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

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

Abstract 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 con...

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Bibliographic Details
Main Authors: Ramon Santamaria, Maria Ballester, Guillem Garcia-Llorens, Francisco Martinez, Marina Blazquez, Carmen Ribes-Koninckx, Jose V. Castell, Torsten Wuestefeld, Roque Bort
Format: Article
Language:English
Published: Nature Portfolio 2022-02-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-022-06184-w

Internet

https://doi.org/10.1038/s41598-022-06184-w

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