Litcius/Paper detail

Personalized Gene Editing to Treat an Inborn Error of Metabolism

Andrea Gropman, Alexis C. Komor

2025New England Journal of Medicine12 citationsDOI

Topics & Concepts

Inborn error of metabolismGenome editingComputer scienceComputational biologyBioinformaticsGeneGeneticsMedicineBiologyCRISPREndocrinologyPluripotent Stem Cells ResearchCRISPR and Genetic EngineeringMetabolism and Genetic Disorders
Personalized Gene Editing to Treat an Inborn Error of Metabolism | Litcius