Nucleic acid-based therapy for brain tumor: Challenges and strategies

Zixia Zhang, João Conniot, Joana Amorim, Yiliang Jin, Rajendra Prasad, Xiyun Yan, Kelong Fan, João Conde

Research output: Contribution to journalArticlepeer-review

9 Citations (Scopus)
44 Downloads (Pure)


Nucleic acid-based therapy emerges as a powerful weapon for the treatment of tumors thanks to its direct, effective, and lasting therapeutic effect. Encouragingly, continuous nucleic acid-based drugs have been approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Despite the tremendous progress, there are few nucleic acid-based drugs for brain tumors in clinic. The most challenging problems lie on the instability of nucleic acids, difficulty in traversing the biological barriers, and the off-target effect. Herein, nucleic acid-based therapy for brain tumor is summarized considering three aspects: (i) the therapeutic nucleic acids and their applications in clinical trials; (ii) the various administration routes for nucleic acid delivery and the respective advantages and drawbacks. (iii) the strategies and carriers for improving stability and targeting ability of nucleic acid drugs. This review provides thorough knowledge for the rational design of nucleic acid-based drugs against brain tumor.

Original languageEnglish
Pages (from-to)80-92
JournalJournal of Controlled Release
Early online date12 Aug 2022
Publication statusPublished - Oct 2022


  • Nucleic acid-based therapy
  • Brain tumor
  • Blood brain barrier
  • Drug delivery
  • Carriers


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