Synthetic Virology & Gene Therapy
Pickar-Oliver, Adrian, Veronica Gough, Joel D. Bohning, Siyan Liu, Jacqueline N. Robinson-Hamm, Heather Daniels, William H. Majoros, Garth Devlin, Aravind Asokan, and Charles A. Gersbach. “Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.” Mol Ther, September 10, 2021. https://doi.org/10.1016/j.ymthe.2021.09.003.
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 95, no. 19 (September 9, 2021): e0058721. https://doi.org/10.1128/JVI.00587-21.
Gemberling, Matthew P., Keith Siklenka, Erica Rodriguez, Katherine R. Tonn-Eisinger, Alejandro Barrera, Fang Liu, Ariel Kantor, et al. “Transgenic mice for in vivo epigenome editing with CRISPR-based systems.” Nat Methods 18, no. 8 (August 2021): 965–74. https://doi.org/10.1038/s41592-021-01207-2.
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.
Das, Anshuman, Patrick Havlik, Jonathan Ark, Mario Mietzsch, Mavis Agbandje-McKenna, and Aravind Asokan. “A CRISPR/Cas9 Genome Wide Screen and Cryo-EM Structure Reveal Receptor Switching by a Synthetic AAV Variant.” In Molecular Therapy, 29:151–52, 2021.
Walton, Eric M., Joshua B. Black, Charles A. Gersbach, and Aravind Asokan. “A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes.” In Molecular Therapy, 29:150–51, 2021.
Gibson, Rebecca, Jeong-A Lim, Su Jin Choi, Leticia Flores, Lani Clinton, Deeksha Bali, Sarah Young, Aravind Asokan, Baodong Sun, and Priya Kishnani. “Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model.” In Molecular Therapy, 29:239–239, 2021.
Havlik, Lawrence Patrick, Daniel K. Oh, Anshuman Das, Ruth M Castellanos Rivera, and Aravind Asokan. “Distinct Functional Roles for AAV Capsid Surface Epitopes Revealed through Structure-Guided Evolution.” In Molecular Therapy, 29:153–153, 2021.
Ark, Jonathan, William Nyberg, Patrick Havlik, Angela To, Justin Eyquem, and Aravind Asokan. “Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations.” In Molecular Therapy, 29:32–32, 2021.