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首页> 外文期刊>Expert opinion on biological therapy >Exploitation of stem cell homing for gene delivery.
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Exploitation of stem cell homing for gene delivery.

机译：利用干细胞归巢进行基因递送。

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摘要

Stem cells have been the focus of numerous investigations to treat diseases as far ranging as diabetes, chronic heart failure and multiple sclerosis over the past decade. The process of stem-cell-based repair of acute injury involves homing and engrafting of the stem cell of interest to the site of injury followed by either differentiation of the stem cell to indigenous end-organ cells or liberation of paracrine factors that lead to preservation and/or optimization of organ function. Recognition of the ability of stem cells to home to sites of acute injury suggests that, if appropriately defined and harnessed, stem cell homing could serve as a means of local drug delivery through the infusion of genetically engineering stem cells that secrete gene products of interest. The authors have recently demonstrated the use of this approach in preclinical studies of acute myocardial function. In addition, the use of engineered cells that home to appropriate niches have been used to correct genetic deficiency states (i.e., severe combined immunodeficiency, diabetes mellitus) in patients with otherwise chronic debilitating diseases. This review focuses on exploiting stem cell homing for gene transfer and on the state of the art and the challenges that face the field.
机译：在过去的十年中，干细胞一直是治疗诸如糖尿病，慢性心力衰竭和多发性硬化症等疾病的众多研究的重点。基于干细胞的急性损伤修复过程涉及将感兴趣的干细胞归巢和移植到损伤部位，然后将干细胞分化为天然终末器官细胞或释放旁分泌因子以保存和/或器官功能的优化。对干细胞适应急性损伤部位的能力的认识表明，如果适当定义和利用干细胞归巢，可以通过输注分泌目的基因产物的基因工程干细胞来作为局部药物递送的手段。作者最近证明了这种方法在急性心肌功能的临床前研究中的使用。另外，已经使用了在适当的壁home处筑巢的工程细胞来纠正患有慢性衰弱性疾病的患者的遗传缺陷状态（即严重的联合免疫缺陷，糖尿病）。这篇综述着重于利用干细胞归巢进行基因转移，并探讨了现有技术和该领域面临的挑战。

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