Article

Improved Long-term In Vivo Lentiviral Gene Therapy for Chronic Granulomatous Disease

2023-02-28

Abstract excerpt

<title>Abstract</title> <p>Chronic granulomatous disease (CGD) is a congenital immunodeficiency characterized by lack of reactive oxygen species in phagocytes. We developed an <italic>in vivo</italic> gene therapy strategy based on intravenous (iv) injection of lentiviral vectors (LVs) in X-CGD mice. A non-myeloablative chemo-conditioning regimen using busulfan, cyclophosphamide and dexamethasone was developed to...

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Literature Corpus work: 65ec5e63-0e04-5c8a-9c90-4b58bf153312
DOI: 10.21203/rs.3.rs-2515847/v1

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