The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...

Researchers at the University of Trento have developed a CRISPR-based adenine base editing therapy that repaired the ...

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 ...

While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...

Artificial intelligence is accelerating advances across genetics, from precision genome editing to complete crop genome mapping and personalized disease risk prediction. Recent achievements include ...

Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...