Global clinical-stage biotech company HuidaGene Therapeutics has received FDA clearance to move forward with its application for its investigational drug, a CRISPR/Cas13 RNA-editing therapy to treat ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism.

Scientists have discovered a new CRISPR mechanism with precise activity, expanding the potential applications of the existing ...

CRISPR was one of the most influential breakthroughs of the last decade, but it's still imperfect. While the gene editing tool is already helping people with genetic ailments, scientists are also ...

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 major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...

For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...

A powerful form of DNA-editing machinery discovered in bacteria might allow us to make much bigger changes to genomes than is currently possible with CRISPR-based techniques. However, it isn’t yet ...

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 ...