, El-Gharbawy A, Gonzalez TJ, et al. Novel Gene Supplementation, Genome Editing and Cellular Therapeutic Approaches to Treat Glutaric Aciduria Type I. In: MOLECULAR THERAPY. 2022. p. 458–458.
Hull JA, Mietzsch M, Tan YZ, Aiyer S, Bennett A, Lakshman R, et al. AAV Capsid Dynamics at the 5-Fold Pore Controls Genome Release. In: MOLECULAR THERAPY. 2022. p. 205–6.
Kesseli S, Krischak M, Gao Q, Halpern S, Zhang M, Abraham N, et al. Adeno-associated virus 9 mediates gene transduction during static cold storage in rodent liver transplantation. In: AMERICAN JOURNAL OF TRANSPLANTATION. 2022. p. 67–67.
Hemmingsen MG, Barzi M, Chen T, Collias S, Beasley J, Zhang G, et al. Validation of therapeutic strategies in a novel compound heterozygote model of Methylmalonic Acidemia. In: HUMAN GENE THERAPY. 2022. p. A174–A174.
Chen T, Barzi M, Pankowicz F, Gonzalez TJ, Bissig-Choisat B, Asokan A, et al. A Novel Human Liver Chimeric Mouse Model Lacking the Murine AAVR Gene for Validation of AAV Gene Therapy Vectors. In: MOLECULAR THERAPY. 2022. p. 43–43.
Jeon K, Gold S, Hu M, Smith JK, O’Banion C, Morra M, et al. A Human iPSC-Derived Neuron and Cardiomyocyte Platform for Assessing Novel Recombinant Adeno-Associated Viral (AAV) Vectors. In: MOLECULAR THERAPY. 2022. p. 482–482.
Walton EM, Black JB, Castillo L, Gersbach CA, Asokan A. Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome. In: MOLECULAR THERAPY. 2022. p. 430–430.
Gibson RA, Lim J-A, Choi SJ, Koch RL, Bali D, Young S, et al. Liver Directed AAV Gene Therapy Reverses Progression of Glycogen Storage Disease Type IX γ2 in Mice. In: MOLECULAR THERAPY. 2022. p. 478–9.
Gonzalez TJ, Blondel L, Rosales A, Daniels H, Gersbach CA, Asokan A. Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant. In: MOLECULAR THERAPY. 2022. p. 211–2.
Smith TJ, Elmore ZC, Asokan A. Furin is a Host Factor Restricting Adeno-Associated Virus 4 Transduction. In: MOLECULAR THERAPY. 2022. p. 206–206.
Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, et al. Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. Mol Ther. 2021 Nov 3;29(11):3243–57.
Elmore ZC, Patrick Havlik L, Oh DK, Anderson L, Daaboul G, Devlin GW, et al. The membrane associated accessory protein is an adeno-associated viral egress factor. Nat Commun. 2021 Oct 29;12(1):6239.
Havlik LP, Das A, Mietzsch M, Oh DK, Ark J, McKenna R, et al. Receptor Switching in Newly Evolved Adeno-associated Viruses. J Virol. 2021 Sep 9;95(19):e0058721.
Gemberling MP, Siklenka K, Rodriguez E, Tonn-Eisinger KR, Barrera A, Liu F, et al. Transgenic mice for in vivo epigenome editing with CRISPR-based systems. Nat Methods. 2021 Aug;18(8):965–74.
Gibson RA, Lim J-A, Choi SJ, Flores L, Clinton L, Bali D, et al. Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2-/- mouse model. Mol Genet Metab. 2021 Jul;133(3):269–76.
Mitchell-Dick A, Asokan A. AAV-CNS matters turn from gray to white. Mol Ther. 2021 May 5;29(5):1659–60.
A Beautiful Mind and the Heart of an Explorer. Human Gene Therapy. 2021 Apr 1;32(7–8):321–6.
Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, et al. The NIH Somatic Cell Genome Editing program. Nature. 2021 Apr;592(7853):195–204.
Hunanyan AS, Kantor B, Puranam RS, Elliott C, McCall A, Dhindsa J, et al. Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood. Hum Gene Ther. 2021 Apr;32(7–8):405–19.
Meganck RM, Liu J, Hale AE, Simon KE, Fanous MM, Vincent HA, et al. Engineering highly efficient backsplicing and translation of synthetic circRNAs. Mol Ther Nucleic Acids. 2021 Mar 5;23:821–34.
Walton EM, Black JB, Gersbach CA, Asokan A. A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes. In: MOLECULAR THERAPY. 2021. p. 150–1.
Das A, Havlik P, Ark J, Mietzsch M, Agbandje-McKenna M, Asokan A. A CRISPR/Cas9 Genome Wide Screen and Cryo-EM Structure Reveal Receptor Switching by a Synthetic AAV Variant. In: MOLECULAR THERAPY. 2021. p. 151–2.
Havlik LP, Oh DK, Das A, Rivera RMC, Asokan A. Mechanistic Elucidation of Adenovirus Mediated Enhancement of Recombinant Adeno-Associated Virus (rAAV) towards Efficient Low Dose Gene Therapy. In: MOLECULAR THERAPY. 2021. p. 153–4.
Havlik LP, Oh DK, Das A, Rivera RMC, Asokan A. Distinct Functional Roles for AAV Capsid Surface Epitopes Revealed through Structure-Guided Evolution. In: MOLECULAR THERAPY. 2021. p. 153–153.
Gibson R, Lim J-A, Choi SJ, Flores L, Clinton L, Bali D, et al. Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model. In: MOLECULAR THERAPY. 2021. p. 239–239.
Vekstein AM, Wendell DC, Bowles DE, DeLuca S, Yan R, Bishawi M, et al. Targeted Intra-Coronary Delivery versus Intra-Myocardial Injection of Therapeutics for Myocardial Recovery: A Nanoparticle Image Guided Porcine Study. In: CIRCULATION. 2021.
Vijayan M, Das A, Stafford G, Elmore Z, Walton E, Asokan A. The Human Silencing Hub (HUSH Complex) Is a Potent Regulator of AAV Transgene Silencing. In: MOLECULAR THERAPY. 2021. p. 47–47.
Meganck RM, Moller-Tank S, Liu J, Tse LV, Vincent HA, Marzluff WF, et al. Subgenomic Flaviviral RNA Elements Enhance rAAV Vector mRNA Stability and Transduction. In: MOLECULAR THERAPY. 2021. p. 151–151.
Ark J, Nyberg W, Havlik P, To A, Eyquem J, Asokan A. Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations. In: MOLECULAR THERAPY. 2021. p. 32–32.
Cannon P, Asokan A, Czechowicz A, Hammond P, Kohn DB, Lieber A, et al. Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development. Hum Gene Ther. 2021 Jan;32(1–2):31–42.
Kwon JB, Ettyreddy AR, Vankara A, Bohning JD, Devlin G, Hauschka SD, et al. In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy. Mol Ther Methods Clin Dev. 2020 Dec 11;19:320–9.
Kang MH, Hu J, Pratt RE, Hodgkinson CP, Asokan A, Dzau VJ. Optimizing delivery for efficient cardiac reprogramming. Biochem Biophys Res Commun. 2020 Nov 26;533(1):9–16.
Madigan VJ, Berry GE, Tyson TO, Nardone-White D, Ark J, Elmore ZC, et al. The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. J Virol. 2020 Oct 14;94(21).
Elmore ZC, Oh DK, Simon KE, Fanous MM, Asokan A. Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme. JCI Insight. 2020 Sep 17;5(19).
Havlik LP, Simon KE, Smith JK, Klinc KA, Tse LV, Oh DK, et al. Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach. J Virol. 2020 Sep 15;94(19).
Das A, Barrientos R, Shiota T, Madigan V, Misumi I, McKnight KL, et al. Gangliosides are essential endosomal receptors for quasi-enveloped and naked hepatitis A virus. Nat Microbiol. 2020 Sep;5(9):1069–78.
Li J, Li Y, Wang J, Gonzalez TJ, Asokan A, Napierala JS, et al. Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy. Hum Gene Ther. 2020 Aug;31(15–16):839–51.
Huang Q, Cohen MA, Alsina FC, Devlin G, Garrett A, McKey J, et al. Intravital imaging of mouse embryos. Science. 2020 Apr 10;368(6487):181–6.
Kwon J, Vankara A, Bohning J, Delvin G, Asokan A, Gersbach C. Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. 2020. p. 330–330.
Elmore ZC, Havlik LP, Oh D, Asokan A. The Membrane-Associated Accessory Protein (MAAP) is Essential for Rapid Extracellular Secretion of Adeno-Associated Viruses. In: MOLECULAR THERAPY. 2020. p. 44–44.
Havlik LP, Elmore ZC, Oh DK, Smith TJ, Asokan A. Evolving New AAV Strains that Demonstrate AAVR Independence. In: MOLECULAR THERAPY. 2020. p. 245–6.
Meganck RM, Gonzalez TJ, Vincent HA, Asokan A. Engineering Backsplicing Introns for Robust Expression of Synthetic Circular RNAs from AAV Vectors. In: MOLECULAR THERAPY. 2020. p. 21–21.
Gonzalez TJ, Havlik LP, Fanous M, Simon K, Edwards L, Kwon J, et al. Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation. In: MOLECULAR THERAPY. 2020. p. 12–3.
McCall AL, de Leon A, Fusco AF, Bailey A, Fanous M, Asokan A, et al. A Novel Capsid for Smooth Muscle Pathology in Pompe Disease. In: MOLECULAR THERAPY. 2020. p. 492–492.
Elmore Z, Oh D, Simon K, Fanous M, Asokan A. A Novel Approach for Rapid and Transient Depletion of Pre-Existing Neutralizing Antibodies Against AAV Vectors. In: MOLECULAR THERAPY. 2020. p. 398–398.
Madigan VJ, Tyson TO, Yuziuk JA, Pillai M, Moller-Tank S, Asokan A. A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes. J Virol. 2019 Nov 1;93(21).
Asokan A. CRISPR genome editing in stem cells turns to gold. Nat Mater. 2019 Oct;18(10):1038–9.
Abdulhaqq SA, Wu H, Schell JB, Hammond KB, Reed JS, Legasse AW, et al. Vaccine-Mediated Inhibition of the Transporter Associated with Antigen Processing Is Insufficient To Induce Major Histocompatibility Complex E-Restricted CD8+ T Cells in Nonhuman Primates. J Virol. 2019 Oct 1;93(19).
Madigan VJ, Yuziuk JA, Chiarella AM, Tyson TO, Meganck RM, Elmore ZC, et al. Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription. PLoS Pathog. 2019 Aug;15(8):e1007988.
Albright BH, Simon KE, Pillai M, Devlin GW, Asokan A. Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses. J Virol. 2019 Jun 1;93(11).
Madigan V, Tyson TO, Asokan A. Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 425–425.
Madigan V, Tyson TO, Yuziuk JA, Pillai M, Moller-Tank S, Asokan A. A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 424–5.
Castle MJ, Cheng Y, Asokan A, Tuszynski MH. Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 97–8.
Bulaklak K, Robinson-Hamm J, Gough V, Nelson CE, Madigan V, Asokan A, et al. AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 377–377.
Gonzalez TJ, Meganck R, Fanous M, Simon K, Asokan A. Design Principles for AAV Mediated Circular RNA Expression in the Brain. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 60–60.
Nelson C, Wu Y, Gemberling M, Oliver M, Bohning JD, Robinson-Hamm JN, et al. Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 46–7.
Havlik LP, Tse LV, Smith JK, Klinc KA, Oh D, Simon K, et al. Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 53–53.
Nelson CE, Wu Y, Gemberling MP, Oliver ML, Waller MA, Bohning JD, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med. 2019 Mar;25(3):427–32.
Mestre H, Hablitz LM, Xavier AL, Feng W, Zou W, Pu T, et al. Aquaporin-4-dependent glymphatic solute transport in the rodent brain. Elife. 2018 Dec 18;7.
Meganck RM, Borchardt EK, Castellanos Rivera RM, Scalabrino ML, Wilusz JE, Marzluff WF, et al. Tissue-Dependent Expression and Translation of Circular RNAs with Recombinant AAV Vectors In Vivo. Mol Ther Nucleic Acids. 2018 Dec 7;13:89–98.
Castle MJ, Cheng Y, Asokan A, Tuszynski MH. Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion. Sci Adv. 2018 Nov;4(11):eaau9859.
Burwitz B, Wettengel J, Muck-Hausl M, Hammond K, Ringelhan M, Ko C, et al. A new HIV/HBV co-infection model: hepatocytic expression of human sodium taurocholate cotransporting polypeptide (NTCP) Enables Hepatitis B Virus Infection of Macaques. In: JOURNAL OF MEDICAL PRIMATOLOGY. WILEY; 2018. p. 324–324.
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Tse LV, Klinc KA, Madigan VJ, Castellanos Rivera RM, Wells LF, Havlik LP, et al. Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion. Proc Natl Acad Sci U S A. 2017 Jun 13;114(24):E4812–21.
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Tarantal AF, Lee CCI, Martinez ML, Asokan A, Samulski RJ. Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector. Hum Gene Ther. 2017 May;28(5):385–91.
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Zeng C, Moller-Tank S, Asokan A, Dragnea B. Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2. J Phys Chem B. 2017 Mar 2;121(8):1843–53.
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Murlidharan G, Asokan A. Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2016. p. S24–S24.
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Berry G, Murlidharan G, Asokan A. 30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS. In: Molecular Therapy. Elsevier BV; 2016. p. S14–S14.
Schreiber CA, Izumiya Y, Asokan A, Ikeda Y. 8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction. In: Molecular Therapy. Elsevier BV; 2016. p. S4–5.
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Berry GE, Asokan A. Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction. J Biol Chem. 2016 Jan 8;291(2):939–47.
Murlidharan G, Samulski RJ, Asokan A. Gene therapy of CNS disorders using recombinant AAV vectors. In: Translational Neuroscience: Fundamental Approaches for Neurological Disorders. 2016. p. 9–24.
Borchardt EK, Vandoros LA, Huang M, Lackey PE, Marzluff WF, Asokan A. Controlling mRNA stability and translation with the CRISPR endoribonuclease Csy4. RNA. 2015 Nov;21(11):1921–30.
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Woodard K, Liang KJ, Lentz T, Bennett W, Asokan A, Samulski R. Transduction of Mouse Retina by Intravitreal Delivery Using Chimeric Capsids with Altered Glycan Interactions. In: INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE. ASSOC RESEARCH VISION OPHTHALMOLOGY INC; 2015.
Tse LV, Moller-Tank S, Asokan A. Strategies to circumvent humoral immunity to adeno-associated viral vectors. Expert Opin Biol Ther. 2015 Jun;15(6):845–55.
Hemphill J, Borchardt EK, Brown K, Asokan A, Deiters A. Optical Control of CRISPR/Cas9 Gene Editing. J Am Chem Soc. 2015 May 6;137(17):5642–5.
Murlidharan G, Asokan A. 265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain. In: Molecular Therapy. Elsevier BV; 2015. p. S106–S106.
Albright BH, Madigan V, Rao L, Chiorini JA, Agbandje-McKenna M, Asokan A. 302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors. In: Molecular Therapy. Elsevier BV; 2015. p. S122–S122.
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Berry GE, Asokan A. 38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction. In: Molecular Therapy. Elsevier BV; 2015. p. S17–S17.
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Rivera RMC, Rao L, Murlidharan G, Corriher T, Asokan A. Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2015. p. S77–S77.
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Murlidharan G, Corriher T, Ghashghaei HT, Asokan A. Unique glycan signatures regulate adeno-associated virus tropism in the developing brain. J Virol. 2015 Apr;89(7):3976–87.
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Pleticha J, Heilmann LF, Evans CH, Asokan A, Samulski RJ, Beutler AS. Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs. Mol Pain. 2014 Sep 2;10:54.
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Huang L-Y, Ng R, Miller EB, McKenna R, Asokan A, Agbandje-McKenna M. Characterizing Essential Receptor Binding Residues for AAV1. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2014. p. S2–S2.
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Murlidharan G, Samulski RJ, Asokan A. Biology of adeno-associated viral vectors in the central nervous system. Front Mol Neurosci. 2014;7:76.
Shen S, Troupes AN, Pulicherla N, Asokan A. Multiple roles for sialylated glycans in determining the cardiopulmonary tropism of adeno-associated virus 4. J Virol. 2013 Dec;87(24):13206–13.
Asokan A, Samulski RJ. An emerging adeno-associated viral vector pipeline for cardiac gene therapy. Hum Gene Ther. 2013 Nov;24(11):906–13.
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Shen S, Asokan A. Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2013. p. S30–S30.
Murlidharan G, Sommerville L, Ghashghaei T, Asokan A. Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2013. p. S1–S1.
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Li C, Diprimio N, Bowles DE, Hirsch ML, Monahan PE, Asokan A, et al. Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles. J Virol. 2012 Aug;86(15):7752–9.
Horowitz ED, Finn MG, Asokan A. Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection. ACS Chem Biol. 2012 Jun 15;7(6):1059–66.
Denard J, Beley C, Kotin R, Lai-Kuen R, Blot S, Leh H, et al. Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. J Virol. 2012 Jun;86(12):6620–31.
Horowitz ED, Asokan A. Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2012. p. S202–S202.
Shen S, Sun J, Hirsch M, Chavala S, Monahan P, Asokan A. Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2012. p. S203–S203.
Asokan A, Schaffer DV, Samulski RJ. The AAV vector toolkit: poised at the clinical crossroads. Mol Ther. 2012 Apr;20(4):699–708.
Li C, Narkbunnam N, Samulski RJ, Asokan A, Hu G, Jacobson LJ, et al. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther. 2012 Mar;19(3):288–94.
Pulicherla N, Kota P, Dokholyan NV, Asokan A. Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsids. PLoS One. 2012;7(2):e32163.
Gray SJ, Choi VW, Asokan A, Haberman RA, McCown TJ, Samulski RJ. Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration. Curr Protoc Neurosci. 2011 Oct;Chapter 4:Unit-4.17.
Pulicherla N, Asokan A. Peptide affinity reagents for AAV capsid recognition and purification. Gene Ther. 2011 Oct;18(10):1020–4.
Monahan PE, Li C, Narkbunnam N, Samulski RJ, Hu G, Asokan A, et al. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. In: JOURNAL OF THROMBOSIS AND HAEMOSTASIS. WILEY-BLACKWELL; 2011. p. 231–2.
Pulicherla N, Shen S, Yadav S, Debbink K, Govindasamy L, Agbandje-McKenna M, et al. Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther. 2011 Jun;19(6):1070–8.
Horowitz ED, Weinberg MS, Asokan A. Glycated AAV vectors: chemical redirection of viral tissue tropism. Bioconjug Chem. 2011 Apr 20;22(4):529–32.
Shen S, Bryant KD, Brown SM, Randell SH, Asokan A. Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. J Biol Chem. 2011 Apr 15;286(15):13532–40.
Kapoor A, Hornig M, Asokan A, Williams B, Henriquez JA, Lipkin WI. Bocavirus episome in infected human tissue contains non-identical termini. PLoS One. 2011;6(6):e21362.
Phillips JL, Hegge J, Wolff JA, Samulski RJ, Asokan A. Systemic gene transfer to skeletal muscle using reengineered AAV vectors. Methods Mol Biol. 2011;709:141–51.
Asokan A. Reengineered AAV vectors: old dog, new tricks. Discov Med. 2010 May;9(48):399–403.
Asokan A, Conway JC, Phillips JL, Li C, Hegge J, Sinnott R, et al. Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol. 2010 Jan;28(1):79–82.
Li C, Hirsch M, DiPrimio N, Asokan A, Goudy K, Tisch R, et al. Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J Virol. 2009 Jul;83(13):6817–24.
Li C, Goudy K, Hirsch M, Asokan A, Fan Y, Alexander J, et al. Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A. 2009 Jun 30;106(26):10770–4.
Xiao P-J, Johnson JS, Neumann A, Zeng X, Asokan A, Samulski RJ. Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2009. p. S38–S38.
Li C, Hirsch M, Carter P, Asokan A, Zhou X, Wu Z, et al. A small regulatory element from chromosome 19 enhances liver-specific gene expression. Gene Ther. 2009 Jan;16(1):43–51.
Asokan A, Johnson JS, Li C, Samulski RJ. Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals. Gene Ther. 2008 Dec;15(24):1618–22.
Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS, et al. Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles. Mol Ther. 2008 Jul;16(7):1252–60.
DiPrimio N, Asokan A, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Surface loop dynamics in adeno-associated virus capsid assembly. J Virol. 2008 Jun;82(11):5178–89.
Li C, Hirsch M, Asokan A, Zeithaml B, Ma H, Kafri T, et al. Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol. 2007 Jul;81(14):7540–7.
Choi VW, Asokan A, Haberman RA, Samulski RJ. Production of recombinant adeno-associated viral vectors. Curr Protoc Hum Genet. 2007 Apr;Chapter 12:Unit-12.9.
Choi VW, Asokan A, Haberman RA, Samulski RJ. Production of recombinant adeno-associated viral vectors for in vitro and in vivo use. Curr Protoc Mol Biol. 2007 Apr;Chapter 16:Unit-16.25.
Wu Z, Asokan A, Grieger JC, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol. 2006 Nov;80(22):11393–7.
Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther. 2006 Sep;14(3):316–27.
Asokan A, Hamra JB, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry. J Virol. 2006 Sep;80(18):8961–9.
Asokan A, Cheung R, Cho MJ. Strategies for the Cytosolic Delivery of Macromolecules: An Overview. In: Handbook of Pharmaceutical Biotechnology. 2006. p. 279–96.
Choi VW, Asokan A, Haberman RA, McCown TJ, Samulski RJ. Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administration. Curr Protoc Neurosci. 2006 May;Chapter 4:Unit-4.17.
Asokan A, Samulski RJ. AAV does the shuffle. Nat Biotechnol. 2006 Feb;24(2):158–60.
Asokan A, Johnson JS, Samulski RJ. 739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue. In: Molecular Therapy. Elsevier BV; 2006. p. S286–S286.
DiPrimio N, Asokan A, Samulski RJ. 104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle. In: Molecular Therapy. Elsevier BV; 2006. p. S43–S43.
Asokan A, Hamra JB, Agbandje-McKenna M, Samulski RJ. 7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry. In: Molecular Therapy. Elsevier BV; 2006. p. S3–S3.
Asokan A, Cho MJ. Cytosolic delivery of macromolecules 4. Head group-dependent membrane permeabilization by pH-sensitive detergents. J Control Release. 2005 Aug 18;106(1–2):146–53.
Asokan A, Samulski RJ. From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop. In: Mol Ther. 2005. p. 656–60.
Asokan A, Cho MJ. Cytosolic delivery of macromolecules. 3. Synthesis and characterization of acid-sensitive bis-detergents. In: Bioconjug Chem. 2004. p. 1166–73.
Asokan A, Cho MJ. Cytosolic delivery of macromolecules. II. Mechanistic studies with pH-sensitive morpholine lipids. Biochim Biophys Acta. 2003 Apr 1;1611(1–2):151–60.
Chen F-J, Asokan A, Cho MJ. Cytosolic delivery of macromolecules: I. Synthesis and characterization of pH-sensitive acyloxyalkylimidazoles. Biochim Biophys Acta. 2003 Apr 1;1611(1–2):140–50.
Asokan A, Cho MJ. Exploitation of intracellular pH gradients in the cellular delivery of macromolecules. J Pharm Sci. 2002 Apr;91(4):903–13.

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