Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-025-04115-6 A blueprint to accelerate rare pediatric gene therapy …
Tag:
Gene Therapy
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Hu, P. et al. Prevalence of familial hypercholesterolemia among the general population and patients with atherosclerotic …
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Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary …
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RESEARCH HIGHLIGHT 30 January 2026 In patients with T cell acute lymphoblastic leukemia, base-edited CAR T …
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Major Accountabilities ~ Prepare an annual purchasing strategy for a small division or business or country/region …
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Reading time: 2 minutes Jesse Owens in his lab at the John A. Burns School of …
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Nature Medicine, Published online: 27 October 2025; doi:10.1038/s41591-025-04070-2 Author Correction: Cardiac gene therapy makes a comeback
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Nature Medicine, Published online: 21 October 2025; doi:10.1038/s41591-025-04002-0 The first-in-human trial of AAV2i8-I1c (AB-1002) — an …
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Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency …
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Chang, Q. et al. Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores …