Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders ...

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources ...

In a step toward treating mitochondrial diseases, researchers at the University Medical Center Utrecht and their colleagues have used mitochondrial base editing (mtBE) to successfully edit harmful ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

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 ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

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 ...

Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common ...