Focused ultrasound gene delivery for the treatment of neurological disorders

Rikke Hahn Kofoed, Isabelle Aubert

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaperReviewResearchpeer-review


The transformative potential of gene therapy has been demonstrated in humans. However, there is an unmet need for non-invasive targeted gene delivery and regulation in the treatment of brain disorders. Transcranial focused ultrasound (FUS) has gained tremendous momentum to address these challenges. FUS non-invasively modulates brain cells and their environment, and is a powerful tool to facilitate gene delivery across the blood–brain barrier (BBB) with millimeter precision and promptly regulate transgene expression. This review highlights technical aspects of FUS-mediated gene therapies for the central nervous system (CNS) and lessons learned from discoveries in other organs. Understanding the possibilities and remaining obstacles of FUS-mediated gene therapy will be necessary to harness remarkable technologies and create life-changing treatments for neurological disorders.
Original languageEnglish
JournalTrends in Molecular Medicine
Pages (from-to)263-277
Number of pages15
Publication statusPublished - Mar 2024


  • blood–brain barrier
  • focused ultrasound
  • gene therapy
  • neurological disorders
  • Gene Transfer Techniques
  • Genetic Therapy
  • Humans
  • Blood-Brain Barrier/metabolism
  • Brain Diseases
  • Brain/metabolism
  • Drug Delivery Systems


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