ArticleProceedings of the National Academy of Sciences of the United States of America2023
Focused ultrasound-mediated brain genome editing.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed.
- Focused ultrasound-mediated lipid nanoparticle delivery for brain gene editing.Molecular therapy. Nucleic acids · 2026Article
- Dual Physiological Barriers Bypassed by a Silk-Based Supramolecular Protein Delivery Platform for Neuroinflammation Mitigation in Alzheimer's Disease.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Towards clinical translation of nanomedicines: Formulation scale-up and model systems.Advanced drug delivery reviews · 2026Review
- Focused Ultrasound Modulation of PGC-1α Pathways in Neurological Disease: Mechanistic Rationale and Translational Opportunities.Molecular neurobiology · 2026Review
- Inflamed vessel-anchored release of HScience advances · 2026Article
- Viral vector-based gene therapies in the clinic: An update.Bioengineering & translational medicine · 2026Review
- Review
- Chemical strategies for brain delivery of genomic therapy.Nature reviews. Chemistry · 2025Review
- Characterization of microbubble cavitation in theranostic ultrasound-mediated blood-brain barrier opening for gene delivery.Journal of controlled release : official journal of the Controlled Release Society · 2025Article
- Acoustofluidic system for targeted antibody removal in transplantation: Enabling small-volume therapeutic apheresis.Science advances · 2025Article
- Focused ultrasound-mediated APOE4 knockdown in mouse brain.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Article
- Exosomes-mediated delivery of miR-486-3p alleviates neuroinflammation via SIRT2-mediated inhibition of mitophagy after subarachnoid hemorrhage.Stroke and vascular neurology · 2025Article
- A combination of systemic mannitol and mannitol modified polyester nanoparticles for caveolae-mediated gene delivery to the brain.Molecular therapy. Nucleic acids · 2025Article
- Focused-ultrasound-mediated CRISPR-Cas9 gene editing in human induced pluripotent stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Focused ultrasound and microbubble-mediated delivery of CRISPR-Cas9 ribonucleoprotein to human induced pluripotent stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Advancements and challenges in brain cancer therapeutics.Exploration (Beijing, China) · 2024Review
- Article
- Strategies for enhanced gene delivery to the central nervous system.Nanoscale advances · 2024Review
- Acoustically targeted noninvasive gene therapy in large brain volumes.Gene therapy · 2024Article
Corrections and comments
- Update of
Authors and funding
23 authors.
Funding
Abstract
Gene editing in the brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches mainly rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions of the brain. We demonstrate a safe and effective gene editing technique by using focused ultrasound (FUS) to transiently open the BBB for the transport of intravenously delivered CRISPR/Cas9 machinery to the brain.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.