吗啉
杜氏肌营养不良
磷酸二酯键
寡核苷酸
肌营养不良
化学
单体
共轭体系
DNA
核糖核酸
立体化学
生物化学
遗传学
生物
基因
有机化学
斑马鱼
聚合物
作者
Shahab Edalatian Zakeri,Sai Pallavi Pradeep,Vishal Kasina,Ankit P. Laddha,José E. Manautou,Raman Bahal
标识
DOI:10.1016/j.tips.2022.04.009
摘要
Casimersen is a single-stranded, antisense phosphorodiamidate morpholino oligonucleotide where six-membered morpholino rings substitute the ribofuranose rings of the natural DNA and RNA backbone. The morpholino is conjugated to the following monomer via a charge-neutral phosphorodiamidate instead of the phosphodiester linkage containing a negative charge. Phosphorodiamidate morpholino oligonucleotides are resistant to enzymatic degradation and their lack of charge facilitates cellular penetration.
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