Recognized in the Biotechnology category of the 2025 Top Innovations contest, small Cas12l nucleases are versatile, ...
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 ...
A new West Coast biotech has emerged into the busy in vivo cell therapy space, this time with the backing of Nobel Prize-winning CRISPR pioneer Jennifer Doudna, Ph.D. Azalea Therapeutics has bloomed ...
Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
When scientists discovered how bacteria protect themselves against viral invaders, called phages, in the early 2000s, little did they know they'd stumbled upon a revolutionary tool researchers could ...
CHICAGO – A CRISPR-Cas9-based investigational therapy was linked with a drop in serum transthyretin (TTR) levels in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), according to ...
In December 2020, the New England Journal of Medicine published a paper titled CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia, marking an exciting new chapter in the treatment of ...
Intellia Therapeutics has paused dosing in its Phase 3 MAGNITUDE trials after a patient experienced severe liver toxicity, as ...