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safeEXO-Cas
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Researchers at Columbia University College of Dental Medicine have developed an exosome-based platform, “safeEXO-Cas,” that significantly enhances the delivery of CRISPR/Cas9 genome editing components to specific cells and tissues.
Research Highlights:
- Targeted Delivery: Research highlights the platform’s potential in treating genetic diseases by providing targeted, efficient, and safe delivery of CRISPR/Cas9.
- Successful Gene Editing: In experimental models, the platform successfully edited the EMX1 gene in lung tissue without causing morbidity or significant immune responses.
- Future Applications: This breakthrough paves the way for further research and clinical applications, offering hope for patients with genetic diseases lacking effective treatments.
- Avenues for Precision Medicine.
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- Customization for Precision: The ability to customise exosomes for specific tissues opens new avenues for precision medicine.
- Scalability and Efficiency: The study emphasises the significance of safeEXO-Cas as a scalable and efficient method for targeted gene editing.
Exosomes and their use in Gene Therapy:
- Exosomes are small, membrane-based vesicles found inside cells, that serve as transporters for molecules, including proteins and genetic material, between cell
- Utilised as efficient and non-immunogenic carriers for delivering CRISPR/Cas9 components
- Exosomes can be obtained from patients’ tissues or body fluids.
- Tissue-Specific Engineering: Exosomes engineered to express tissue-specific moieties, enhancing uptake by target cells and minimising off-target effects.
- These small, membrane-bound structures are essential for cell-to-cell communication and hold promise for applications in drug delivery, diagnostics, and therapeutics.
- Overcoming Limitations: Exosomes offer potential solutions to limitations of current delivery methods, including immunogenicity, limited cargo capacity, and off-target effects.
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CRISPR-Cas9 Overview:
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