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From design to cellular processing: Insights into sequencing of vectorized therapeutic small RNAs.

作者信息

Fiszer Agnieszka, Kozlowska Emilia, Jazurek-Ciesiolka Magdalena

机构信息

Department of Medical Biotechnology, Institute of Bioorganic Chemistry, Polish Academy of Sciences, Noskowskiego 12/14, 61-704 Poznan, Poland.

出版信息

Mol Ther Nucleic Acids. 2024 Jul 31;35(3):102277. doi: 10.1016/j.omtn.2024.102277. eCollection 2024 Sep 10.

DOI:10.1016/j.omtn.2024.102277
PMID:39184191
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11342168/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a276/11342168/6388ecbb7085/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a276/11342168/6388ecbb7085/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a276/11342168/6388ecbb7085/gr1.jpg

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本文引用的文献

1
Sequencing-guided design of genetically encoded small RNAs targeting CAG repeats for selective inhibition of mutant .针对CAG重复序列的基因编码小RNA的测序引导设计，用于选择性抑制突变体 。
Mol Ther Nucleic Acids. 2024 Apr 29;35(2):102206. doi: 10.1016/j.omtn.2024.102206. eCollection 2024 Jun 11.
2
A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease.一种靶向CAG重复序列的人工微小RNA可降低亨廷顿病YAC128模型中突变型亨廷顿蛋白的水平。
Mol Ther Nucleic Acids. 2022 May 5;28:702-715. doi: 10.1016/j.omtn.2022.04.031. eCollection 2022 Jun 14.
3
The MicroRNA Family Gets Wider: The IsomiRs Classification and Role.
微小RNA家族不断扩大：异源微小RNA的分类及作用
Front Cell Dev Biol. 2021 Jun 9;9:668648. doi: 10.3389/fcell.2021.668648. eCollection 2021.
4
Artificial miRNAs targeting CAG repeat expansion in ORFs cause rapid deadenylation and translation inhibition of mutant transcripts.靶向 ORF 中 CAG 重复扩展的人工 miRNA 导致突变转录本的快速去腺苷酸化和翻译抑制。
Cell Mol Life Sci. 2021 Feb;78(4):1577-1596. doi: 10.1007/s00018-020-03596-7. Epub 2020 Jul 21.
5
AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes.腺相关病毒5型-微小亨廷顿蛋白在患者来源的神经元培养物和星形胶质细胞中降低亨廷顿蛋白信使核糖核酸和蛋白质水平且无脱靶效应
Mol Ther Methods Clin Dev. 2019 Oct 4;15:275-284. doi: 10.1016/j.omtm.2019.09.010. eCollection 2019 Dec 13.
6
Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.针对亨廷顿蛋白的治疗性微小RNA的设计、表征及先导物筛选，用于开发亨廷顿舞蹈症的基因疗法
Mol Ther Nucleic Acids. 2016 Mar 22;5(3):e297. doi: 10.1038/mtna.2016.7.
7
Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway.靶向 CAG 重复序列的双链 RNA 对亨廷顿表达的等位基因选择性抑制作用的机制：通过 RNAi 途径发挥作用。
Nucleic Acids Res. 2012 Dec;40(22):11270-80. doi: 10.1093/nar/gks907. Epub 2012 Oct 4.