Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage

Gene Ther. 2023 Feb;30(1-2):1-7. doi: 10.1038/s41434-021-00304-3. Epub 2021 Nov 10.


Intracerebral hemorrhage (ICH) is a life-threatening condition with a high mortality rate. For survivors, quality of life is determined by primary and secondary phases of injury. The prospects for injury repair and recovery after ICH are highly dependent on the extent of secondary injury. Currently, no effective treatments are available to prevent secondary injury or its long-term effects. One promising strategy that has recently garnered attention is gene therapy, in particular, small interfering RNAs (siRNA), which silence specific genes responsible for destructive effects after hemorrhage. Gene therapy as a potential treatment for ICH is being actively researched in animal studies. However, there are many barriers to the systemic delivery of siRNA-based therapy, as the use of naked siRNA has limitations. Recently, the Food and Drug Administration approved two siRNA-based therapies, and several are undergoing Phase 3 clinical trials. In this review, we describe the advancements in siRNA-based gene therapy for ICH and also summarize its advantages and disadvantages.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cerebral Hemorrhage* / complications
  • Cerebral Hemorrhage* / genetics
  • Cerebral Hemorrhage* / therapy
  • Quality of Life*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use
  • Treatment Outcome
  • United States


  • RNA, Small Interfering