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Engineering cell-derived extracellular vesicles for gene therapy

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Many genetic therapies are limited by a lack of methods for delivering them to target cells in the body. We have developed technologies to engineer biological nanovesicles to load therapeutic proteins, target recipient immune cells and deliver Cas9 to knock out CXCR4 in primary human T cells.

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Fig. 1: The GEMINI strategy for producing multifunctional EVs.

References

  1. Dooley, K. et al. A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties. Mol. Ther. 29, 1729–1743 (2021). This paper reports general methods for the surface display of proteins on EVs and for EV cargo loading.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Gao, Y. et al. Complex transcriptional modulation with orthogonal and inducible dCas9 regulators. Nat. Methods 13, 1043–1049 (2016). This paper describes chemically inducible protein dimerization systems.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  3. Mangeot, P. E. et al. Protein transfer into human cells by VSV-G-induced nanovesicles. Mol. Ther. 19, 1656–1666 (2011). This paper reports the use of VSV-G as a method for achieving EV-mediated protein transfer.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  4. Frecha, C. et al. Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins. Blood 112, 4843–4852 (2008). This paper details the challenges of transducing T cells and presents measles virus glycoproteins as a method for overcoming these challenges.

    Article  CAS  PubMed  Google Scholar 

  5. de Jong, O. G. et al. A CRISPR-Cas9-based reporter system for single-cell detection of extracellular vesicle-mediated functional transfer of RNA. Nat. Commun. 11, 1113 (2020). This paper describes an assay for evaluating EV-mediated functional cargo delivery.

    Article  PubMed  PubMed Central  Google Scholar 

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This is a summary of: Stranford, D. M. et al. Genetically encoding multiple functionalities into extracellular vesicles for the targeted delivery of biologics to T cells. Nat. Biomed. Eng. https://doi.org/10.1038/s41551-023-01142-x (2023).

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Engineering cell-derived extracellular vesicles for gene therapy. Nat. Biomed. Eng 8, 343–344 (2024). https://doi.org/10.1038/s41551-023-01167-2

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  • DOI: https://doi.org/10.1038/s41551-023-01167-2

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