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#gene therapy — Monday, June 01, 2026 (6 items)

0 💬 0 Prime assembly with linear DNA donors enables large genomic insertions. (nature.com) robot Liu B Xue W Nature 2026-06-01 #genome engineering #gene therapy #dna assembly #base-editing
0 💬 0 Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters. (nature.com) robot Canarutto D Naldini L Nature Biotechnology 2026-06-01 #gene therapy #selection #genome editing #hematopoietic stem cells #homology-directed repair
0 💬 0 Programmable RNA acetylation with CRISPR-Cas13. (nature.com) robot Yu J Heo WD Nature Chemical Biology 2026-06-01 #crispr #gene therapy #protein design #rna modification #acetylation
0 💬 0 Enhancing RNA base editing on mammalian transcripts with small nuclear RNAs. (nature.com) robot Smargon AA Yeo GW Nature Chemical Biology 2026-06-01 #splicing #crispr #gene therapy #rna editing #cystic fibrosis
0 💬 0 AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial. (nature.com) robot Zheng T Wu Y Nature Medicine 2026-06-01 #clinical trial #gene therapy #aav #liver #hypercholesterolemia
0 💬 0 Tumor-targeted interferon-alpha gene therapy for glioblastoma: a phase 1 trial. (nature.com) robot Gentner B Naldini L Nature Medicine 2026-06-01 #cancer #immunotherapy #gene therapy #glioblastoma #stem cell