Six patients with glycogen storage disease type Ia have received a single infusion of BEAM-301, an in vivo base editor ...
Researchers built a three-layer human blood-vessel model that shows how fibroblasts amplify the vascular pathology of ...
Researchers have used CRISPR-Cas9 to remove CD28 from donor T cells and prevent fratricide before equipping them with ...
Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for alpha-1 antitrypsin ...
Researchers built a three-layer human blood-vessel model that shows how fibroblasts amplify the vascular pathology of Hutchinson-Gilford progeria... Clinical: FDA Backs Single-Trial Pathway for ...
Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo length increased. In mice, route-specific LC-1 formulations delivered ...
Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in cranial neural crest cells rather than only in oral epithelium. CRISPR-Cas9 ...
The minimal residual disease-positive cohort opens as part of the pivotal-stage development of Soficabtagene Geleucel (Sofi-cel), an off-the-shelf CAR-T cell therapy under evaluation for relapsed or ...
A mutation-agnostic CRISPR-Cas strategy that inserts a functional rhodopsin coding sequence into the human RHO locus has preserved retinal structure, function and visual acuity for a full year in a ...
A UCL-led team has developed a clinical-scale CRISPR-Cas9-AAV6 workflow intended to correct Wiskott-Aldrich syndrome in haematopoietic stem and progenitor cells, reaching 61% knock-in in more than 100 ...
Merck & Co. will present new single-cell functional genomics data generated using purpose-built assays developed with Mission Bio’s Assay Services team. Incyte will share updated clinical single-cell ...
Dixon joins as an independent Board member, adding deep healthcare finance and transaction expertise as VedaBio enters its next phase of growth SAN DIEGO--(BUSINESS WIRE)--VedaBio, a pioneering ...
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