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