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本文引用的文献

1
Glis2 is an early effector of polycystin signaling and a target for therapy in polycystic kidney disease.Glis2 是多囊肾病中 polycystin 信号的早期效应物，也是治疗的靶点。
Nat Commun. 2024 May 1;15(1):3698. doi: 10.1038/s41467-024-48025-6.
2
Eplontersen: First Approval.依洛硫酸酯酶纳：美国首次批准
Drugs. 2024 Apr;84(4):473-478. doi: 10.1007/s40265-024-02008-5.
3
Personalized antisense oligonucleotides 'for free, for life' - the n-Lorem Foundation.“免费且终身提供”的个性化反义寡核苷酸——n-Lorem基金会。
Nat Med. 2023 Jun;29(6):1302-1303. doi: 10.1038/s41591-023-02335-2.
4
A Potential Therapy Using Antisense Oligonucleotides to Treat Autosomal Recessive Polycystic Kidney Disease.一种使用反义寡核苷酸治疗常染色体隐性多囊肾病的潜在疗法。
J Clin Med. 2023 Feb 10;12(4):1428. doi: 10.3390/jcm12041428.
5
Antisense technology: an overview and prospectus.反义技术：概述与展望。
Nat Rev Drug Discov. 2021 Jun;20(6):427-453. doi: 10.1038/s41573-021-00162-z. Epub 2021 Mar 24.
6
Antisense drug discovery and development technology considered in a pharmacological context.从药理学角度考虑的反义药物发现和开发技术。
Biochem Pharmacol. 2021 Jul;189:114196. doi: 10.1016/j.bcp.2020.114196. Epub 2020 Aug 13.
7
Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5.开发一种针对 X 连锁 Alport 综合征的外显子跳跃治疗方法，该方法针对 COL4A5 中的截断变异。
Nat Commun. 2020 Jun 2;11(1):2777. doi: 10.1038/s41467-020-16605-x.
8
Characterization of the Activity and Distribution of a 2'-O-Methoxyethyl-Modified Antisense Oligonucleotide in Models of Acute and Chronic Kidney Disease. characterization of the activity and distribution of a 2'-o-methoxyethyl-modified antisense oligonucleotide in models of acute and chronic kidney disease
Nucleic Acid Ther. 2018 Oct;28(5):297-306. doi: 10.1089/nat.2018.0723. Epub 2018 Aug 22.
9
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.依洛瑟那治疗遗传性转甲状腺素蛋白淀粉样变性病患者。
N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.
10
Comparative Renal Toxicopathology of Antisense Oligonucleotides.反义寡核苷酸的比较肾脏毒理病理学
Nucleic Acid Ther. 2016 Aug;26(4):199-209. doi: 10.1089/nat.2015.0598. Epub 2016 Mar 16.

Treating the Untreatable: Antisense Oligonucleotides as an Individualized Therapy for Rare Genetic Kidney Diseases.

作者信息

Tekendo-Ngongang Cedrik, Gleeson Joseph G, Mignon Laurence

机构信息

n-Lorem Foundation, San Diego, California.

Rady Children's Institute for Genomic Medicine, San Diego, California.

出版信息

J Am Soc Nephrol. 2024 Dec 1;35(12):1774-1777. doi: 10.1681/ASN.0000000532. Epub 2024 Sep 27.

DOI:10.1681/ASN.0000000532
PMID:39331470
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11617478/
Abstract
摘要