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

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...

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Bibliographic Details
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
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Institution: Nanyang Technological University
Language: English

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https://hdl.handle.net/10356/163355

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