ArticleDrug delivery2022
Microbubble drug conjugate and focused ultrasound blood brain barrier delivery of AAV-2 SIRT-3.
Article in Drug delivery, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 32 citations in OpenAlex.
- Antibody Conjugation Strategies for Ultrasound Microbubbles: A Review of Bioconjugation Chemistry and Clinical Translation.Ultrasound in medicine & biology · 2026Review
- Intranasal vs. Device-Assisted Drug Delivery: Advantages and Limitations for the Delivery of Biopharmaceuticals to the CNS.Pharmaceutics · 2026Review
- Focused Ultrasound for the Treatment of Circuit and Molecular Pathology in Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026Review
- Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.Advanced drug delivery reviews · 2024Review
- Miniaturized therapeutic systems for ultrasound-modulated drug delivery to the central and peripheral nervous system.Advanced drug delivery reviews · 2024Review
- Noninvasive focal transgene delivery with viral neuronal tracers in the marmoset monkey.Cell reports methods · 2024Article
- The role of SIRT3 in homeostasis and cellular health.Frontiers in cellular neuroscience · 2024Review
- Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression.Gene therapy · 2023Article
- Noninvasive disruption of the blood-brain barrier in the marmoset monkey.Communications biology · 2023Article
- Virus-Based Biological Systems as Next-Generation Carriers for the Therapy of Central Nervous System Diseases.Pharmaceutics · 2023Review
- Nonspherical ultrasound microbubbles.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Preclinical Research on Focused Ultrasound-Mediated Blood-Brain Barrier Opening for Neurological Disorders: A Review.Neurology international · 2023Review
- Low intensity focused ultrasound: a new prospect for the treatment of Parkinson's disease.Annals of medicine · 2023Review
- Focused Ultrasound and Ultrasound Stimulated Microbubbles in Radiotherapy Enhancement for Cancer Treatment.Technology in cancer research & treatmentReview
Corrections and comments
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Authors and funding
7 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundDelivery of viral vectors as gene therapies to treat neurodegenerative diseases has been hampered by the inability to penetrate the blood brain barrier (BBB) and invasive or non-targeted delivery options prone to inducing immune responses. MR guided focused ultrasound (MR-g-FUS) and microbubbles have demonstrated safe, temporary, targeted BBB permeabilization clinically.
methodsWe developed clinically scalable, microbubble drug conjugates (MDCs) for the viral gene therapy, AAV.SIRT3-myc [adeno-associated virus expressing myc-tagged SIRT3], which has previously been shown to have disease modifying effects in animal models of Parkinson's disease (PD). The lipid shells of the perfluorocarbon gas MDCs were covalently conjugated to antibodies with binding specificity to AAVs. Following systemic (iv) delivery of AAV.SIRT3-myc MDCs, MR-g-FUS was used to deliver SIRT3-myc to brain regions affected in PD. SIRT3-myc expression was determined post mortem, using immunohistochemistry.
resultsAn
conclusionThese studies demonstrate that MDCs combined with MR-g-FUS are an effective method for delivery of viral vector gene therapies, such as AAV.SIRT3, to brain regions affected in PD. This technology may prove useful as a disease-modifying strategy in PD and other neurodegenerative disorders.
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