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The expanding application of antisense oligonucleotides to neurodegenerative diseases.

作者信息

Sumner Charlotte J, Miller Timothy M

机构信息

Departments of Neurology, Neuroscience, and Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore Maryland, USA.

Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA.

出版信息

J Clin Invest. 2024 Oct 1;134(19):e186116. doi: 10.1172/JCI186116.

DOI:10.1172/JCI186116
PMID:39352381
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11444189/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2712/11444189/db813a09b964/jci-134-186116-g024.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2712/11444189/db813a09b964/jci-134-186116-g024.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2712/11444189/db813a09b964/jci-134-186116-g024.jpg

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2
Tau-targeting antisense oligonucleotide MAPT in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial.靶向 Tau 的反义寡核苷酸 MAPT 治疗轻度阿尔茨海默病的 1b 期、随机、安慰剂对照试验。
Nat Med. 2023 Jun;29(6):1437-1447. doi: 10.1038/s41591-023-02326-3. Epub 2023 Apr 24.
3
Trial of Antisense Oligonucleotide Tofersen for ALS.针对肌萎缩侧索硬化症的反义寡核苷酸药物 Tofersen 的试验。
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ACS Pharmacol Transl Sci. 2025 Feb 5;8(2):592-601. doi: 10.1021/acsptsci.4c00698. eCollection 2025 Feb 14.
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mRNA Degradation as a Therapeutic Solution for Mucopolysaccharidosis Type IIIC: Use of Antisense Oligonucleotides to Promote Downregulation of Heparan Sulfate Synthesis.mRNA降解作为IIIC型黏多糖贮积症的一种治疗方案:使用反义寡核苷酸促进硫酸乙酰肝素合成的下调
Int J Mol Sci. 2025 Feb 1;26(3):1273. doi: 10.3390/ijms26031273.
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Nat Rev Dis Primers. 2022 Aug 4;8(1):52. doi: 10.1038/s41572-022-00380-8.
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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis.反义寡核苷酸沉默 FUS 表达作为肌萎缩侧索硬化症的治疗方法。
Nat Med. 2022 Jan;28(1):104-116. doi: 10.1038/s41591-021-01615-z. Epub 2022 Jan 24.
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