For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

(RTTNews) - Vertex Pharmaceuticals (VRTX), Thursday reported compelling long-term data for its CRISPR/Cas9 gene-edited therapy, PrCASGEVY, at the EHA Congress. In sickle cell disease trials, 95.6 ...

Scientists have taken an important step toward a gene therapy that could one day turn off the extra genetic material that ...

Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...

A graphic of a purple and magenta Cas9 protein, which is a somewhat round, but uneven, ball, bound to a yellow and orange ...

The rapid evolution of CRISPR/Cas genome editing has redefined the possibilities of cellular and gene therapy, enabling ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

If CRISPR stays active too long, it could cut unintended parts of the genome. To reduce this risk, the researchers designed a self-inactivating CRISPR system. This means that CRISPR edits the gene and ...

An appeals court has opened another chapter in the long-running CRISPR patent saga. Three years after a patent body ruled in favor of the teams behind Editas Medicine’s intellectual property, an ...