Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Colin L. Sweeney, Mara Pavel-Dinu, Uimook Choi, Julie Brault, Taylor Liu, Sherry Koontz, Linhong Li, Narda Theobald, Janet S. Lee, Ezekiel Bello, Xiaolin Wu, Ronald J. Meis, Gary A. Dahl, Matthew H. Porteus, Harry L. Malech, Suk See De Ravin
Topics & Concepts
BiologyExonWoodchuck hepatitis virusMolecular biologyNon-homologous end joiningCRISPRInsertionGeneticsGeneMutationDNA repairVirusHepatitis B virusHepadnaviridaeCRISPR and Genetic EngineeringCytomegalovirus and herpesvirus researchVirus-based gene therapy research