A hybrid CRISPR design pipeline improves the predictability of genome editing outcomes in mouse embryos. By favouring microhomology-mediated end joining (MMEJ) and validating guides in stem cells, the ...

Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo ...

Incyte will share updated clinical single-cell data characterizing its calreticulin-targeted therapy for myeloproliferative ...

Zugocaptagene geleucel; zugo-cel (formerly CTX112) is an investigational, allogeneic, off-the-shelf CD19-directed CAR T-cell therapy under development for autoimmune diseases and B-cell malignancies.

A genome-wide CRISPR-Cas9 screen to identify transcription factor SOX2 as a critical regulator of PROM1, the gene encoding CD133, in glioblastoma stem cells. Knocking out SOX2 reduced CD133 expression ...

Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in ...

A portable CRISPR-Cas13 assay detected mpox virus with 100% sensitivity and specificity in clinical samples from Sierra Leone. The SHINE test identified as few as 10 copies/µL and delivered results in ...

The first approved CRISPR medicine has now shown similarly strong results in children as young as five years old with sickle cell disease and transfusion-dependent β-thalassaemia. In a pair of phase 3 ...

Researchers have identified how the bacteriophage protein AcrIIA27 inhibits diverse Cas9 enzymes by binding solvent-exposed regions of the guide RNA rather than the protein itself. This structural ...

Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA... Cargo-Sized Lipid Screen Extends Gene Editing ...

Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA... Rival Editors Converge on AATD as Data Mount Alpha ...