靶向 RNA 的合成寡核苷酸：临床成功带来新的挑战。
Targeting RNA with synthetic oligonucleotides: Clinical success invites new challenges.
机构信息
The Departments of Pharmacology and Biochemistry, UT Southwestern Medical Center, 6001 Forest Park Road, Dallas, TX 75390-9041, USA.
The Departments of Pharmacology and Biochemistry, UT Southwestern Medical Center, 6001 Forest Park Road, Dallas, TX 75390-9041, USA.
出版信息
Cell Chem Biol. 2024 Jan 18;31(1):125-138. doi: 10.1016/j.chembiol.2023.09.005. Epub 2023 Oct 6.
Abstract
Synthetic antisense oligonucleotides (ASOs) and duplex RNAs (dsRNAs) are an increasingly successful strategy for drug development. After a slow start, the pace of success has accelerated since the approval of Spinraza (nusinersen) in 2016 with several drug approvals. These accomplishments have been achieved even though oligonucleotides are large, negatively charged, and have little resemblance to traditional small-molecule drugs-a remarkable achievement of basic and applied science. The goal of this review is to summarize the foundation underlying recent progress and describe ongoing research programs that may increase the scope and impact of oligonucleotide therapeutics.
摘要
合成反义寡核苷酸 (ASO) 和双链 RNA (dsRNA) 是一种日益成功的药物开发策略。自 2016 年批准Spinraza（nusinersen）以来，尽管寡核苷酸体积大、带负电荷且与传统小分子药物几乎没有相似之处，但已经取得了几项药物批准，这一成功速度加快。这是基础科学和应用科学的一项非凡成就。本文综述的目的是总结近期进展的基础，并描述可能扩大寡核苷酸治疗学范围和影响的正在进行的研究计划。
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