[造血干细胞靶向基因疗法的发展]。

Naoya Uchida
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引用次数: 0

摘要

造血干细胞(HSC)靶向基因疗法可治愈各种遗传性血液病,其疗效已在近期的临床试验中得到证实。造血干细胞具有自我更新和造血多能性,因此,修复造血干细胞的病理突变或缺陷,只需一次治疗就能达到终身治愈的目的。自体造血干细胞基因疗法是通过慢病毒基因添加或基因编辑开发出来的,由于不需要匹配的供体,因此是大多数患者的选择。目前的造血干细胞基因疗法是基于体外方法,即采集患者的造血干细胞,在体外对其进行基因修饰,然后自体移植到患者体内。然而,这一过程的复杂性和高昂的治疗费用阻碍了基因疗法的推广。因此,目前正在开发体内造血干细胞基因疗法,通过给药将基因治疗工具直接注入骨髓造血干细胞,而无需进行体外培养。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Development of hematopoietic stem cell-targeted gene therapy].

Hematopoietic stem cell (HSC)-targeted gene therapy is curative for various genetic blood diseases, and its efficacy has been demonstrated in recent clinical trials. HSCs have self-renewal and hematopoietic multipotency; therefore, repairing pathological mutations or defects in HSCs allows for a lifelong cure with a single treatment. Autologous HSC gene therapy has been developed by lentiviral gene addition or gene editing, and is an option for most patients because it does not require a compatible donor. Current HSC gene therapy is based on ex vivo methods, in which patient HSCs are harvested, genetically modified ex vivo, and autologously transplanted into patients. However, the complexity of this process and the high cost of treatment are hindering the spread of gene therapy. Therefore, in vivo HSC gene therapy is being developed to deliver gene therapy tools directly into bone marrow HSCs by administration without ex vivo culture.

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