TY - JOUR
T1 - The challenge and prospect of mRNA therapeutics landscape
AU - Weng, Yuhua
AU - Li, Chunhui
AU - Yang, Tongren
AU - Hu, Bo
AU - Zhang, Mengjie
AU - Guo, Shuai
AU - Xiao, Haihua
AU - Liang, Xing Jie
AU - Huang, Yuanyu
N1 - Publisher Copyright:
© 2020 Elsevier Inc.
PY - 2020/5/1
Y1 - 2020/5/1
N2 - Messenger RNA (mRNA)-based therapeutics hold the potential to cause a major revolution in the pharmaceutical industry because they can be used for precise and individualized therapy, and enable patients to produce therapeutic proteins in their own bodies without struggling with the comprehensive manufacturing issues associated with recombinant proteins. Compared with the current therapeutics, the production of mRNA is much cost-effective, faster and more flexible because it can be easily produced by in vitro transcription, and the process is independent of mRNA sequence. Moreover, mRNA vaccines allow people to develop personalized medications based on sequencing results and/or personalized conditions rapidly. Along with the great potential from bench to bedside, technical obstacles facing mRNA pharmaceuticals are also obvious. The stability, immunogenicity, translation efficiency, and delivery are all pivotal issues need to be addressed. In the recently published research results, these issues are gradually being overcome by state-of-the-art development technologies. In this review, we describe the structural properties and modification technologies of mRNA, summarize the latest advances in developing mRNA delivery systems, review the preclinical and clinical applications, and put forward our views on the prospect and challenges of developing mRNA into a new class of drug.
AB - Messenger RNA (mRNA)-based therapeutics hold the potential to cause a major revolution in the pharmaceutical industry because they can be used for precise and individualized therapy, and enable patients to produce therapeutic proteins in their own bodies without struggling with the comprehensive manufacturing issues associated with recombinant proteins. Compared with the current therapeutics, the production of mRNA is much cost-effective, faster and more flexible because it can be easily produced by in vitro transcription, and the process is independent of mRNA sequence. Moreover, mRNA vaccines allow people to develop personalized medications based on sequencing results and/or personalized conditions rapidly. Along with the great potential from bench to bedside, technical obstacles facing mRNA pharmaceuticals are also obvious. The stability, immunogenicity, translation efficiency, and delivery are all pivotal issues need to be addressed. In the recently published research results, these issues are gradually being overcome by state-of-the-art development technologies. In this review, we describe the structural properties and modification technologies of mRNA, summarize the latest advances in developing mRNA delivery systems, review the preclinical and clinical applications, and put forward our views on the prospect and challenges of developing mRNA into a new class of drug.
KW - Gene therapy
KW - In vitro transcription
KW - Nucleic acid therapeutics
KW - mRNA delivery
KW - mRNA modification
KW - mRNA therapeutics
UR - http://www.scopus.com/inward/record.url?scp=85081002313&partnerID=8YFLogxK
U2 - 10.1016/j.biotechadv.2020.107534
DO - 10.1016/j.biotechadv.2020.107534
M3 - Review article
C2 - 32088327
AN - SCOPUS:85081002313
SN - 0734-9750
VL - 40
JO - Biotechnology Advances
JF - Biotechnology Advances
M1 - 107534
ER -
