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在二维和三维hiPSC衍生神经模型中,通过增强的基于介质的交付Splice调节的反意义寡核化物

Ronald A M Buijsen¹, Linda M van der Graaf¹, Elsa C Kuijper¹

¹Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.

|September 28, 2024

概括

加增强介质 (CEM) 有效地向人类诱导多能干细胞 (hiPSC) 衍生的神经元和大脑器官提供反感性寡核酸 (ASO). 这种方法具有较低的毒性,为神经疾病研究和治疗开发提供了一个有前途的战略.

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
分子治疗学分子治疗学

背景情况:

反意义技术对于治疗遗传性脑疾病至关重要,许多ASO疗法正在开发中.
人类诱导的多能干细胞 (hiPSCs) 对于模拟神经疾病和测试疗法非常有价值.
向hiPSC衍生的神经元模型提供反感性寡核酸 (ASO) 存在重大挑战.

研究的目的:

在人类hiPSC衍生的神经元模型中评估ASO的各种传递方法.
在2D和3D神经培养中确定一种高效和低毒性的ASO传递方法.
为了优化神经系统疾病治疗的临床前ASO策略.

主要方法:

使用2D和3DhiPSC衍生的神经元模型.
测试了四种ASO输送方法:基于脂质的转染,体操吸收,基于Ca2+增强介质 (CEM) 的输送和电穿孔.
评估了输送效率和细胞毒性.

主要成果:

在2D神经元培养和3D大脑器官中,基于CEM的传递显示出高效率和低毒性.
与星球细胞相比,CEM传递在神经元中稍微更有效.
其他经过测试的方法显示出不同程度的成功和毒性.

结论:

基于CEM的交付是将ASO引入hiPSC衍生神经模型的一个有希望的方法.
这种方法促进了使用ASO疗法的神经系统疾病的研究和治疗.
进一步优化CEM交付具有推进临床前ASO策略的潜力.

关键词:

提供ASO交付服务.在 CEM CEM 中.有机生物有机物

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