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In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery

11 Mar 2020

Abstract excerpt

Abstract Ex-vivo gene therapy using stem cells or T cells transduced by retroviral or lentiviral vectors has shown remarkable efficacy in the treatment of immunodeficiencies and cancer. However, the process is expensive, technically challenging, and not readily scalable to large patient populations, particularly in underdeveloped parts of the world. Direct in vivo gene therapy would avoid these issues, and such...

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PMID: 32161326
DOI: 10.1038/s41598-020-61518-w

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