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한국과학기술연구원 (KIST)

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Adv. Drug Deliv. Rev., Volume 201, October 2023, 115073 | https://doi.org/10.1016/j.addr.2023.115073 RNAi therapies: Expanding applications for extrahepatic diseases and overcoming delivery challenges.

Jong Won Lee ^a,b,1, Man Kyu Shim ^b,1, Hyosuk Kim ^b, Hochung Jang ^b,c, Yuhan Lee ^d, Sun Hwa Kim^a,b

^aKU-KIST Graduate School of Converging Science and Technology, Korea University, Seoul 02841, Republic of Korea

^bMedicinal Materials Research Center, Biomedical Research Division, Korea Institute of Science and Technology (KIST), Seoul 02792, Republic of Korea

^cDivision of Bio-Medical Science and Technology, KIST School, Korea University of Science and Technology, Seoul 02792, Republic of Korea

^dDepartment of Anesthesiology, Perioperative, and Pain Medicine, Center for Accelerated Medical Innovation & Center for Nanomedicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA

¹These authors contributed equally to this work.

Corresponding authors : Yuhan Lee, Sun Hwa Kim

Abstract

The era of RNA medicine has become a reality with the success of messenger RNA (mRNA) vaccines against COVID-19 and the approval of several RNA interference (RNAi) agents in recent years. Particularly, therapeutics based on RNAi offer the promise of targeting intractable and previously undruggable disease genes. Recent advances have focused in developing delivery systems to enhance the poor cellular uptake and insufficient pharmacokinetic properties of RNAi therapeutics and thereby improve its efficacy and safety. However, such approach has been mainly achieved via lipid nanoparticles (LNPs) or chemical conjugation with N-Acetylgalactosamine (GalNAc), thus current RNAi therapy has been limited to liver diseases, most likely to encounter liver-targeting limitations. Hence, there is a huge unmet medical need for intense evolution of RNAi therapeutics delivery systems to target extrahepatic tissues and ultimately extend their indications for treating various intractable diseases. In this review, challenges of delivering RNAi therapeutics to tumors and major organs are discussed, as well as their transition to clinical trials. This review also highlights innovative and promising preclinical RNAi-based delivery platforms for the treatment of extrahepatic diseases.

논문정보

형식Review Paper
게재일2023년 10월 (BRIC 등록일 2023-09-06)
연구진국내(교신)+국외 연구진
분야 바이오·의료융합 > 바이오센싱 및 나노바이오물질

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