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Updated: Jun 13, 2026

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修改自放大RNA在哺乳动物大脑中调解了强大的和长时间的基因表达

Jennifer Freire1,2, Joshua E McGee1, Dana Shaw1,3

  • 1Department of Biomedical Engineering, Boston University, Boston, MA, USA.

bioRxiv : the preprint server for biology
|November 24, 2025
PubMed
概括

脂质纳米颗粒中的修改自放大RNA (saRNA) 在脑细胞中显示出强大的长期蛋白质表达,性能优于mRNA. 这种非病毒性方法为基因疗法应用提供了潜在的潜力.

关键词:
免疫逃逸是一种免疫逃避.星球细胞是星球细胞.中枢神经系统中枢神经系统基因转移是转移基因的过程.修改后的saRNA是什么神经元神经元的神经元

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The structure and stability of mRNA molecules regulates gene expression, as mRNAs are a key step in the pathway from gene to protein. In eukaryotes, the half-life of mRNA varies from a few minutes up to several days. mRNA stability is essential in growth and development. The absence of the proteins regulating its stability, such as tristetraprolin in mice, can cause systemic issues, including bone marrow overgrowth, inflammation, and autoimmunity.
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mRNA Stability and Gene Expression

The structure and stability of mRNA molecules regulates gene expression, as mRNAs are a key step in the pathway from gene to protein. In eukaryotes, the half-life of mRNA varies from a few minutes up to several days. mRNA stability is essential in growth and development. The absence of the proteins regulating its stability, such as tristetraprolin in mice, can cause systemic issues, including bone marrow overgrowth, inflammation, and autoimmunity.
Cis-acting Elements involved in mRNA stability
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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 the pre-miRNA...