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AAV-RNAi constructs promote DRG toxicity.

作者信息

Xie Jun, Tai Phillip W L

机构信息

Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Department of Microbiology, UMass Chan Medical School, Worcester, MA, USA; Department of Genetic and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.

Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA; Department of Microbiology, UMass Chan Medical School, Worcester, MA, USA; Department of Genetic and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.

出版信息

Mol Ther. 2025 Jan 8;33(1):21-22. doi: 10.1016/j.ymthe.2024.12.027. Epub 2024 Dec 24.

DOI:10.1016/j.ymthe.2024.12.027
PMID:39719700
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11764781/
Abstract
摘要

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本文引用的文献

1
Dorsal root ganglion toxicity after AAV intra-CSF delivery of a RNAi expression construct into non-human primates and mice.在非人灵长类动物和小鼠的脑脊液中注射携带RNAi表达构建体的腺相关病毒（AAV）后出现的背根神经节毒性。
Mol Ther. 2025 Jan 8;33(1):215-234. doi: 10.1016/j.ymthe.2024.11.029. Epub 2024 Nov 19.
2
Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species.循环神经丝轻链作为非临床毒理学物种中AAV诱导的背根神经节毒性的一种有前景的生物标志物。
Mol Ther Methods Clin Dev. 2022 Mar 28;25:264-277. doi: 10.1016/j.omtm.2022.03.017. eCollection 2022 Jun 9.
3
Characterization of AAV-mediated dorsal root ganglionopathy.腺相关病毒介导的背根神经节病变的特征描述。
Mol Ther Methods Clin Dev. 2022 Feb 1;24:342-354. doi: 10.1016/j.omtm.2022.01.013. eCollection 2022 Mar 10.
4
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.微小 RNA 介导的转基因表达抑制可减少灵长类动物 AAV 载体引起的背根神经节毒性。
Sci Transl Med. 2020 Nov 11;12(569). doi: 10.1126/scitranslmed.aba9188.
5
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology.腺相关病毒诱导的背根神经节病理学。
Hum Gene Ther. 2020 Aug;31(15-16):808-818. doi: 10.1089/hum.2020.167. Epub 2020 Jul 31.
6
Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.家族性肌萎缩侧索硬化症中的腺相关病毒和 microRNA 抑制。
N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056.
7
Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin.使用RNA聚合酶II（Pol II）而非RNA聚合酶III（Pol III）启动子表达靶向人类亨廷顿蛋白的人工微小RNA进行安全有效的基因沉默。
Mol Ther Nucleic Acids. 2017 Jun 16;7:324-334. doi: 10.1016/j.omtn.2017.04.011. Epub 2017 Apr 14.
8
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.人工微小RNA减轻短发夹RNA介导的脑毒性：对RNA干扰治疗开发的启示
Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5868-73. doi: 10.1073/pnas.0801775105. Epub 2008 Apr 8.
9
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.细胞微小RNA/短发夹RNA通路过度饱和导致小鼠死亡。
Nature. 2006 May 25;441(7092):537-41. doi: 10.1038/nature04791.