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Long non-coding RNAs: From disease code to drug role

Journal

ACTA PHARMACEUTICA SINICA B
Volume 11, Issue 2, Pages 340-354

Publisher

INST MATERIA MEDICA, CHINESE ACAD MEDICAL SCIENCES
DOI: 10.1016/j.apsb.2020.10.001

Keywords

LncRNAs; Targeted drug; Gene therapy; Small molecules; Delivery; ASncmtRNA; Translational medicine; Clinical trials

Funding

  1. Drug Innovation Major Project of China [2018ZX09711001-002-010]
  2. Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences [2016-I2M-3e011]
  3. Beijing Natural Science Foundation [7202138, 7181007]

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Extensive studies have shown that long non-coding RNAs (lncRNAs) play crucial roles in physiological processes and are closely linked to various diseases. Using lncRNAs as biomarkers or intervention targets can provide new perspectives for disease diagnosis and treatment. Research is shifting towards utilizing lncRNAs as pharmacological targets, with a focus on preclinical research and clinical trial progress.
Enormous studies have corroborated that long non-coding RNAs (lncRNAs) extensively participate in crucial physiological processes such as metabolism and immunity, and are closely related to the occurrence and development of tumors, cardiovascular diseases, nervous system disorders, nephropathy, and other diseases. The application of lncRNAs as biomarkers or intervention targets can provide new insights into the diagnosis and treatment of diseases. This paper has focused on the emerging research into lncRNAs as pharmacological targets and has reviewed the transition of lncRNAs from the role of disease coding to acting as drug candidates, including the current status and progress in preclinical research. Cutting-edge strategies for lncRNA modulation have been summarized, including the sources of lncRNA-related drugs, such as genetic technology and small-molecule compounds, and related delivery methods. The current progress of clinical trials of lncRNA-targeting drugs is also discussed. This information will form a latest updated reference for research and development of lncRNA-based drugs. (C) 2021 Chinese Pharmaceutical Association and Institute of Materia Medica, Chinese Academy of Medical Sciences. Production and hosting by Elsevier B.V.

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