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人类神经瘤抗原Ma2 (PNMA2) 形成二元体体,可被设计为mRNA输送

Victoria Madigan^1,2,3,4,5, Yugang Zhang^1,2,3,4,5, Rumya Raghavan^1,2,3,4,5

¹Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142.

Proceedings of the National Academy of Sciences of the United States of America

|March 4, 2024

概括

人体细胞分泌的是神经瘤抗原Ma (PNMA) 蛋白质,这些蛋白质可以形成囊. 设计的PNMA2颗粒有效地包装并将RNA传递到细胞中,显示出核酸治疗的潜力.

科学领域:

遗传学是一个遗传学.
分子生物学分子生物学
生物化学生物化学

背景情况:

人体内源的逆元件为宿主基因组贡献了基因,包括编码复原病毒像蛋白质的基因.
瘤性马抗原 (PNMA) 蛋白质家族具有类似的囊体域,但它们的组装,细胞间贩运和货物封装能力在很大程度上是未知的.

研究的目的:

研究人类PNMA蛋白质的分泌,组合和核酸包装潜力.
为有效的RNA封装设计PNMA2,并评估其作为核酸输送载体的实用性.

主要方法:

对人类PNMA蛋白质分泌的系统研究.
确定PNMA2囊形成和核酸结合能力.
电子显微镜 (cryo-EM) 结构分析PNMA2.2.
用于RNA包装的PNMA2 (ePNMA2) 的设计和工程.
在体外mRNA包装试验和哺乳动物细胞输送实验.

主要成果:

几种人类PNMA蛋白质是由细胞分泌的.
PNMA2有效地形成二元体体,但不会自然封装核酸.
确定了PNMA2的冷EM结构.
设计的PNMA2 (ePNMA2) 颗粒成功地被设计成包装mRNA.
ePNMA2成功地将mRNA输入哺乳动物细胞系.

结论:

人类PNMA蛋白质被分泌出来,并且能够形成囊.
经过工程设计的PNMA2代表了针对性核酸输送的新平台.
内源性囊系统为开发基于核酸的新疗法提供了潜力.

关键词:

在PNMA中,PNMA是PNMA.这就是VLP的VLP.卡普西德 (Capsid) 是一种体.基因治疗的基因疗法提供mRNA的交付.

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