Cystic fibrosis (CF) therapeutics refer to treatments designed to manage and alleviate the symptoms of cystic fibrosis, a ...

Classical CRISPR systems use molecular scissors to snip through DNA ... Credit: CHRISTOPH BURGSTEDT/SCIENCE PHOTO LIBRARY via ...

Clonal hematopoiesis of indeterminate potential (CHIP) is a known risk factor for coronary artery disease, though its precise role in disease progression continues to emerge. This study leverages ...

A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes towards the controversial approach.

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...

Most current approaches rely on adding nuclear localization signal (NLS) motifs to the ends of Cas9 to facilitate nuclear entry. However, this method is inefficient, and much of the Cas9 that is ...

Vertex Pharmaceuticals (VRTX) is rated as a "Strong Buy" due to robust revenue growth and multiple expansion opportunities ...

Intellia Therapeutics, Inc. is rated Hold due to uncertainty from dosing pause in its MAGNITUDE program for ATTR. Learn more ...

Two days after Intellia voluntarily paused dosing due to safety concerns, the regulator stepped in with an official hold that ...

First company to enable precision in vivo genome engineering, combining both cell-specific delivery and programmable, locus-specific gene insertion, with the potential to achieve physiological, durabl ...

Cancer cells contained hubs that accelerated diseases, mediated by hijacked RNA, but scientists found how to cut them off at ...