Researchers have used CRISPR-Cas9 to remove CD28 from donor T cells and prevent fratricide before equipping them with CD28-targeting CARs. In...
Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA...
Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo length increased. In mice,...
Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in cranial neural crest cells...
A nuclease-inactive CRISPR-Cas9 system increased endogenous expression of two protective HDL components without cutting genomic DNA. In a small mouse...
A UCL-led team has developed a clinical-scale CRISPR-Cas9-AAV6 workflow intended to correct Wiskott-Aldrich syndrome in haematopoietic stem and...
Packing chemical modifications throughout the prime editing guide RNA, rather than only at its ends, raised editing in mouse liver to 67% after a...
Delivering a CRISPR-Cas-derived adenine base editor as a short-lived protein-guide RNA complex enabled efficient PCSK9 editing while human embryos...
