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 ...
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.
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 ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
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 ...
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases. Last February ...
Last year, Verve Therapeutics started the first human trial of a CRISPR treatment that could benefit most people—a signal that gene editing may be ready to go mainstream. CRISPR for high cholesterol ...
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 ...
Redwood City, California – May 16, 2017 – Synthego, a leading provider of genome engineering solutions, announces powerful online CRISPR tools that make accessible over 100,000 genomes for fast and ...