反义寡核苷酸:概念和药物应用

Ariana Araya, David Arias, Karla Coto, María Rebeca López, Joselyn Rivera, Juan José Mora
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引用次数: 0

摘要

反义寡核苷酸是一种机制基于与RNA靶序列结合的药物。为此,它们通过位阻和外显子遗漏来修饰蛋白表达。它的生产包括几个步骤:合成、纯化和冻干。通常,最复杂的过程是合成,因为需要化学反应来添加所需的寡核苷酸碱基。BP1001、intertersen、nusinersen、eteplirsen和golodirsen是一些用于治疗神经退行性和神经肌肉疾病的反义药物。虽然反义寡核苷酸存在脱靶反应,但多项研究正在进行中。以下综述显示了有关工业生产的药物特性和临床应用现状的信息。总之,一些分子已经被批准商业化(intertersen、nusinersen、ataluren、eteplirsen和golodirsen),表明它们在短期和中期有希望治疗由特定遗传因素引起的疾病。
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Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
Antisense oligonucleotides are drugs whose mechanism is based on binding to RNA target sequences. For this purpose, they modify the protein expression through steric hindrance and exon omission. Its production involves several steps: synthesis, purification, and lyophilization. Usually, the most complicated procedure is synthesis due to the chemical reactions necessary to add the required oligonucleotide bases. BP1001, inotersen, nusinersen, eteplirsen, and golodirsen are a few antisense drugs developed for treating neurodegenerative and neuromuscular diseases. Although antisense oligonucleotides present off-target reactions, multiple studies are being performed. The following review shows information regarding the pharmaceutical characteristics for industrial production and the current state of applicability in clinical practice. In conclusion, some molecules have already been approved for commercialization (inotersen, nusinersen, ataluren, eteplirsen, and golodirsen), showing them as promising therapeutic solutions in the short and medium term for disorders developed by specific genetic factors.
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