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 ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

In the world of biopharmaceutical innovation, 2024 will be remembered as CRISPR’s breakout year. In the spring, five patients with sickle cell disease began treatment with Casgevy, the first ...

Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...

Gene-editing techniques such as CRISPR-Cas9 have many uses in the area of food and agriculture. They can combat persistent drought and disease, and improve the colour and nutritional content of food ...

Since the landmark discovery that a bacterial defense mechanism could be repurposed into programmable “molecular scissors,” CRISPR-Cas9 gene-editing technology has undergone a major evolution. Over ...

NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...

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 ...