Review,
Focused ultrasound gene delivery for the treatment of neurological disorders
Contributors
Kofoed, Rikke Hahn
0000-0003-0177-0739
(Corresponding author)
[1]
Aubert, Isabelle
0000-0001-8466-0919
(Corresponding author)
[2]
[3]
[4]
Affiliations
- [1] Aarhus Univ Hosp, Ctr Expt Neurosci CENSE, Dept Neurosurg, Palle Juul Jensens Blvd 165, DK-8200 Aarhus N, Denmark [NORA names: AU Aarhus University; University; Denmark; Europe, EU; Nordic; OECD];
- [2] Sunnybrook Res Inst, Hurvitz Brain Sci Res Program, Biol Sci, Toronto, ON, Canada [NORA names: Canada; America, North; OECD];
- [3] Sunnybrook Res Inst, Hurvitz Brain Sci Res Program, Biol Sci, Toronto, ON, Canada [NORA names: Canada; America, North; OECD];
- [4] Sunnybrook Res Inst, Hurvitz Brain Sci Res Program, Biol Sci, Toronto, ON, Canada [NORA names: Canada; America, North; OECD]
Abstract
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.
