Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.

Autor: Balakrishnan B; Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA., Yan X; Moderna, Cambridge, MA 02139, USA., McCue MD; Sable Systems International, North Las Vegas, NV 89032, USA., Bellagamba O; Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA., Guo A; Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA., Winkler F; Moderna, Cambridge, MA 02139, USA., Thall J; Moderna, Cambridge, MA 02139, USA., Crawford L; Moderna, Cambridge, MA 02139, USA., Dimen R; Moderna, Cambridge, MA 02139, USA., Chen S; Moderna, Cambridge, MA 02139, USA., McEnaney S; Moderna, Cambridge, MA 02139, USA., Wu Y; Moderna, Cambridge, MA 02139, USA., Zimmer M; Moderna, Cambridge, MA 02139, USA., Sarkis J; Moderna, Cambridge, MA 02139, USA., Martini PGV; Moderna, Cambridge, MA 02139, USA., Finn PF; Moderna, Cambridge, MA 02139, USA., Lai K; Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Jan 17; Vol. 32 (1), pp. 101191. Date of Electronic Publication: 2024 Jan 17 (Print Publication: 2024).