微小RNA的治疗进展：临床前与临床最新情况

Chakraborty Chiranjib, Sharma Ashish Ranjan, Sharma Garima, Lee Sang-Soo

Department of Biotechnology, School of Life Science and Biotechnology, Adamas University, Barasat-Barrackpore Rd, Kolkata, West Bengal 700126, India.

Institute for Skeletal Aging & Orthopedic Surgery, Hallym University-Chuncheon Sacred Heart Hospital, Chuncheon, Gangwon-Do 24252, Republic of Korea.

J Adv Res. 2020 Aug 29;28:127-138. doi: 10.1016/j.jare.2020.08.012. eCollection 2021 Feb.

miRNAs, a class of small endogenous RNAs, are one of the essential biopharmaceuticals which are in commercial spans as next-generation medicine in recent times. A snapshot of the current scenario regarding the miRNAs as biopharmaceuticals have been discussed. In this work, biopharmaceutical companies working with miRNAs and the current status of preclinical/clinical trials about miRNA therapeutics have been reviewed. Finally, recent updates on the absorption, distribution, metabolism, and excretion (ADME), as well as a delivery system of miRNAs, have been illustrated.

微小RNA（miRNA）是一类小的内源性RNA，是近年来作为下一代药物进入商业领域的重要生物制药之一。本文讨论了有关miRNA作为生物制药的当前情况概述。在这项工作中，对致力于miRNA的生物制药公司以及miRNA疗法的临床前/临床试验现状进行了综述。最后，阐述了miRNA在吸收、分布、代谢和排泄（ADME）方面的最新进展以及miRNA的递送系统。