Therapeutic siRNA: state of the art

ABSTRACT RNA interference (RNAi) is an ancient biological mechanism used to defend against external invasion. It theoretically can silence any disease-related genes in a sequence-specific manner, making small interfering RNA (siRNA) a promising therapeutic modality. After a two-decade journey from its discovery, two approvals of siRNA therapeutics, ONPATTRO ® (patisiran) and GIVLAARI™ (givosiran), have been achieved by Alnylam Pharmaceuticals. Reviewing the long-term pharmaceutical history of human beings, siRNA therapy currently has set up an extraordinary milestone, as it has already changed and will continue to change the treatment and management of human diseases. It can be administered quarterly, even…

• NN
  National Natural Science Foundation of China

  Awards: 31871003, 31901053, 31901053, IRT_15R13, 31871003
• NS
  Natural Science Foundation of Hunan Province

  Awards: 2019JJ50196, 2018JJ1019
• BI
  Beijing Institute of Technology
• BI
  Beijing Institute of Technology Research Fund Program for Young Scholars
• NS
  National Science and Technology Major Project
• FR
  Fundamental Research Funds for the Central Universities

  Award: 3052018065
• PF
  Program for Changjiang Scholars and Innovative Research Team in University

  Award: IRT_15R13