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Updated: May 7, 2025

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

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一个全合一的基因疗法替代DBAS

Susana Navarro1, Paula Río1

  • 1Division of Hematopoietic Innovative Therapies, CIEMAT, Madrid, Spain; Instituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBERER), Madrid, Spain; Advanced Therapies Unit, IIS-Fundación Jimenez Diaz (IIS-FJD, UAM), 28040 Madrid, Spain.

Cell stem cell
|January 3, 2025
PubMed
概括

钻石-布莱克芬贫血是一种罕见的骨髓衰竭综合征,具有许多遗传原因. 临床前发现表明,一种通用的lentiviral基因疗法方法可以有效地治疗患者,无论其特定的遗传变异.

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