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

Perelli RM, Sitton MJ, Bohning JD, Pickar-Oliver A, McCullough KT, Moya-Mendez ME, et al. Deletion of Exon 51 in a Humanized Duchenne Muscular Dystrophy Mouse Model Abolishes Ventricular Arrhythmia Predisposition. Circulation Genomic and precision medicine. 2025 Apr;e004867.
Asokan A, Wilson MH. Filtering through AAV capsid libraries for effective kidney gene transfer. Kidney Int. 2025 Apr;107(4):591u20133.
Cheng M, Cruz DDL, Crain AV, Espinoza P, Ng C, Elmore ZC, et al. Probing aspects of extracellular vesicle associated AAV allows increased vector yield and insight into its transduction and immune-evasive properties. Mol Ther Methods Clin Dev. 2025 Mar 13;33(1):101407.
Shapiro DM, Deshpande S, Eghtesadi SA, Zhong M, Fontes CM, Fiflis D, et al. Synthetic biomolecular condensates enhance translation from a target mRNA in living cells. Nat Chem. 2025 Mar;17(3):448u201356.
Nyberg WA, Wang CH, Ark J, Liu C, Clouden S, Qualls A, et al. Inu00a0vivo engineering of murine Tu00a0cells using the evolved adeno-associated virus variant Ark313. Immunity. 2025 Feb 11;58(2):499-512.e7.
Rosales A, Blondel LO, Hull J, Gao Q, Aykun N, Peek JL, et al. Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries. Nat Biomed Eng. 2025 Feb 5;
Martinino A, Smith TJ, Elmore ZC, Yoon J, Ladowski J, Schiliro D, et al. An IgM Cleaving Enzyme for Clearance of Anti-Pig Xenoreactive Antibodies in a Nonhuman Primate Model. Xenotransplantation. 2025;32(1):e70015.
Martinino A, Smith TJ, Elmore ZC, Yoon J, Ladowski J, Schiliro D, et al. An IgM Cleaving Enzyme for Clearance of Anti-Pig Xenoreactive Antibodies in a Nonhuman Primate Model. Xenotransplantation. 2025;32(1):e70023.
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 u03b32 models human disease. Mol Genet Metab. 2024 Dec;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):2080u201393.
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. Res Sq. 2024 Jun 12;
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 to translational science. 2024 May;9(5):674u201386.
Gao Q, Kahan R, Gonzalez TJ, Zhang M, Alderete IS, DeLaura I, et al. Gene delivery followed by exu00a0vivo lung perfusion using an adeno-associated viral vector in a rodent lung transplant model. J Thorac Cardiovasc Surg. 2024 May;167(5):e131u20139.
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.
Cheng M, de la Cruz D, Ng C, Elmore Z, Asokan A, Maguire CA. New Biological Insights into Extracellular Vesicle Associated AAV Help Improve Vector Yield and Provides Rational for Intraluminal AAV Packing. In: MOLECULAR THERAPY. 2024. p. 118u20139.
Alderete I, Kahan R, Gao Q, Zhang M, Abraham N, Gonzalez T, et al. AAV Delivery of PD-L1 with Concomitant CTLA-4 Immunoglobulin Attenuates Acute Cellular Rejection in a Rat Lung Transplant Model. In: JOURNAL OF HEART AND LUNG TRANSPLANTATION. 2024. p. S94u20135.
Clements KN, Gonzalez TJ, Asokan A. Engineering Synthetic circRNAs for Efficient CNS Expression. Methods Mol Biol. 2024;2765:227u201346.
Asokan A, Shen S. Redirecting AAV vectors to extrahepatic tissues. Mol Ther. 2023 Dec 6;31(12):3371u20135.
Asokan A. AAV vector immunotoxicity: Stopping the domino effect. Mol Ther. 2023 Dec 6;31(12):3357u20138.
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):3413u201359.
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):255u201365.
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. e38u201349.
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. 107377u2013107377.
Nyberg WA, Ark J, To A, Clouden S, Reeder G, Muldoon JJ, et al. An evolved AAV variant enables efficient genetic engineering of murine Tu00a0cells. 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.
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. S150u2013S150.
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. 136u2013136.
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. 460u20131.
Loeb E, Havlik LP, Asokan A. Capsid Mediated Control of Adeno-Associated Viral Genome Transcription. In: MOLECULAR THERAPY. 2023. p. 137u2013137.
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. 670u2013670.
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. 538u2013538.
Tarantal AF, Martinez M, Sanz L, Lee C, Ou2019Geen H, Asokan A, et al. AAV Serotype Tropism and Editing in Young Rhesus Monkeys. In: MOLECULAR THERAPY. 2023. p. 42u201342.
Fiflis DN, Rey N, Milo S, Asokan A. CRISPR Assisted Trans-Splicing of RNA Fragments. In: MOLECULAR THERAPY. 2023. p. 182u2013182.
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.
Fusco RM, Walton E, Asokan A. A Platform for Interrogating Transgene Silencing and Epigenetic Signatures of AAV Genomes. In: MOLECULAR THERAPY. 2023. p. 239u2013239.
Liang R, Press K, Gonzalez T, Asokan A, Su H. Evaluating AAV vectors for HHT gene therapy. In: ANGIOGENESIS. 2023. p. S4u2013S4.
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. S178u2013S178.
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. 180u2013180.
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. 788u2013788.
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. 96u201396.
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. 104u2013104.
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. 113u2013113.
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. Front Transplant. 2023;2:1171272.
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.
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.
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. Journal of virology. 2022 Feb;96(4):e0203921.
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. 389u2013389.
Jeon K, Gold S, Hu M, Smith JK, Ou2019Banion 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. 482u2013482.
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. 43u201343.
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. 340u2013340.
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. 272u2013272.
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. 204u2013204.
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. A174u2013A174.
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.
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. 339u2013339.
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. 425u20136.
Smith TJ, Elmore ZC, Asokan A. Furin is a Host Factor Restricting Adeno-Associated Virus 4 Transduction. In: MOLECULAR THERAPY. 2022. p. 206u2013206.
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. 430u2013430.
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 u03b32 in Mice. In: MOLECULAR THERAPY. 2022. p. 478u20139.
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. 458u2013458.
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. S38u20139.
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. 67u201367.
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.
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. 211u20132.
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. 205u20136.
Elmore Z, Oh D, Asokan A. AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress. In: MOLECULAR THERAPY. 2022. p. 71u201371.
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. 555u2013555.
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. Molecular therapyu202f: the journal of the American Society of Gene Therapy. 2021 Nov;29(11):3243u201357.
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):965u201374.
Gibson RA, Lim J-A, Choi SJ, Flores L, Clinton L, Bali D, et al. Characterization of liver GSD IX u03b32 pathophysiology in a novel Phkg2^-/- mouse model. Molecular genetics and metabolism. 2021 Jul;133(3):269u201376.
Mitchell-Dick A, Asokan A. AAV-CNS matters turn from gray to white. Mol Ther. 2021 May 5;29(5):1659u201360.
A Beautiful Mind and the Heart of an Explorer. Human Gene Therapy. 2021 Apr 1;32(7u20138):321u20136.
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(7u20138):405u201319.
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):195u2013204.
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;23:821u201334.
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.
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(1u20132):31u201342.
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. 153u20134.
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. 47u201347.
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. 151u2013151.
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. 151u20132.
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. 150u20131.
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. 239u2013239.
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. 153u2013153.
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. 32u201332.
Kwon JB, Ettyreddy AR, Vankara A, Bohning JD, Devlin G, Hauschka SD, et al. Inu00a0Vivo 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;19:320u20139.
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):9u201316.
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).
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):1069u201378.
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. Journal of virology. 2020 Sep;94(19):e00976u2013e00920.
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(15u201316):839u201351.
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):181u20136.
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. 330u2013330.
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. 398u2013398.
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. 492u2013492.
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. 12u20133.
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. 21u201321.
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. 44u201344.
Havlik LP, Elmore ZC, Oh DK, Smith TJ, Asokan A. Evolving New AAV Strains that Demonstrate AAVR Independence. In: MOLECULAR THERAPY. 2020. p. 245u20136.
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):1038u20139.
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Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther. 2006 Sep;14(3):316u201327.
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):8961u20139.
Asokan A, Cheung R, Cho MJ. Strategies for the Cytosolic Delivery of Macromolecules: An Overview. In: Handbook of Pharmaceutical Biotechnology. 2006. p. 279u201396.
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):158u201360.
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. S286u2013S286.
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. S3u2013S3.
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. S43u2013S43.
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(1u20132):146u201353.
Asokan A, Samulski RJ. From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop. In: Mol Ther. 2005. p. 656u201360.
Asokan A, Cho MJ. Cytosolic delivery of macromolecules. 3. Synthesis and characterization of acid-sensitive bis-detergents. In: Bioconjug Chem. 2004. p. 1166u201373.
Asokan A, Cho MJ. Cytosolic delivery of macromolecules. II. Mechanistic studies with pH-sensitive morpholine lipids. Biochim Biophys Acta. 2003 Apr 1;1611(1u20132):151u201360.
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(1u20132):140u201350.
Asokan A, Cho MJ. Exploitation of intracellular pH gradients in the cellular delivery of macromolecules. J Pharm Sci. 2002 Apr;91(4):903u201313.

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