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带有MicroRNA137的脂质纳米粒子调节前额叶皮层中的突触蛋白

Michelle C Palumbo¹, Milan Gautam², Alex Sonneborn¹

¹Department of Behavioral Neuroscience, Oregon Health & Science University, Portland, OR 97239, USA.

Molecular therapy : the journal of the American Society of Gene Therapy

|August 30, 2023

概括

研究人员开发了新的脂质纳米颗粒 (LNP) 来直接向大脑输送微RNA137 (miR137),针对与精神分裂症 (SCZ) 风险相关的突触功能障碍. 这一进步显示出治疗中枢神经系统疾病的潜力.

科学领域:

神经科学是一个神经科学.
遗传学是一个遗传学.
纳米技术纳米技术

背景情况:

精神分裂症 (SCZ) 风险与微RNA137 (miR137) 的基因MIR137中的遗传变异有关.
失调的miR137及其标与SCZ相关的突触功能障碍有关.
脂质纳米颗粒 (LNP) 对RNA输送具有前景,但特定于大脑的应用是有限的.

研究的目的:

为了研究工程LNP的潜力,为大脑特定的miR137.7.传递miR137.
评估通过LNP传递的miR137对SCZ相关分子点的治疗能力.

主要方法:

这些LNP被设计为封装和传递miR137和mRNA有效载荷.
在体外研究中使用神经母细胞细胞来确认LNP货物释放和目标抑制.
在小鼠体内研究评估了LNP生物分布,细胞特异性表达 (神经元与质细胞) 以及前额叶皮质 (PFC) 中的蛋白质变化.

主要成果:

LNP成功释放了miR137载荷,并在神经母细胞细胞中抑制了标转录.
在体内,LNP递送的mRNA局部化到小鼠PFC,在神经元中具有优先表达.
定量蛋白质组学揭示,miR137调节了与SCZ相关的谷氨基质突触蛋白网络.

结论:

工程LNP可以向特定的大脑区域输送RNA疗法,包括miR137.
这种LNP技术证明了准SCZ中涉及的分子途径的潜力.
进一步开发特定于大脑的LNP可能会导致治疗中枢神经系统疾病的新方法.

关键词:

大脑传递大脑传递谷氨酸酸盐网络是一种谷氨酸盐网络.脂质纳米颗粒是一种纳米粒子.这是一个微型RNA.神经元神经元的神经元前额叶皮层前额叶皮层.精神分裂症是一种精神分裂症.突触蛋白质是一种突触蛋白质.

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相关概念视频

MicroRNAs

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After...