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Updated: May 6, 2026

Direct Induction of Hemogenic Endothelium and Blood by Overexpression of Transcription Factors in Human Pluripotent Stem Cells
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在人类β-thalassaemia的基因治疗后,输血独立性和HMGA2激活

Marina Cavazzana-Calvo1, Emmanuel Payen, Olivier Negre

  • 1Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique-Hôpitaux de Paris, Paris 75015, France.

Nature
|September 17, 2010
PubMed
概括

基因疗法为严重的β-thalassemia提供了一种潜在的治疗方法. 一名患有β (E) /β (O) -thalassemia的患者在lentiviral基因转移后实现了输血独立,显示了持续的治疗益处.

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相关概念视频

Hematopoiesis01:21

Hematopoiesis

The process of blood cell formation is called hematopoiesis. Hematopoiesis starts early during development, on the seventh day of embryogenesis. This phase of hematopoiesis is called the primitive wave, wherein the extraembryonic yolk sac allows the production of erythroid cells and endothelial cells from a common precursor called hemangioblast. The erythroid cells provide oxygen to support the growth of the rapidly dividing embryo. Hemangioblasts later develop into hematopoietic stem cells or...
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