Scholars@Duke

• Background
• 

  Education, Training, & Certifications

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

  Duke Appointment History

  • Instructor in the Department of Surgery, Surgery, Surgical Sciences, Surgery 2018
• 

  Leadership & Clinical Positions at Duke

  • Director of Gene Therapy
    Translating Duke Health Scholar
    Professor
    Department of Surgery
    Department of Molecular Genetics & Microbiology
    Department of Biomedical Engineering
• Research
• 

  External Relationships

  • AstraZeneca
  • Novartis Institutes for BioMedical Research, Inc.
  • StrideBio, Inc.
  • TorqueBio LLC
  • UCB Pharma
• Publications & Artistic Works
• 

  Selected Publications

  • Academic Articles

    • Madigan, Victoria J., Tyne O. Tyson, Julianne A. Yuziuk, Minakshi Pillai, Sven Moller-Tank, and Aravind Asokan. “A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes.” J Virol 93, no. 21 (November 1, 2019). https://doi.org/10.1128/JVI.00943-19.
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    • Abdulhaqq, Shaheed A., Helen Wu, John B. Schell, Katherine B. Hammond, Jason S. Reed, Alfred W. Legasse, Michael K. Axthelm, 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 93, no. 19 (October 1, 2019). https://doi.org/10.1128/JVI.00592-19.
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    • Asokan, Aravind. “CRISPR genome editing in stem cells turns to gold.” Nat Mater 18, no. 10 (October 2019): 1038–39. https://doi.org/10.1038/s41563-019-0491-4.
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    • Madigan, Victoria J., Julianne A. Yuziuk, Anna M. Chiarella, Tyne O. Tyson, Rita M. Meganck, Zachary C. Elmore, Longping V. Tse, Nathaniel A. Hathaway, and Aravind Asokan. “Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription.” Plos Pathog 15, no. 8 (August 2019): e1007988. https://doi.org/10.1371/journal.ppat.1007988.
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    • Albright, Blake H., Katherine E. Simon, Minakshi Pillai, Garth W. Devlin, and Aravind Asokan. “Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses.” J Virol 93, no. 11 (June 1, 2019). https://doi.org/10.1128/JVI.00332-19.
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    • Nelson, Christopher E., Yaoying Wu, Matthew P. Gemberling, Matthew L. Oliver, Matthew A. Waller, Joel D. Bohning, Jacqueline N. Robinson-Hamm, et al. “Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.” Nat Med 25, no. 3 (March 2019): 427–32. https://doi.org/10.1038/s41591-019-0344-3.
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    • Mestre, Humberto, Lauren M. Hablitz, Anna Lr Xavier, Weixi Feng, Wenyan Zou, Tinglin Pu, Hiromu Monai, et al. “Aquaporin-4-dependent glymphatic solute transport in the rodent brain.” Elife 7 (December 18, 2018). https://doi.org/10.7554/eLife.40070.
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    • Meganck, Rita M., Erin K. Borchardt, Ruth M. Castellanos Rivera, Miranda L. Scalabrino, Jeremy E. Wilusz, William F. Marzluff, and Aravind Asokan. “Tissue-Dependent Expression and Translation of Circular RNAs with Recombinant AAV Vectors In Vivo.” Mol Ther Nucleic Acids 13 (December 7, 2018): 89–98. https://doi.org/10.1016/j.omtn.2018.08.008.
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    • Castle, Michael J., Yuhsiang Cheng, Aravind Asokan, and Mark H. Tuszynski. “Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion.” Sci Adv 4, no. 11 (November 2018): eaau9859. https://doi.org/10.1126/sciadv.aau9859.
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    • Tse, Longping V., Sven Moller-Tank, Rita M. Meganck, and Aravind Asokan. “Mapping and Engineering Functional Domains of the Assembly-Activating Protein of Adeno-associated Viruses.” J Virol 92, no. 14 (July 15, 2018). https://doi.org/10.1128/JVI.00393-18.
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    • Crowther, Andrew J., Szu-Aun Lim, Brent Asrican, Blake H. Albright, Josh Wooten, Chia-Yu Yeh, Hechen Bao, et al. “An Adeno-Associated Virus-Based Toolkit for Preferential Targeting and Manipulating Quiescent Neural Stem Cells in the Adult Hippocampus.” Stem Cell Reports 10, no. 3 (March 13, 2018): 1146–59. https://doi.org/10.1016/j.stemcr.2018.01.018.
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    • Albright, Blake H., Claire M. Storey, Giridhar Murlidharan, Ruth M. Castellanos Rivera, Garrett E. Berry, Victoria J. Madigan, and Aravind Asokan. “Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier.” Mol Ther 26, no. 2 (February 7, 2018): 510–23. https://doi.org/10.1016/j.ymthe.2017.10.017.
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    • Burwitz, Benjamin J., Jochen M. Wettengel, Martin A. Mück-Häusl, Marc Ringelhan, Chunkyu Ko, Marvin M. Festag, Katherine B. Hammond, et al. “Hepatocytic expression of human sodium-taurocholate cotransporting polypeptide enables hepatitis B virus infection of macaques.” Nat Commun 8, no. 1 (December 15, 2017): 2146. https://doi.org/10.1038/s41467-017-01953-y.
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    • Guo, Jiami, James M. Otis, Holden Higginbotham, Chase Monckton, JrGang Cheng, Aravind Asokan, Kirk Mykytyn, Tamara Caspary, Garret D. Stuber, and E. S. Anton. “Primary Cilia Signaling Shapes the Development of Interneuronal Connectivity.” Dev Cell 42, no. 3 (August 7, 2017): 286-300.e4. https://doi.org/10.1016/j.devcel.2017.07.010.
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    • Tse, Longping Victor, Kelli A. Klinc, Victoria J. Madigan, Ruth M. Castellanos Rivera, Lindsey F. Wells, L Patrick Havlik, J Kennon Smith, Mavis Agbandje-McKenna, and Aravind Asokan. “Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion.” Proc Natl Acad Sci U S A 114, no. 24 (June 13, 2017): E4812–21. https://doi.org/10.1073/pnas.1704766114.
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    • Borchardt, Erin K., Rita M. Meganck, Heather A. Vincent, Christopher B. Ball, Silvia B. V. Ramos, Nathaniel J. Moorman, William F. Marzluff, and Aravind Asokan. “Inducing circular RNA formation using the CRISPR endoribonuclease Csy4.” Rna 23, no. 5 (May 2017): 619–27. https://doi.org/10.1261/rna.056838.116.
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    • Tarantal, Alice F., C Chang I. Lee, Michele L. Martinez, Aravind Asokan, and R Jude Samulski. “Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.” Hum Gene Ther 28, no. 5 (May 2017): 385–91. https://doi.org/10.1089/hum.2016.130.
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    • Zeng, Cheng, Sven Moller-Tank, Aravind Asokan, and Bogdan Dragnea. “Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2.” J Phys Chem B 121, no. 8 (March 2, 2017): 1843–53. https://doi.org/10.1021/acs.jpcb.6b10131.
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    • Berry, Garrett Edward, and Aravind Asokan. “Cellular transduction mechanisms of adeno-associated viral vectors.” Curr Opin Virol 21 (December 2016): 54–60. https://doi.org/10.1016/j.coviro.2016.08.001.
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    • Tseng, Yu-Shan, Kim Van Vliet, Lavanya Rao, Robert McKenna, Barry J. Byrne, Aravind Asokan, and Mavis Agbandje-McKenna. “Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies.” J Virol Methods 236 (October 2016): 105–10. https://doi.org/10.1016/j.jviromet.2016.07.009.
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    • Murlidharan, Giridhar, Andrew Crowther, Rebecca A. Reardon, Juan Song, and Aravind Asokan. “Glymphatic fluid transport controls paravascular clearance of AAV vectors from the brain.” Jci Insight 1, no. 14 (September 8, 2016): e88034. https://doi.org/10.1172/jci.insight.88034.
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    • Murlidharan, Giridhar, Kensuke Sakamoto, Lavanya Rao, Travis Corriher, Dan Wang, Guangping Gao, Patrick Sullivan, and Aravind Asokan. “CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector.” Mol Ther Nucleic Acids 5, no. 7 (July 19, 2016): e338. https://doi.org/10.1038/mtna.2016.49.
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    • Pierson, Elizabeth E., David Z. Keifer, Aravind Asokan, and Martin F. Jarrold. “Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry.” Anal Chem 88, no. 13 (July 5, 2016): 6718–25. https://doi.org/10.1021/acs.analchem.6b00883.
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    • Huang, Lin-Ya, Ami Patel, Robert Ng, Edward Blake Miller, Sujata Halder, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site.” J Virol 90, no. 11 (June 1, 2016): 5219–30. https://doi.org/10.1128/JVI.00161-16.
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    • Madigan, Victoria J., and Aravind Asokan. “Engineering AAV receptor footprints for gene therapy.” Curr Opin Virol 18 (June 2016): 89–96. https://doi.org/10.1016/j.coviro.2016.05.001.
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    • Leeuw, Charles N. de, Andrea J. Korecki, Garrett E. Berry, Jack W. Hickmott, Siu Ling Lam, Tess C. Lengyell, Russell J. Bonaguro, et al. “rAAV-compatible MiniPromoters for restricted expression in the brain and eye.” Mol Brain 9, no. 1 (May 10, 2016): 52. https://doi.org/10.1186/s13041-016-0232-4.
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    • Vance, Melisa, Telmo Llanga, Will Bennett, Kenton Woodard, Giridhar Murlidharan, Neil Chungfat, Aravind Asokan, et al. “AAV Gene Therapy for MPS1-associated Corneal Blindness.” Sci Rep 6 (February 22, 2016): 22131. https://doi.org/10.1038/srep22131.
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    • Li, Chengwen, Shuqing Wu, Blake Albright, Matthew Hirsch, Wuping Li, Yu-Shan Tseng, Mavis Agbandje-McKenna, Scott McPhee, Aravind Asokan, and R Jude Samulski. “Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.” Mol Ther 24, no. 1 (February 2016): 53–65. https://doi.org/10.1038/mt.2015.134.
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    • Nelson, Christopher E., Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, Eirik A. Moreb, Ruth M. Castellanos Rivera, Sarina Madhavan, et al. “In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.” Science 351, no. 6271 (January 22, 2016): 403–7. https://doi.org/10.1126/science.aad5143.
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    • Berry, Garrett E., and Aravind Asokan. “Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction.” J Biol Chem 291, no. 2 (January 8, 2016): 939–47. https://doi.org/10.1074/jbc.M115.687657.
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    • Borchardt, Erin K., Leonidas A. Vandoros, Michael Huang, Patrick E. Lackey, William F. Marzluff, and Aravind Asokan. “Controlling mRNA stability and translation with the CRISPR endoribonuclease Csy4.” Rna 21, no. 11 (November 2015): 1921–30. https://doi.org/10.1261/rna.051227.115.
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    • Schreiber, Claire A., Toshie Sakuma, Yoshihiro Izumiya, Sara J. Holditch, Raymond D. Hickey, Robert K. Bressin, Upamanyu Basu, Kazunori Koide, Aravind Asokan, and Yasuhiro Ikeda. “An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses.” Plos Pathog 11, no. 8 (August 2015): e1005082. https://doi.org/10.1371/journal.ppat.1005082.
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    • Tse, Longping V., Sven Moller-Tank, and Aravind Asokan. “Strategies to circumvent humoral immunity to adeno-associated viral vectors.” Expert Opin Biol Ther 15, no. 6 (June 2015): 845–55. https://doi.org/10.1517/14712598.2015.1035645.
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    • Hemphill, James, Erin K. Borchardt, Kalyn Brown, Aravind Asokan, and Alexander Deiters. “Optical Control of CRISPR/Cas9 Gene Editing.” J Am Chem Soc 137, no. 17 (May 6, 2015): 5642–45. https://doi.org/10.1021/ja512664v.
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    • Murlidharan, Giridhar, Travis Corriher, H Troy Ghashghaei, and Aravind Asokan. “Unique glycan signatures regulate adeno-associated virus tropism in the developing brain.” J Virol 89, no. 7 (April 2015): 3976–87. https://doi.org/10.1128/JVI.02951-14.
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    • Shen, Shen, Garrett E. Berry, Ruth M. Castellanos Rivera, Roland Y. Cheung, Andrew N. Troupes, Sarah M. Brown, Tal Kafri, and Aravind Asokan. “Functional analysis of the putative integrin recognition motif on adeno-associated virus 9.” J Biol Chem 290, no. 3 (January 16, 2015): 1496–1504. https://doi.org/10.1074/jbc.M114.608281.
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    • Ishikawa, Kiyotake, Kenneth M. Fish, Lisa Tilemann, Kleopatra Rapti, Jaume Aguero, Carlos G. Santos-Gallego, Ahyoung Lee, et al. “Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.” Mol Ther 22, no. 12 (December 2014): 2038–45. https://doi.org/10.1038/mt.2014.127.
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    • Pleticha, Josef, Lukas F. Heilmann, Christopher H. Evans, Aravind Asokan, Richard Jude Samulski, and Andreas S. Beutler. “Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs.” Mol Pain 10 (September 2, 2014): 54. https://doi.org/10.1186/1744-8069-10-54.
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    • O’Reilly, Marina, Howard J. Federoff, Yuman Fong, Donald B. Kohn, Amy P. Patterson, Nabil Ahmed, Aravind Asokan, et al. “Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.” Hum Gene Ther 25, no. 6 (June 2014): 488–97. https://doi.org/10.1089/hum.2014.045.
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    • Murlidharan, Giridhar, Richard J. Samulski, and Aravind Asokan. “Biology of adeno-associated viral vectors in the central nervous system.” Front Mol Neurosci 7 (2014): 76. https://doi.org/10.3389/fnmol.2014.00076.
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    • Shen, Shen, Andrew N. Troupes, Nagesh Pulicherla, and Aravind Asokan. “Multiple roles for sialylated glycans in determining the cardiopulmonary tropism of adeno-associated virus 4.” J Virol 87, no. 24 (December 2013): 13206–13. https://doi.org/10.1128/JVI.02109-13.
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    • Asokan, Aravind, and R Jude Samulski. “An emerging adeno-associated viral vector pipeline for cardiac gene therapy.” Hum Gene Ther 24, no. 11 (November 2013): 906–13. https://doi.org/10.1089/hum.2013.2515.
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    • Shen, Shen, Eric D. Horowitz, Andrew N. Troupes, Sarah M. Brown, Nagesh Pulicherla, Richard J. Samulski, Mavis Agbandje-McKenna, and Aravind Asokan. “Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency.” J Biol Chem 288, no. 40 (October 4, 2013): 28814–23. https://doi.org/10.1074/jbc.M113.482380.
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    • Horowitz, Eric D., K Shefaet Rahman, Brian D. Bower, David J. Dismuke, Michael R. Falvo, Jack D. Griffith, Stephen C. Harvey, and Aravind Asokan. “Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release.” J Virol 87, no. 6 (March 2013): 2994–3002. https://doi.org/10.1128/JVI.03017-12.
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    • Shen, Shen, Kelli D. Bryant, Junjiang Sun, Sarah M. Brown, Andrew Troupes, Nagesh Pulicherla, and Aravind Asokan. “Glycan binding avidity determines the systemic fate of adeno-associated virus type 9.” J Virol 86, no. 19 (October 2012): 10408–17. https://doi.org/10.1128/JVI.01155-12.
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    • Li, Chengwen, Nina Diprimio, Dawn E. Bowles, Matthew L. Hirsch, Paul E. Monahan, Aravind Asokan, Joseph Rabinowitz, Mavis Agbandje-McKenna, and R Jude Samulski. “Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles.” J Virol 86, no. 15 (August 2012): 7752–59. https://doi.org/10.1128/JVI.00675-12.
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    • Horowitz, Eric D., M. G. Finn, and Aravind Asokan. “Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection.” Acs Chem Biol 7, no. 6 (June 15, 2012): 1059–66. https://doi.org/10.1021/cb3000265.
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    • Denard, Jerome, Cyriaque Beley, Robert Kotin, René Lai-Kuen, Stéphane Blot, Hervé Leh, Aravind Asokan, et al. “Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.” J Virol 86, no. 12 (June 2012): 6620–31. https://doi.org/10.1128/JVI.00297-12.
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    • Asokan, Aravind, David V. Schaffer, and R Jude Samulski. “The AAV vector toolkit: poised at the clinical crossroads.” Mol Ther 20, no. 4 (April 2012): 699–708. https://doi.org/10.1038/mt.2011.287.
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    • Li, C., N. Narkbunnam, R. J. Samulski, A. Asokan, G. Hu, L. J. Jacobson, M. J. Manco-Johnson, P. E. Monahan, and P. E. Joint Outcome Study Investigators. “Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.” Gene Ther 19, no. 3 (March 2012): 288–94. https://doi.org/10.1038/gt.2011.90.
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    • Pulicherla, Nagesh, Pradeep Kota, Nikolay V. Dokholyan, and Aravind Asokan. “Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsids.” Plos One 7, no. 2 (2012): e32163. https://doi.org/10.1371/journal.pone.0032163.
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    • Gray, Steven J., Vivian W. Choi, Aravind Asokan, Rebecca A. Haberman, Thomas J. McCown, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration.” Curr Protoc Neurosci Chapter 4 (October 2011): Unit-4.17. https://doi.org/10.1002/0471142301.ns0417s57.
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    • Pulicherla, N., and A. Asokan. “Peptide affinity reagents for AAV capsid recognition and purification.” Gene Ther 18, no. 10 (October 2011): 1020–24. https://doi.org/10.1038/gt.2011.46.
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    • Pulicherla, Nagesh, Shen Shen, Swati Yadav, Kari Debbink, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and Aravind Asokan. “Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.” Mol Ther 19, no. 6 (June 2011): 1070–78. https://doi.org/10.1038/mt.2011.22.
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    • Horowitz, Eric D., Marc S. Weinberg, and Aravind Asokan. “Glycated AAV vectors: chemical redirection of viral tissue tropism.” Bioconjug Chem 22, no. 4 (April 20, 2011): 529–32. https://doi.org/10.1021/bc100477g.
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    • Shen, Shen, Kelli D. Bryant, Sarah M. Brown, Scott H. Randell, and Aravind Asokan. “Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.” J Biol Chem 286, no. 15 (April 15, 2011): 13532–40. https://doi.org/10.1074/jbc.M110.210922.
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    • Kapoor, Amit, Mady Hornig, Aravind Asokan, Brent Williams, Jose A. Henriquez, and W Ian Lipkin. “Bocavirus episome in infected human tissue contains non-identical termini.” Plos One 6, no. 6 (2011): e21362. https://doi.org/10.1371/journal.pone.0021362.
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    • Phillips, Jana L., Julia Hegge, Jon A. Wolff, R Jude Samulski, and Aravind Asokan. “Systemic gene transfer to skeletal muscle using reengineered AAV vectors.” Methods Mol Biol 709 (2011): 141–51. https://doi.org/10.1007/978-1-61737-982-6_9.
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    • Asokan, Aravind. “Reengineered AAV vectors: old dog, new tricks.” Discov Med 9, no. 48 (May 2010): 399–403.
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    • Asokan, Aravind, Julia C. Conway, Jana L. Phillips, Chengwen Li, Julia Hegge, Rebecca Sinnott, Swati Yadav, et al. “Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle.” Nat Biotechnol 28, no. 1 (January 2010): 79–82. https://doi.org/10.1038/nbt.1599.
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    • Li, Chengwen, Matt Hirsch, Nina DiPrimio, Aravind Asokan, Kevin Goudy, Roland Tisch, and R Jude Samulski. “Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo.” J Virol 83, no. 13 (July 2009): 6817–24. https://doi.org/10.1128/JVI.00278-09.
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    • Li, Chengwen, Kevin Goudy, Matt Hirsch, Aravind Asokan, Yun Fan, Jeff Alexander, Junjiang Sun, et al. “Cellular immune response to cryptic epitopes during therapeutic gene transfer.” Proc Natl Acad Sci U S A 106, no. 26 (June 30, 2009): 10770–74. https://doi.org/10.1073/pnas.0902269106.
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    • Li, C., M. Hirsch, P. Carter, A. Asokan, X. Zhou, Z. Wu, and R. J. Samulski. “A small regulatory element from chromosome 19 enhances liver-specific gene expression.” Gene Ther 16, no. 1 (January 2009): 43–51. https://doi.org/10.1038/gt.2008.134.
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    • Asokan, A., J. S. Johnson, C. Li, and R. J. Samulski. “Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals.” Gene Ther 15, no. 24 (December 2008): 1618–22. https://doi.org/10.1038/gt.2008.127.
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    • Li, Wuping, Aravind Asokan, Zhijian Wu, Terry Van Dyke, Nina DiPrimio, Jarrod S. Johnson, Lakshmanan Govindaswamy, et al. “Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.” Mol Ther 16, no. 7 (July 2008): 1252–60. https://doi.org/10.1038/mt.2008.100.
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    • DiPrimio, Nina, Aravind Asokan, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and R Jude Samulski. “Surface loop dynamics in adeno-associated virus capsid assembly.” J Virol 82, no. 11 (June 2008): 5178–89. https://doi.org/10.1128/JVI.02721-07.
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    • Li, Chengwen, Matthew Hirsch, Aravind Asokan, Brian Zeithaml, Hong Ma, Tal Kafri, and R Jude Samulski. “Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo.” J Virol 81, no. 14 (July 2007): 7540–47. https://doi.org/10.1128/JVI.00529-07.
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    • Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors for in vitro and in vivo use.” Curr Protoc Mol Biol Chapter 16 (April 2007): Unit-16.25. https://doi.org/10.1002/0471142727.mb1625s78.
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    • Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors.” Curr Protoc Hum Genet Chapter 12 (April 2007): Unit-12.9. https://doi.org/10.1002/0471142905.hg1209s53.
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    • Wu, Zhijian, Aravind Asokan, Joshua C. Grieger, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and R Jude Samulski. “Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes.” J Virol 80, no. 22 (November 2006): 11393–97. https://doi.org/10.1128/JVI.01288-06.
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    • Asokan, Aravind, Julie B. Hamra, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, and Richard J. Samulski. “Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry.” J Virol 80, no. 18 (September 2006): 8961–69. https://doi.org/10.1128/JVI.00843-06.
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    • Wu, Zhijian, Aravind Asokan, and R Jude Samulski. “Adeno-associated virus serotypes: vector toolkit for human gene therapy.” Mol Ther 14, no. 3 (September 2006): 316–27. https://doi.org/10.1016/j.ymthe.2006.05.009.
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    • Choi, Vivian W., Aravind Asokan, Rebecca A. Haberman, Thomas J. McCown, and Richard Jude Samulski. “Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administration.” Curr Protoc Neurosci Chapter 4 (May 2006): Unit-4.17. https://doi.org/10.1002/0471142301.ns0417s35.
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    • Asokan, Aravind, and R Jude Samulski. “AAV does the shuffle.” Nat Biotechnol 24, no. 2 (February 2006): 158–60. https://doi.org/10.1038/nbt0206-158.
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    • Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules 4. Head group-dependent membrane permeabilization by pH-sensitive detergents.” J Control Release 106, no. 1–2 (August 18, 2005): 146–53. https://doi.org/10.1016/j.jconrel.2005.04.010.
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    • Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules. II. Mechanistic studies with pH-sensitive morpholine lipids.” Biochim Biophys Acta 1611, no. 1–2 (April 1, 2003): 151–60. https://doi.org/10.1016/s0005-2736(03)00050-6.
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    • Chen, Feng-Jing, Aravind Asokan, and Moo J. Cho. “Cytosolic delivery of macromolecules: I. Synthesis and characterization of pH-sensitive acyloxyalkylimidazoles.” Biochim Biophys Acta 1611, no. 1–2 (April 1, 2003): 140–50. https://doi.org/10.1016/s0005-2736(03)00049-x.
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    • Asokan, Aravind, and Moo J. Cho. “Exploitation of intracellular pH gradients in the cellular delivery of macromolecules.” J Pharm Sci 91, no. 4 (April 2002): 903–13. https://doi.org/10.1002/jps.10095.
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  • Book Sections

    • Murlidharan, G., R. J. Samulski, and A. Asokan. “Gene therapy of CNS disorders using recombinant AAV vectors.” In Translational Neuroscience: Fundamental Approaches for Neurological Disorders, 9–24, 2016. https://doi.org/10.1007/978-1-4899-7654-3_2.
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    • Asokan, A., R. Cheung, and M. J. Cho. “Strategies for the Cytosolic Delivery of Macromolecules: An Overview.” In Handbook of Pharmaceutical Biotechnology, 279–96, 2006. https://doi.org/10.1002/9780470117118.ch3c.
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  • Conference Papers

    • Bulaklak, Karen, Jacqueline Robinson-Hamm, Veronica Gough, Christopher E. Nelson, Victoria Madigan, Aravind Asokan, and Charles A. Gersbach. “AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy.” In Molecular Therapy, 27:377–377. CELL PRESS, 2019.
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    • Castle, Michael J., Yuhsiang Cheng, Aravind Asokan, and Mark H. Tuszynski. “Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9.” In Molecular Therapy, 27:97–98. CELL PRESS, 2019.
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    • Gonzalez, Trevor J., Rita Meganck, Marco Fanous, Kate Simon, and Aravind Asokan. “Design Principles for AAV Mediated Circular RNA Expression in the Brain.” In Molecular Therapy, 27:60–60. CELL PRESS, 2019.
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    • Havlik, L Patrick, L Victor Tse, J Kennon Smith, Kelli A. Klinc, Daniel Oh, Katherine Simon, Wenwei Shao, Chengwen Li, Mavis Agbandje-McKenna, and Aravind Asokan. “Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution.” In Molecular Therapy, 27:53–53. CELL PRESS, 2019.
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    • Madigan, Victoria, Tyne O. Tyson, Julianne A. Yuziuk, Minakshi Pillai, Sven Moller-Tank, and Aravind Asokan. “A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor.” In Molecular Therapy, 27:424–25. CELL PRESS, 2019.
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    • Madigan, Victoria, Tyne O. Tyson, and Aravind Asokan. “Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription.” In Molecular Therapy, 27:425–425. CELL PRESS, 2019.
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    • Nelson, Christopher, Yaoying Wu, Matthew Gemberling, Matthew Oliver, Joel D. Bohning, Jacqueline N. Robinson-Hamm, Karen Bulaklak, et al. “Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy.” In Molecular Therapy, 27:46–47. CELL PRESS, 2019.
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    • Burwitz, Benjamin, Jochen Wettengel, Martin Muck-Hausl, Katherine Hammond, Marc Ringelhan, Chunkyu Ko, Jason Reed, 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, 47:324–324. WILEY, 2018.
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    • Albright, Blake H., Claire Storey, Garth Devlin, Mavis Agbandje-McKenna, and Aravind Asokan. “Capsid-Glycan Receptor Interactions Influence AAV Transport across the Blood-Brain Barrier.” In Molecular Therapy, 26:29–29. CELL PRESS, 2018.
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    • Madigan, Victoria J., Julianne Yuziuk, Longping V. Tse, Anna Chiarella, Tyne Tyson, Nate Hathaway, and Aravind Asokan. “RNF121 is a Key Transcriptional Regulator of AAV Genome Expression.” In Molecular Therapy, 26:27–27. CELL PRESS, 2018.
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    • Meganck, Rita M., Erin K. Bochardt, Ruth M. Castellanos, Miranda L. Scalabrino, Jeremy Wilusz, William F. Marzluff, and Aravind Asokan. “Systemic Expression of Translatable Circular Rnas Using Recombinant AAV Vectors.” In Molecular Therapy, 26:273–273. CELL PRESS, 2018.
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    • Nelson, Christopher, Matthew Gemberling, Matthew L. Oliver, Chady H. Hakim, Ruth M Castellanos Rivera, Aravind Asokan, Dongsheng Duan, and Charles A. Gersbach. “Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy.” In Molecular Therapy, 26:431–32. CELL PRESS, 2018.
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    • Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Matthew Gemberling, Veronica Gough, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9.” In Molecular Therapy, 26:118–19. CELL PRESS, 2018.
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    • Tse, Longping V., Sven Moller-Tank, Rita M. Meganck, Mavis Agbandje-McKenna, and Aravind Asokan. “Mapping and Engineering Functional Domains of the Assembly Activating Protein of Adeno-Associated Viruses.” In Molecular Therapy, 26:27–28. CELL PRESS, 2018.
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    • Albright, Blake, Claire Storey, Giridhar Murlidharan, Ruth Castellanos Rivera, and Aravind Asokan. “Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain Barrier.” In Molecular Therapy, 25:230–31. CELL PRESS, 2017.
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    • Moller-Tank, Sven, Ruth M. Castellanos, Daniel Oh, Longping V. Tse, Miranda L. Scalabrino, and Aravind Asokan. “Expanding the CNS Transduction Profile of AAV4 by Structure-Guided Evolution.” In Molecular Therapy, 25:247–247. CELL PRESS, 2017.
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    • Nelson, Christopher, Matthew Gemberling, Sarina Madhavan, Ruth M Castellanos Rivera, Aravind Asokan, and Charles A. Gersbach. “Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy.” In Molecular Therapy, 25:100–101. CELL PRESS, 2017.
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    • Rivera, Ruth M Castellanos, Austin D. Sun, Marco Fanous, and Aravind Asokan. “Disruption of Water Homeostasis Affects AAV Transport Across the Blood-Brain Barrier.” In Molecular Therapy, 25:109–109. CELL PRESS, 2017.
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    • Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Matthew Gemberling, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9.” In Molecular Therapy, 25:17–17. CELL PRESS, 2017.
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    • Smith, J Kennon S., Yu-Shan Tseng, Daniel Noe, Mathias Kaplan, Lavanya Rao, Robert McKenna, Sergei Zolotukhin, et al. “Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape Variants.” In Molecular Therapy, 25:337–337. CELL PRESS, 2017.
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    • Tse, Longping V., Kelli A. Klinc, Victoria J. Madigan, Ruth M Castellanos Rivera, Lindsey F. Wells, L Patrick Havlik, J Kennon Smith, Mavis Agbandje-McKenna, and Aravind Asokan. “Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene Transfer.” In Molecular Therapy, 25:232–232. CELL PRESS, 2017.
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    • Berry, Garrett, Giridhar Murlidharan, and Aravind Asokan. “30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS.” In Molecular Therapy, 24:S14–S14. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32839-8.
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    • Huang, Lin-Ya, Ami Patel, Robert Ng, Edward B. Miller, Sujata Halder, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “727. Characterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopes.” In Molecular Therapy, 24:S287–S287. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33535-3.
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    • Murlidharan, Giridhar, and Aravind Asokan. “Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer.” In Molecular Therapy, 24:S24–S24. NATURE PUBLISHING GROUP, 2016.
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    • Nelson, Christopher, Matthew Gemberling, Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, Ruth M. Castellanos, Sarina Madhavan, et al. “482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy.” In Molecular Therapy, 24:S191–S191. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33291-9.
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    • Robinson-Hamm, Jacqueline N., Christopher E. Nelson, Ruth M Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, and Charles A. Gersbach. “504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy.” In Molecular Therapy, 24:S201–S201. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)33313-5.
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    • Schreiber, Claire A., Yoshihiro Izumiya, Aravind Asokan, and Yasuhiro Ikeda. “8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction.” In Molecular Therapy, 24:S4–5. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32817-9.
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    • Schreiber, C. A., T. Sakuma, Y. Izumiya, R. K. Bressin, U. Basu, K. Koide, A. Asokan, and Y. Ikeda. “Enhanced AAV vector transduction through genetic and pharmacological inhibition of U2 snRNP components.” In Human Gene Therapy, 26:A3–4. MARY ANN LIEBERT, INC, 2015.
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    • Woodard, Kenton, Katharine J. Liang, Thomas Lentz, William Bennett, Aravind Asokan, and Richard Samulski. “Transduction of Mouse Retina by Intravitreal Delivery Using Chimeric Capsids with Altered Glycan Interactions.” In Investigative Ophthalmology & Visual Science, Vol. 56. ASSOC RESEARCH VISION OPHTHALMOLOGY INC, 2015.
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    • Albright, Blake H., Victoria Madigan, Lavanya Rao, John A. Chiorini, Mavis Agbandje-McKenna, and Aravind Asokan. “302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors.” In Molecular Therapy, 23:S122–S122. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33911-9.
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    • Berry, Garrett E., and Aravind Asokan. “38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction.” In Molecular Therapy, 23:S17–S17. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33643-7.
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    • Murlidharan, Giridhar, Lavanya Rao, Dan Wang, Travis Corriher, Kyung Seok-Oh, Guangping Gao, R. Jude Samulski, Alice F. Tarantal, and Aravind Asokan. “14. Next Generation AAV Vectors for Limiting Systemic Leakage and Improving Safety Following CNS Administration.” In Molecular Therapy, 23:S6–S6. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33618-8.
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    • Murlidharan, Giridhar, and Aravind Asokan. “265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain.” In Molecular Therapy, 23:S106–S106. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33874-6.
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    • Nelson, Christopher E., Ruth M. Castellanos, Fei Ann Ran, Winston Yan, Feng Zhang, Aravind Asokan, and Charles A. Gersbach. “397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy.” In Molecular Therapy, 23:S157–58. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)34006-0.
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    • Rao, Lavanya, Blake H. Albright, Travis Corriher, Giridhar Murlidharan, and Aravind Asokan. “42. Differential Transduction Profiles of AAV Vectors in a Mouse Model of Human Glycosylation.” In Molecular Therapy, 23:S18–19. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33647-4.
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    • Rivera, Ruth M Castellanos, Lavanya Rao, Giridhar Murlidharan, Travis Corriher, and Aravind Asokan. “Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors.” In Molecular Therapy, 23:S77–S77. NATURE PUBLISHING GROUP, 2015.
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    • Schreiber, Claire, Toshie Sakuma, Yoshihiro Izumiya, Sara Holditch, Raymond Hickey, Robert Bressin, Upamanyu Basu, Kazunori Koide, Aravind Asokan, and Yasuhiro Ikeda. “41. U2 snRNP Spliceosome Proteins Block Recombinant AAV Vector Transduction.” In Molecular Therapy, 23:S18–S18. Elsevier BV, 2015. https://doi.org/10.1016/s1525-0016(16)33646-2.
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    • Huang, Lin-Ya, Robert Ng, Edward B. Miller, Robert McKenna, Aravind Asokan, and Mavis Agbandje-McKenna. “Characterizing Essential Receptor Binding Residues for AAV1.” In Molecular Therapy, 22:S2–S2. NATURE PUBLISHING GROUP, 2014.
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    • Simpson, Elizabeth M., Charles N. de Leeuw, Siu Ling Lam, Andrea J. Korecki, Russell J. Bonaguro, Kaelan Wong, Michelle Zhou, et al. “Regional-CNS MiniPromoters for AAV Are Identified in a High Through-Put Pipeline.” In Molecular Therapy, 22:S112–S112. NATURE PUBLISHING GROUP, 2014.
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    • Tseng, Yu-Shan, Kim Van Vliet, Matias Kaplan, J Kennon Smith, Lavanya Rao, Robert McKenna, Barry J. Byrne, Sergei Zolotukhin, Aravind Asokan, and Mavis Agbandje-McKenna. “Mapping the Antigenic Structure of Adeno-Associated Virus Serotypes 8 and 9.” In Molecular Therapy, 22:S1–S1. NATURE PUBLISHING GROUP, 2014.
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    • Murlidharan, Giridhar, Laura Sommerville, Troy Ghashghaei, and Aravind Asokan. “Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4.” In Molecular Therapy, 21:S1–S1. NATURE PUBLISHING GROUP, 2013.
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    • Shen, Shen, Andrew Troupes, Nagesh Pulicherla, and Aravind Asokan. “Sialic Acids and Erythrocyte Interactions Are Critical Determinants of Cardiopulmonary Tropism Displayed by AAV Serotype 4.” In Molecular Therapy, 21:S31–S31. NATURE PUBLISHING GROUP, 2013.
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    • Shen, Shen, and Aravind Asokan. “Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo.” In Molecular Therapy, 21:S30–S30. NATURE PUBLISHING GROUP, 2013.
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    • Denard, Jerome, Cyriaque Beley, Robert Kotin, Rene Lai-Kuen, Stephane Blot, Herve Leh, Aravind Asokan, et al. “Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.” In Human Gene Therapy, 23:A122–A122. MARY ANN LIEBERT INC, 2012.
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    • Horowitz, Eric D., and Aravind Asokan. “Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating.” In Molecular Therapy, 20:S202–S202. NATURE PUBLISHING GROUP, 2012.
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    • Shen, Shen, Junjiang Sun, Matthew Hirsch, Sai Chavala, Paul Monahan, and Aravind Asokan. “Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors.” In Molecular Therapy, 20:S203–S203. NATURE PUBLISHING GROUP, 2012.
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    • Monahan, P. E., C. Li, N. Narkbunnam, R. J. Samulski, G. Hu, A. Asokan, and M. J. Manco-Johnson. “Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.” In Journal of Thrombosis and Haemostasis, 9:231–32. WILEY-BLACKWELL, 2011.
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    • Xiao, Ping-Jie, Jarrod S. Johnson, Aaron Neumann, Xinghua Zeng, Aravind Asokan, and Richard J. Samulski. “Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells.” In Molecular Therapy, 17:S38–S38. NATURE PUBLISHING GROUP, 2009.
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    • Asokan, Aravind, Jarrod S. Johnson, and Richard J. Samulski. “739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue.” In Molecular Therapy, 13:S286–S286. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.821.
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    • Asokan, Aravind, Julie B. Hamra, Mavis Agbandje-McKenna, and Richard J. Samulski. “7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry.” In Molecular Therapy, 13:S3–S3. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.017.
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    • DiPrimio, Nina, Aravind Asokan, and Richard J. Samulski. “104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle.” In Molecular Therapy, 13:S43–S43. Elsevier BV, 2006. https://doi.org/10.1016/j.ymthe.2006.08.124.
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    • Asokan, Arvind, and Richard J. Samulski. “From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop.” In Mol Ther, 11:656–60, 2005. https://doi.org/10.1016/j.ymthe.2005.03.014.
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    • Asokan, Aravind, and Moo J. Cho. “Cytosolic delivery of macromolecules. 3. Synthesis and characterization of acid-sensitive bis-detergents.” In Bioconjug Chem, 15:1166–73, 2004. https://doi.org/10.1021/bc049880n.
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