RNA interference therapy for glioblastoma

Expert Opin Biol Ther. 2010 Jun;10(6):927-36. doi: 10.1517/14712598.2010.481667.


Importance of the field: Despite numerous advances made during the last decade in brain tumor therapy, the prognosis of glioblastoma has not improved and these tumors inevitably recur with no effective treatment. Thus, any new therapeutic strategy to target this most malignant tumor will be of significant benefit. RNAi is a powerful gene silencing method that might be used in combination with other agents to improve the efficacy of glioblastoma treatment.

Areas covered in this review: Recent progress and challenges of pre-clinical and clinical research of RNAi therapy for glioblastoma. The review covers literature from 2003 to 2009.

What the reader will gain: The principle of RNA interference therapy, three categories of RNAi triggers, different RNAi delivery system and pre-clinical and clinical studies that are currently underway to evaluate the validity of RNAi as a potential therapeutic strategy against glioblastoma are discussed.

Take home message: RNA inference therapy combined with other therapeutics may offer therapeutic potential for glioblastoma multiforme. Further studies are required to develop more efficient and specific delivery systems, select suitable gene targets, optimize treatment dose and administration schedule, evaluate the efficacy of combination treatment strategies, establish a validated clinical response measure system etc.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Brain Neoplasms / therapy*
  • Clinical Trials as Topic
  • Drug Delivery Systems
  • Glioblastoma / therapy*
  • Humans
  • RNA Interference*
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / adverse effects
  • RNA, Small Interfering / therapeutic use*


  • RNA, Small Interfering