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...

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Detalles Bibliográficos
Autores principales: Santamaria, Ramon, Ballester, Maria, Garcia-Llorens, Guillem, Martinez, Francisco, Blazquez, Marina, Ribes-Koninckx, Carmen, Castell, Jose V., Wuestefeld, Torsten, Bort, Roque
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
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

Internet

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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