A new developing class of gene delivery: messenger RNA-based therapeutics

作者:Meng Zhao; O'Keeffe-Ahern Jonathan; Lyu Jing; Pierucci Luca; Zhou Dezhong*; Wang Wenxin*
来源:Biomaterials Science, 2017, 5(12): 2381-2392.
DOI:10.1039/c7bm00712d

摘要

Gene therapy has long been held as having the potential to become a front line treatment for various genetic disorders. However, the direct delivery of nucleic acids to correct a genetic disorder has numerous limitations owing to the inability of naked nucleic acids (DNA and RNA) to traverse the cell membrane. Recently, messenger RNA (mRNA) based delivery has become a more attractive alternative to DNA due to the relatively easier transfection process, higher efficiency and safety profile. As with all gene therapies, the central challenge that remains is the efficient delivery of nucleic acids intracellularly. This review presents the recent progress in mRNA delivery, focusing on comparing the advantages and limitations of non-viral based delivery vectors.