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The Journal of Clinical Investigation|August 5, 2003
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virusBruno B Bordier, Junko Ohkanda, Ping Liu, et al.
Hepatology (Baltimore, Md.)|September 25, 2020
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic AcidemiaRandy J Chandler, Leah E Venturoni, Jing Liao, et al.
Molecular Metabolism|February 12, 2024
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapySergio Muñoz, Joan Bertolin, Veronica Jimenez, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 8, 2021
Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase ComplexGustavo de Alencastro, Francesco Puzzo, Mara Pavel-Dinu, et al.
Nature Biotechnology|April 8, 2024
Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectorsFabian P Suchy, Daiki Karigane, Yusuke Nakauchi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 12, 2022
Evaluating the state of the science for adeno-associated virus integration: An integrated perspectiveDenise E Sabatino, Frederic D Bushman, Randy J Chandler, et al.
Nature|November 30, 2017
A transfer-RNA-derived small RNA regulates ribosome biogenesisHak Kyun Kim, Gabriele Fuchs, Shengchun Wang, et al.
Nature Communications|June 22, 2023
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathyMarkus Grosch, Laura Schraft, Adrian Chan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 28, 2020
Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round ScreeningKathleen Börner, Eike Kienle, Lin-Ya Huang, et al.
Communications Biology|July 15, 2024
Influence of AAV vector tropism on long-term expression and Fc-γ receptor binding of an antibody targeting SARS-CoV-2Jannik T Wagner, Sandra M Müller-Schmucker, Wenjun Wang, et al.
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