Synthetic Virology & Gene Therapy
Havlik, L Patrick, Anshuman Das, Mario Mietzsch, Daniel K. Oh, Jonathan Ark, Robert McKenna, Mavis Agbandje-McKenna, and Aravind Asokan. “Receptor switching in newly evolved adeno-associated viruses.” J Virol, July 7, 2021, JVI0058721. https://doi.org/10.1128/JVI.00587-21.
Gibson, Rebecca A., Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya S. Kishnani. “Characterization of liver GSD IX γ2 pathophysiology in a novel Phkg2-/- mouse model.” In Mol Genet Metab, 133:269–76, 2021. https://doi.org/10.1016/j.ymgme.2021.05.008.
Mitchell-Dick, Aaron, and Aravind Asokan. “AAV-CNS matters turn from gray to white.” Mol Ther 29, no. 5 (May 5, 2021): 1659–60. https://doi.org/10.1016/j.ymthe.2021.04.012.
Hunanyan, Arsen S., Boris Kantor, Ram S. Puranam, Courtney Elliott, Angela McCall, Justin Dhindsa, Promila Pagadala, et al. “Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.” Hum Gene Ther 32, no. 7–8 (April 2021): 405–19. https://doi.org/10.1089/hum.2020.191.
Saha, Krishanu, Erik J. Sontheimer, P. J. Brooks, Melinda R. Dwinell, Charles A. Gersbach, David R. Liu, Stephen A. Murray, et al. “The NIH Somatic Cell Genome Editing program.” Nature 592, no. 7853 (April 2021): 195–204. https://doi.org/10.1038/s41586-021-03191-1.
Meganck, Rita M., Jiacheng Liu, Andrew E. Hale, Katherine E. Simon, Marco M. Fanous, Heather A. Vincent, Jeremy E. Wilusz, Nathaniel J. Moorman, William F. Marzluff, and Aravind Asokan. “Engineering highly efficient backsplicing and translation of synthetic circRNAs.” Mol Ther Nucleic Acids 23 (March 5, 2021): 821–34. https://doi.org/10.1016/j.omtn.2021.01.003.
Cannon, Paula, Aravind Asokan, Agnieszka Czechowicz, Paula Hammond, Donald B. Kohn, Andre Lieber, Punam Malik, et al. “Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development.” Hum Gene Ther 32, no. 1–2 (January 2021): 31–42. https://doi.org/10.1089/hum.2020.263.
Kwon, Jennifer B., Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, and Charles A. Gersbach. “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 19 (December 11, 2020): 320–29. https://doi.org/10.1016/j.omtm.2020.09.016.
Kang, Martin H., Jiabiao Hu, Richard E. Pratt, Conrad P. Hodgkinson, Aravind Asokan, and Victor J. Dzau. “Optimizing delivery for efficient cardiac reprogramming.” Biochem Biophys Res Commun 533, no. 1 (November 26, 2020): 9–16. https://doi.org/10.1016/j.bbrc.2020.08.104.
Madigan, Victoria J., Garrett E. Berry, Tyne O. Tyson, Dasean Nardone-White, Jonathan Ark, Zachary C. Elmore, Giridhar Murlidharan, Heather A. Vincent, and Aravind Asokan. “The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.” J Virol 94, no. 21 (October 14, 2020). https://doi.org/10.1128/JVI.01604-20.