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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 containing t...
Autores principales: | 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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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8831560/ https://www.ncbi.nlm.nih.gov/pubmed/35145162 http://dx.doi.org/10.1038/s41598-022-06184-w |
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