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Updated: Jun 26, 2026

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
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脂质纳米颗粒在脑内注射后向大脑输送mRNA

Jan Tuma1,2, Yu-Ju Chen1, Michael G Collins1

  • 1The Department of Cellular and Integrative Physiology, The University of Texas Health Science Center at San Antonio, San Antonio, Texas 78229, United States.

Biochemistry
|September 20, 2023
PubMed
概括

脂质纳米颗粒有效地向成年老鼠大脑输送mRNA治疗药物,准多种细胞类型. 这一突破为治疗神经系统疾病提供了新的希望.

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In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing specific...
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The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
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