한빛사논문
- 조회471
Siyoung A. Lima,1, Alysia Coxa,1, Madelynn Tunga, Eun Ji Chunga,b,c,d,*
aDepartment of Biomedical Engineering, University of Southern California, Los Angeles, CA, USA
bMork Family Department of Chemical Engineering and Materials Science, University of Southern California, Los Angeles, CA, USA
cDepartment of Medicine, Division of Nephrology and Hypertension, University of Southern California, Los Angeles, CA, USA
dDepartment of Surgery, Division of Vascular Surgery and Endovascular Therapy, University of Southern California, Los Angeles, CA, USA
1These authors contributed equally to the preparation of this review.
*Corresponding author
Abstract
The clinical application of nanoparticles (NPs) to deliver RNA for therapy has progressed rapidly since the FDA approval of Onpattro® in 2018 for the treatment of polyneuropathy associated with hereditary transthyretin amyloidosis. The emergency use authorization or approval and widespread global use of two mRNA-NP based vaccines developed by Moderna Therapeutics Inc. and Pfizer-BioNTech in 2021 has highlighted the translatability of NP technology for RNA delivery. Furthermore, in clinical trials, a wide variety of NP formulations have been found to extend the half-life of RNA molecules such as microRNA, small interfering RNA, and messenger RNA, with limited safety issues. In this review, we discuss the NP formulations that are already used in the clinic to deliver therapeutic RNA and highlight examples of RNA-NPs which are currently under evaluation for human use. We also detail NP formulations that failed to progress through clinical trials, in hopes of guiding future successful translation of nanomedicine-based RNA therapeutics into the clinic.
Keywords : Nanoparticles, RNA therapy, Drug delivery, Nanomedicine, Clinical trials, COVID-19
논문정보
- Review Paper
- 2021년 10월 (BRIC 등록일 2022-03-14)
- 국외 연구진
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