关键词: SELEX aptamers nucleic acid therapeutics

Mesh : Aptamers, Nucleotide / therapeutic use Humans Animals SELEX Aptamer Technique / methods

来  源:   DOI:10.3390/ijms25126742   PDF(Pubmed)

Abstract:
Affinity reagents, or target-binding molecules, are quite versatile and are major workhorses in molecular biology and medicine. Antibodies are the most famous and frequently used type and they have been used for a wide range of applications, including laboratory techniques, diagnostics, and therapeutics. However, antibodies are not the only available affinity reagents and they do have significant drawbacks, including laborious and costly production. Aptamers are one potential alternative that have a variety of unique advantages. They are single stranded DNA or RNA molecules that can be selected for binding to many targets including proteins, carbohydrates, and small molecules-for which antibodies typically have low affinity. There are also a variety of cost-effective methods for producing and modifying nucleic acids in vitro without cells, whereas antibodies typically require cells or even whole animals. While there are also significant drawbacks to using aptamers in therapeutic applications, including low in vivo stability, aptamers have had success in clinical trials for treating a variety of diseases and two aptamer-based drugs have gained FDA approval. Aptamer development is still ongoing, which could lead to additional applications of aptamer therapeutics, including antitoxins, and combinatorial approaches with nanoparticles and other nucleic acid therapeutics that could improve efficacy.
摘要:
亲和试剂,或靶结合分子,是相当多才多艺,是主要的工作在分子生物学和医学。抗体是最著名和经常使用的类型,它们已被用于广泛的应用。包括实验室技术,诊断,和治疗学。然而,抗体不是唯一可用的亲和试剂,它们确实有明显的缺点,包括费力和昂贵的生产。适体是一种具有多种独特优势的潜在替代品。它们是单链DNA或RNA分子,可以选择与许多靶标(包括蛋白质)结合。碳水化合物,和小分子-抗体通常对其具有低亲和力。还有多种经济有效的方法,用于在没有细胞的情况下在体外产生和修饰核酸,而抗体通常需要细胞甚至整个动物。虽然在治疗应用中使用适体也有明显的缺点,包括低体内稳定性,适体已经在用于治疗多种疾病的临床试验中取得了成功,并且两种基于适体的药物已经获得FDA的批准。适体开发仍在进行中,这可能会导致适体疗法的额外应用,包括抗毒素,以及与纳米颗粒和其他核酸疗法的组合方法,可以提高疗效。
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