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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
mRNA delivery via non-viral carriers for biomedical applications
Qi Shuai1, Fangtao Zhu2, Mengdan Zhao3
1College of Pharmaceutical Sciences and Collaborative Innovation Center of Yangtze River Delta Region Green Pharmaceuticals, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, China; Women's Hospital, School of Medicine, Zhejiang University and Key Laboratory of Women's Reproductive Health Research of Zhejiang Province, Hangzhou, Zhejiang 310006, China.
Abstract:
As an emerging new class of nucleic acid drugs, messenger RNA (mRNA) has huge potential in immunotherapy, regenerative medicine, vaccine, and gene editing. Comparing with siRNA and pDNA, mRNA is more vulnerable to nucleases in vivo. However, the lack of effective and safe delivery methods impedes the broad application of mRNA-based therapeutics. Up to now, the delivery of mRNA remains largely unexplored, and therefore, is a hot topic in the field of gene therapy. In this review, we will summarize the ongoing challenges in mRNA-based therapeutics and unmet requirements for delivery vehicles in terms of the unique structure of mRNA. We then highlight the advancement in mRNA delivery in both fundamental research and clinical applications. Finally, a prospective will be proposed upon reviewing the current progress in mRNA delivery.
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