In back-to-back studies published in Nature, researchers from Purdue University and Columbia University report a naturally evolved gene-editing system that can activate genes, offering an advantage ...

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 ...

One year ago, a baby from Delaware County, Pennsylvania, became the first person in the world to receive a new revolutionary therapy at Children's Hospital of Philadelphia. "We didn't know that we ...

Gene editing is a numbers game. For any genetic tweaks to have notable impact, a sufficient number of targeted cells need to have the disease-causing gene deleted or replaced. Despite a growing ...

If you never eat grapefruit because you find them far too bitter, CRISPR varieties may be more to your taste. It has been shown that disabling one gene via gene editing can greatly reduce the level of ...

Crispr Therapeutics remains highly volatile, disconnected from near-term fundamentals, yet I see long-term value far above current levels. CRSP's business progress is slow, with pipeline milestones ...

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid ...

Menlo Ventures has made a $16 million bet that the “baby KJ” custom CRISPR therapy success story is repeatable. The funding has enabled CRISPR co-inventor Jennifer Doudna, Ph.D., and baby KJ scientist ...

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 ...

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 ...