Progress of nucleic acid drugs and non-viral carriers in gene therapy

oleh: LIU Jian

Format: Article
Diterbitkan: Institute of Basic Medical Sciences and Peking Union Medical College Hospital, Chinese Academy of Medical Sciences / Peking Union Medical College. 2022-01-01

Deskripsi

Gene therapy based on nucleic acid drugs is the ultimate treatment technology for gene abnormality related diseases. However, nucleic acid drugs need the help of carriers to enter target cells and reach specific intracellular locations, therefore, the development of safe and efficient delivery systems for nucleic acid drugs is one of the fundamental technologies for gene therapy. Compared with viral vectors, non-viral vectors have higher safety, but are still facing the challenge of relative low transfection efficiency. With the advances of nanotechnology, the efficiency of non-viral carriers has been gained significantly improvements. In this paper, some representative nucleic acid-based drugs and vaccines are introduced, and the progresses of non-viral delivery carriers for uses in gene therapy have been reviewed.