Aravind Asokan
Surgery, Surgical Sciences
Professor in Surgery
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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Liang R, Press K, Gonzalez T, Asokan A, Su H. Evaluating AAV vectors for HHT gene therapy. In: ANGIOGENESIS. 2023. p. S4–S4.
- 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.
- Fiflis DN, Rey N, Milo S, Asokan A. CRISPR Assisted Trans-Splicing of RNA Fragments. In: MOLECULAR THERAPY. 2023. p. 182–182.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Elmore Z, Oh D, Asokan A. AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress. In: MOLECULAR THERAPY. 2022. p. 71–71.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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(1–2):31–42.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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).
- 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).
- Asokan A. CRISPR genome editing in stem cells turns to gold. Nat Mater. 2019 Oct;18(10):1038–9.
- 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).
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
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