Advances in oligonucleotide drug delivery
Author:
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Link
https://www.nature.com/articles/s41573-020-0075-7.pdf
Reference302 articles.
1. Kim, J. et al. Patient-customized oligonucleotide therapy for a rare genetic disease. N. Engl. J. Med. 381, 1644–1652 (2019). This is the first study to utilize an oligonucleotide therapy tailored to a single patient.
2. Giorgio, E. et al. Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy. Brain 142, 1905–1920 (2019).
3. Southwell, A. L. et al. In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol. Ther. 22, 2093–2106 (2014).
4. Miller, V. M. et al. Allele-specific silencing of dominant disease genes. Proc. Natl Acad. Sci. USA 100, 7195–7200 (2003).
5. Klein, A. F. et al. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J. Clin. Invest. 129, 4739–4744 (2019).
Cited by 1228 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Targeting heterozygous dominant negative variant of KCNA2 using Gapmer ASO for the treatment of drug-resistant epilepsy;Molecular Therapy - Nucleic Acids;2024-12
2. Development of a multi-level pH-responsive lipid nanoplatform for efficient co-delivery of siRNA and small-molecule drugs in tumor treatment;Chinese Chemical Letters;2024-12
3. Model-based optimization strategy for intensification in the chromatographic purification of oligonucleotides;Journal of Chromatography A;2024-11
4. Anti-SARS-CoV-2 gapmer antisense oligonucleotides targeting the main protease region of viral RNA;Antiviral Research;2024-10
5. Thiol-Specific Linkers for the Synthesis of Oligonucleotide Conjugates via Metal-Free Thiol–Ene Click Reaction;Bioconjugate Chemistry;2024-09-12
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3