Researchers at Monash University in Melbourne, Australia, have uncovered the hidden code governing how genetic mutations affect RNA splicing and result in disease. The researchers were able to ...

Although the genetic cause of many diseases have been identified, it's estimated that as many as 70% of patients with a rare ...

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

Around 25 million Americans have a rare genetic disease, and many of them struggle with not only a lack of effective ...

Researchers at Google DeepMind, the tech giant's artificial intelligence arm, on Tuesday introduced a tool that predicts whether genetic mutations are likely to cause harm, a breakthrough that could ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects ...

Scientists say they have found a pattern of so-called epigenetic 'marks' in a transition state between normal and pancreatic cancer cells in mice, and that the normal cells may keep at least a ...

Identifying and classifying gene mutations - which are the permanent changes in a person's DNA genetic code - are critical in better understanding, and with research, eventually treating or preventing ...

Researchers at Memorial Sloan Kettering Cancer Center (MSK) have developed some of the first robust laboratory models of a ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...