ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
Focused ultrasound and microbubble-mediated delivery of CRISPR-Cas9 ribonucleoprotein to human induced pluripotent stem cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed.
- Therapeutic Ultrasound in Cardiovascular Medicine: Applications of Low-intensity Pulsed Ultrasound and Ultrasound-targeted Microbubble Destruction.Cardiovascular drugs and therapy · 2026Review
- Ultrasound triggered microbubbles enhance the diagnosis and therapy of colorectal cancer.Discover nano · 2026Review
- Engineering delivery platforms for CRISPR-Cas and their applications in healthcare, agriculture and beyond.Nanoscale advances · 2026Review
- Ultrasound-triggered amygdalin drug release across U-87 glioblastoma cell lines from gH625-tagged phase convertible nanodroplets.Scientific reports · 2026Article
- Ultrasound medicine in the era of precision theranostics: Mechanisms, molecular strategies, and clinical translation.Iranian journal of basic medical sciences · 2026Review
- CRISPR-Based Functional Genomics in Pluripotent Stem Cells.Stem cell reviews and reports · 2026Review
- Spatiotemporal analysis of Escherichia coli membrane permeabilization and uptake kinetics induced by a single microbubble cavitation event.Scientific reports · 2025Article
- Nanoparticle-based systems for liver therapy: Overcoming fibrosis and enhancing drug efficacy.World journal of hepatology · 2025Review
- Therapeutic pCRISPRi Delivery to Lung Squamous Cell Carcinoma by Combining Nanobubbles and Ultrasound.Pharmaceutics · 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
- Application and prospects of ultrasound combined with immunotherapy in cancer treatment of intensive care.Frontiers in immunology · 2025Review
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
CRISPR-Cas9 ribonucleoproteins (RNPs) have been heavily considered for gene therapy due to their high on-target efficiency, rapid activity, and lack of insertional mutagenesis relative to other CRISPR-Cas9 delivery formats. Genetic diseases such as hypertrophic cardiomyopathy currently lack effective treatment strategies and are prime targets for CRISPR-Cas9 gene editing technology. However, current in vivo delivery strategies for Cas9 pose risks of unwanted immunogenic responses. This proof-of-concept study aimed to demonstrate that focused ultrasound (FUS) in combination with microbubbles can be used to deliver Cas9-sgRNA (single-guide RNA) RNPs and functionally edit human induced pluripotent stem cells (hiPSCs) in vitro, a model system that can be expanded to cardiovascular research via hiPSC-derived cardiomyocytes. Here, we first determine acoustic conditions suitable for the viable delivery of large proteins to hiPSCs with clinical Definity microbubble agents using our customized experimental platform. From here, we delivered Cas9-sgRNA RNP complexes targeting the EGFP (enhanced green fluorescent protein) gene to EGFP-expressing hiPSCs for EGFP knockout. Simultaneous acoustic cavitation detection during treatment confirmed a strong correlation between microbubble disruption and viable FUS-mediated protein delivery in hiPSCs. This study shows for the first time the potential for an FUS-mediated technique for targeted and precise CRISPR-Cas9 gene editing in human stem cells.
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