Derivation of healthy hepatocyte-like cells from a female patient with ornithine transcarbamylase deficiency through X-inactivation selection

• Main Authors: Santamaria, Ramon, Ballester, Maria, Garcia-Llorens, Guillem, Martinez, Francisco, Blazquez, Marina, Ribes-Koninckx, Carmen, Castell, Jose V., Wuestefeld, Torsten, Bort, Roque
• Other Authors: School of Biological Sciences
• Format: Article
• Language: English
• Published: 2022
• Subjects: Science::Biological sciences; Biological Therapy; Fibroblast
• Online Access: https://hdl.handle.net/10356/163355
• Institution: Nanyang Technological University

Autologous cell replacement therapy for inherited metabolic disorders requires the correction of the underlying genetic mutation in patient's cells. An unexplored alternative for females affected from X-linked diseases is the clonal selection of cells randomly silencing the X-chromosome contain...