RNA 疗法。
RNA therapeutics.
机构信息
Rosalind Franklin University of Medicine and Science, Chicago, Illinois 60064, USA.
Cold Spring Harbor Laboratory, Cold Spring Harbor, New York 11724, USA
出版信息
RNA. 2023 Apr;29(4):393-395. doi: 10.1261/rna.079626.123.
"RNA therapeutics" refers to a disease treatment or drug that utilizes RNA as a component. In this context, RNA may be the direct target of a small-molecule drug or RNA itself may be the drug, designed to bind to a protein, or to mimic or target another RNA. RNA has gained attention in the drug-development world, as recent clinical successes and breakthrough technologies have revolutionized the drug-like qualities of the molecule or its usefulness as a drug target. In this special issue of , we gathered expert perspectives on the past, present, and future of the field, to serve as a primer and also a challenge to the broad scientific community to incorporate RNA into their experimental design and problem-solving process, and to imagine and realize the potential of RNA as a therapeutic drug or target.
“RNA 疗法”是指利用 RNA 作为成分的疾病治疗或药物。在这种情况下，RNA 可以是小分子药物的直接靶标，也可以是药物本身，旨在与蛋白质结合，或模拟或针对另一种 RNA。由于最近的临床成功和突破性技术改变了该分子的类药性或作为药物靶点的用途，RNA 在药物开发领域引起了关注。在本期 特刊中，我们汇集了该领域过去、现在和未来的专家观点，旨在作为入门读物，同时也是对广大科学界的挑战，即把 RNA 纳入他们的实验设计和解决问题的过程，并想象和实现 RNA 作为治疗药物或靶标的潜力。
Zotero 插件