Hepatocellular carcinoma (HCC) is a leading cause of cancer-related death globally, with incidence continuing to rise in many regions. Despite advances in ...

Genome editing is revolutionizing the medical landscape by offering new therapeutic possibilities for genetic diseases, which collectively affect hundreds ...

Charles Gersbach and his colleagues are pursuing promising CRISPR technologies focused on controlling gene activity rather ...

A platform approach to gene editing brings together design, manufacturing, validation, analytics, and regulatory considerations right from the start.

In research published today in Nucleic Acid Research, co-first author Seong Guk Park, PhD, and corresponding author Elizabeth Kellogg, PhD, St. Jude Department of Structural Biology, designed a ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

I was born in Washington, DC, in 1964, the oldest of three sisters, but the place that truly formed me as a scientist was Hilo, on the Big Island of Hawaii. Growing up there, far from the mainland ...

CRISPR-based therapy is a transformative approach to medicine that allows for targeted changes to the genome by directly addressing the source of disease, or to the epigenome through silencing of ...

Powered by gene editing advances like CRISPR and base editing, cell and gene therapy (CGT) is delivering on the promise of genomic medicine. First-generation CAR T-cell therapies, for example, have ...