Science· Jacob Helm, Elena Buena-Atienza, Clemens M. Sauter, Linus Wiora, Melanie Kraft, Yvonne Schelling, Jakob Admard, Jeannette Hübener-Schmid, Thomas Klockgether, Matthis Synofzik, Luís Pereira de Almeida, Mafalda Raposo, Paola Giunti, Hector Garcia-Moreno, Magda M. Santana, Jennifer Faber, Holger Hengel, Nicolas Casadei, Ludger Schöls, Stefan Hauser·· 16 小时前评分62
基于单核苷酸多态性设计脊髓小脑共济失调3型等位基因特异性 ASO
Single-nucleotide polymorphism–directed design of allele-specific antisense oligonucleotides for spinocerebellar ataxia type 3
导读
这项研究以单核苷酸多态性为指导，设计用于脊髓小脑共济失调3型的等位基因特异性 ASO。文章发表于《Science Translational Medicine》Volume 18, Issue 872（October 2026），DOI 为 10.1126/scitranslmed.adz2140。当前材料仅提供题名与卷期信息，未披露实验设计、数据或结论。
来源：Science