Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement

Gene Therapy, Published online: 20 November 2023; doi:10.1038/s41434-023-00428-8Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement

Source: Gene Therapy - November 20, 2023 Category: Genetics & Stem Cells Authors: A. Mateu-Bosch E. Segur-Bailach J. Garc ía-Villoria S. Gea-Sorl í I. Ruiz J. del Rey J. Camps M. Guitart-Mampel G. Garrabou F. Tort A. Ribes C. Fillat Source Type: research