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Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia.

Autor: Arnaoutova I; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Aratyn-Schaus Y; BEAM Therapeutics, Cambridge, MA, 02142, USA., Zhang L; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Packer MS; BEAM Therapeutics, Cambridge, MA, 02142, USA., Chen HD; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Lee C; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Gautam S; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Gregoire FM; BEAM Therapeutics, Cambridge, MA, 02142, USA., Leboeuf D; BEAM Therapeutics, Cambridge, MA, 02142, USA., Boule S; BEAM Therapeutics, Cambridge, MA, 02142, USA., Fernandez TP; BEAM Therapeutics, Cambridge, MA, 02142, USA., Huang V; BEAM Therapeutics, Cambridge, MA, 02142, USA., Cheng LI; BEAM Therapeutics, Cambridge, MA, 02142, USA., Lung G; BEAM Therapeutics, Cambridge, MA, 02142, USA., Bannister B; BEAM Therapeutics, Cambridge, MA, 02142, USA., Decker J; BEAM Therapeutics, Cambridge, MA, 02142, USA., Leete T; BEAM Therapeutics, Cambridge, MA, 02142, USA., Shuang LS; BEAM Therapeutics, Cambridge, MA, 02142, USA., Bock C; BEAM Therapeutics, Cambridge, MA, 02142, USA., Kothiyal P; BEAM Therapeutics, Cambridge, MA, 02142, USA., Grayson P; BEAM Therapeutics, Cambridge, MA, 02142, USA., Mok KW; BEAM Therapeutics, Cambridge, MA, 02142, USA., Quinn JJ; BEAM Therapeutics, Cambridge, MA, 02142, USA., Young L; BEAM Therapeutics, Cambridge, MA, 02142, USA., Barrera L; BEAM Therapeutics, Cambridge, MA, 02142, USA., Ciaramella G; BEAM Therapeutics, Cambridge, MA, 02142, USA., Mansfield BC; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA., Chou JY; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, 20892, USA. chouja@mail.nih.gov.

Publikováno v: Nature communications [Nat Commun] 2024 Nov 10; Vol. 15 (1), pp. 9729. Date of Electronic Publication: 2024 Nov 10.