Ultrasound targeted CNS gene delivery for Parkinson's disease treatment
Abstract
Parkinson's disease (PD) is a potent neurodegenerative disease in which a progressive loss of dopaminergic neurons eventually produces a loss of movement control and other symptoms. To date, in addition to pharmacological, non-pharmacological, and neurosurgical therapies, gene delivery has emerged as a potential therapeutic modality for PD. Effective targeted gene delivery is complicated in that gene vectors cannot penetrate the blood-brain barrier (BBB), thus clinical tests must rely on invasive intracerebral gene vector injection. Burst low-pressure focused ultrasound exposure with microbubbles has been demonstrated to noninvasively target and temporally open the BBB, opening new opportunities to transport large molecule substances into the brain for central nervous system (CNS) disease treatment, and raising the potential for noninvasive gene delivery for PD treatment. This paper reviews the underlying mechanism and current progress for focused ultrasound induced CNS gene delivery, and summarizes potential directions for further ultrasound-medicated PD gene therapy.
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FUS waves induce oscillation of intravenously injected microbubbles in the desired brain region, leading to transient BBB compromise by physically disrupting tight junctions between endothelial cells.
“The FUS waves induce oscillation of intravenously injected microbubbles in the desired brain region, leading to transient BBB compromise by physically disrupting tight junctions between endothelial cells [138, 139].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [138]
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- Authors
- Fan, C.-H., Lin, C.-Y., Liu, H.-L., & Yeh, C.-K.
- Affiliations
- National Tsing Hua University (TW)
- Chang Gung University (TW)
- Corresponding author
- Liu, Hao-Li
- Yeh, Chih-Kuang
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2017-09
- Volume
- 261
- Pages
- 246-262
- ISSN
- 0168-3659 (ISSN-L)
- OpenAlex
- W2730613845
- Cited by
- 97
- References
- 164
- Citations by year
- 2026: 3 · 2025: 6 · 2024: 14 · 2023: 5 · 2022: 10 · 2021: 19 · 2020: 11 · 2019: 15 · 2018: 11 · 2017: 1
- FWCI
- 0.83
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Controlled Release (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Ministry of Science and Technology, Taiwan (105-2221-E-182-022, 105-2221-E-007-055, 105-2119-M-182-001)
- National Tsing Hua University (106N522CE1)
- Chang Gung Memorial Hospital (CIRPD2E0051-53, CMRPD2D0111-13)
- Topics
- Physical Sciences › Engineering › Biomedical Engineering › Ultrasound and Hyperthermia Applications
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Biotechnology › Microbial Inactivation Methods
- Keywords
- Parkinson's disease, focused ultrasound, microbubbles, blood-brain barrier disruption, central nervous system gene therapy, dopaminergic neurons, neurodegeneration
- MeSH terms
- Animals, Blood-Brain Barrier, Brain, Genetic Vectors, Humans, Parkinson Disease, Ultrasonography, Genetic Therapy, Gene Transfer Techniques, Microbubbles
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