David H Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Cambridge, MA 02139, USA.
David H Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Cambridge, MA 02139, USA; Department of Chemistry, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Adv Drug Deliv Rev. 2021 Nov;178:113834. doi: 10.1016/j.addr.2021.113834. Epub 2021 Sep 4.
Recent medical advances have exploited the ability to address a given disease at the underlying level of transcription and translation. These treatment paradigms utilize nucleic acids - including short interfering RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA) - to achieve a desired outcome ranging from gene knockdown to induced expression of a selected target protein. Towards this end, numerous strategies for encapsulation or stabilization of various nucleic acid structures have been developed in order to achieve intracellular delivery. In this review, we discuss several therapeutic applications of nucleic acids directed towards specific diseases and tissues of interest, in particular highlighting recent technologies which have reached late-stage clinical trials and received FDA approval.
最近的医学进展利用了在转录和翻译的基础水平上解决特定疾病的能力。这些治疗模式利用核酸 - 包括短干扰 RNA (siRNA)、微小 RNA (miRNA)、反义寡核苷酸 (ASO) 和信使 RNA (mRNA) - 来实现从基因敲低到选定靶蛋白诱导表达的预期结果。为此，已经开发了许多用于封装或稳定各种核酸结构的策略，以实现细胞内递药。在这篇综述中，我们讨论了针对特定疾病和感兴趣组织的几种核酸治疗应用，特别是强调了最近已进入后期临床试验并获得 FDA 批准的技术。
