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...
| Main Authors: | , , , , , , , , |
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| 其他作者: | |
| 格式: | Journal Article |
| 语言: | English |
| 出版: |
2022
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| 主题: | |
| 在线阅读: | https://hdl.handle.net/10356/163355 |