A Cleveland Clinic first-in-human clinical trial has shown that a one-time infusion of a gene-editing therapy using ...

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 ...

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 ...

In 2020, Jennifer Doudna, Ph.D., received the Nobel Prize in Chemistry, CRISPR-Cas9, a method for genome editing. Often referred to as “molecular scissors,” CRISPR cuts DNA at specific locations that ...

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 ...

The practice of genetic modification is as old as humanity. For thousands of years, humans have bred crops, livestock and even pets that possess desirable traits. This selective process, which alters ...

Scientists at the Department of Energy (DOE)'s Oak Ridge National Laboratory (ORNL) have shown that more than 93% of possible ...

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 ...

Researchers have identified critical spots on the genome where gene editing could cause an unwanted response, and they provide recommendations for safer approaches. CRISPR/Cas9 is a commonly used, ...

As far back as 2015, scientists have been warning against using CRISPR-Cas9 technology to modify germline genomes. Indeed, a perspective published that year in Science suggested that otherwise ...