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OS-034 - A gene editing approach for chronic hepatitis B: elimination of hepatitis B virus in vivo by targeting cccDNA and integrated viral genomes with a sequence-specific ARCUS nuclease

Autor: Gorsuch, Cassandra, Nemec, Paige, Yu, Mei, Xu, Simin, Han, Dong, Smith, Jeff, Lape, Janel, Van Buuren, Nicholas, Ramirez, Ricardo, Muench, Robert, Holdorf, Meghan, Feierbach, Becket, Falls, Greg, Holt, Jason, Shoop, Wendy, Sevigny, Emma, Karriker, Forrest, Brown, Robert, Joshi, Amod, Goodwin, Tyler, Tam, Ying, Lin, Paulo, Semple, Sean, Leatherbury, Neil, Delaney, William E., Jantz, Derek, Harrison, Emily, Smith, Amy Rhoden

Publikováno v: In Journal of Hepatology June 2023 78 Supplement 1:S37-S37

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Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease.

Autor: Shoop WK; Precision BioSciences, Durham, NC, USA.; Department of Biochemistry and Molecular Biology, University of Miami Miller School of Medicine, Miami, FL, USA., Lape J; Precision BioSciences, Durham, NC, USA., Trum M; Precision BioSciences, Durham, NC, USA., Powell A; Precision BioSciences, Durham, NC, USA., Sevigny E; Precision BioSciences, Durham, NC, USA., Mischler A; Precision BioSciences, Durham, NC, USA., Bacman SR; Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA., Fontanesi F; Department of Biochemistry and Molecular Biology, University of Miami Miller School of Medicine, Miami, FL, USA., Smith J; Precision BioSciences, Durham, NC, USA., Jantz D; Precision BioSciences, Durham, NC, USA., Gorsuch CL; Precision BioSciences, Durham, NC, USA. cassie.gorsuch@precisionbiosciences.com., Moraes CT; Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA. CMoraes@med.miami.edu.

Publikováno v: Nature metabolism [Nat Metab] 2023 Dec; Vol. 5 (12), pp. 2169-2183. Date of Electronic Publication: 2023 Nov 30.