#gene therapy — Thursday, September 24, 2026 (2 items)
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Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.
(linkinghub.elsevier.com)
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Cell Stem Cell 2026-09-24 | #gene therapy #lentiviral #sickle cell disease #hematopoietic #stem cell | |
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Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome.
(nejm.org)
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New England Journal of Medicine 2026-09-24 | #gene therapy #hematopoietic stem cells #immunodeficiency #wiskott-aldrich-syndrome #lentiviral vector |