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METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC PROGENITOR CELLS AND USES OF THE MODIFIED CELLS

机译:造血祖细胞的遗传修饰方法及修饰细胞的用途

摘要

Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.
机译:描述了与基因治疗有关的组合物和方法,特别是应用于造血祖细胞(HP),转导的细胞及其获得方法,以及使用它们提供修饰的造血细胞在人类受试者中长期植入的方法。本发明尤其涉及用于治疗或预防HIV感染的HP细胞的离体基因治疗。

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