Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Genome editing has advanced at a rapid pace with promising results for treating genetic conditions—but there is always room for improvement. A new paper by investigators from Mass General Brigham ...

A team of doctors and scientists has used a tailor-made gene-editing therapy to treat an infant with a rare genetic condition, a medical first that opens the door to a new era of individualized ...

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Future AI-guided genomic analysis offers the potential to further optimize gene constructs, expression profiles, and performance traits, accelerating the development of next-generation materials.

Gabriella Ngang, 18, of Farmington Hills is one of just three people in Michigan to take part in a clinical trial testing a new gene-editing therapy with the potential to cure her sickle cell disease.