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开发人类iPSC衍生的低免疫原体原生细胞作为用于髓疾病的现成细胞疗法

Lizhao Feng¹, Jianfei Chao¹, Peng Ye¹

¹Department of Neurodegenerative Diseases, Beckman Research Institute of City of Hope, 1500 E. Duarte Rd., Duarte, CA, 91010, USA.

Advanced science (Weinheim, Baden-Wurttemberg, Germany)

|June 5, 2023

概括

低免疫性干细胞为卡纳万病提供了潜在的"现成"疗法. 这些细胞在移植到小鼠时,纠正了主要的病理特征并恢复了运动功能,显示出治疗脱髓化疾病的前景.

科学领域:

神经科学是一个神经科学.
干细胞生物学干细胞生物学
遗传学遗传学是一个

背景情况:

像卡纳万病 (CD) 这样的脱氨酸性疾病是常见的,使人衰弱的神经系统疾病.
疾病是由ASPA基因突变引起的,导致有毒的N-乙-l-酸盐 (NAA) 积累和大脑损伤.
目前的治疗方法有限,需要新的治疗方法.

研究的目的:

开发和评估由低免疫性诱导的多能干细胞 (iPSC) 衍生的寡干细胞原生细胞 (OPC) 作为对卡纳万病的"现成"疗法.
在临床前模型中评估这些工程OPC的安全性,有效性和免疫性.

主要方法:

使用CRISPR/Cas9编辑人类白细胞抗原 (HLA) 基因生成的低免疫性iPSCs.
将IPSC分化为OPC,并将其植入CD模型小鼠的大脑中 (nur7).
评估了OPC的分布,分化,基因表达 (ASPA),NAA水平,病理和运动功能.

主要成果:

植入的OPC分布广泛,成熟为寡细胞,并表达了功能性人类ASPA.
移植的小鼠显示NAA水平降低,并挽救了髓化和真空化缺陷.
在接受治疗的小鼠中,运动功能缺陷显著改善.
低免疫性OPCs在体外和体内表现出较低的免疫性.

结论:

低免疫性iPSC衍生的OPCs代表了对卡纳万病的有前途的普遍供体细胞疗法.
这种方法有可能用于治疗各种脱髓化疾病,包括多发性硬化症等自身免疫性疾病.

关键词:

卡纳万病就是卡纳万病.全源细胞疗法全源细胞疗法脱髓化疾病是一种脱髓化疾病.这些是iPSCs.现成的细胞疗法寡基细胞原生细胞 (OPC)这是一个普遍的捐赠者.

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