Stereopure ASOs: An unanticipated increase in selectivity for targeting mutant HTT.
作者信息
Gulumkar Vijay N, Dowdy Steven F
机构信息
Department of Cellular & Molecular Medicine, University of California, San Diego, School of Medicine, La Jolla, CA, USA.
出版信息
Mol Ther Nucleic Acids. 2024 Aug 28;35(3):102312. doi: 10.1016/j.omtn.2024.102312. eCollection 2024 Sep 10.
Abstract
摘要
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Endosomal escape of RNA therapeutics: How do we solve this rate-limiting problem?RNA 治疗药物的内体逃逸：我们如何解决这个限速问题？
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Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.通过短暂抑制亨廷顿合成实现亨廷顿病的持续治疗逆转。
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Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.针对亨廷顿病基因中单核苷酸多态性的有效且选择性的反义寡核苷酸/突变亨廷顿蛋白的等位基因特异性沉默。
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The clinical and genetic features of Huntington disease.亨廷顿病的临床和遗传特征。
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Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts.亨廷顿舞蹈病患者来源的成纤维细胞中突变型亨廷顿蛋白等位基因的鉴定及等位基因特异性沉默
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