Researchers at the University of Tsukuba have developed a vector that integrates the Cre enzyme gene with the specific DNA ...

ECURE previously announced that the first patient dosed with ECUR-506 for OTC deficiency achieved a complete clinical ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

The team of researchers found that a gene in Poncirus trifoliata, a hardy citrus relative, is what's behind the plant's ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Researchers have engineered yeast to efficiently convert methanol into D-lactic acid, a key compound for biodegradable plastics and pharmaceuticals. By optimizing gene and promoter combinations, they ...

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead ...

The round will fund a “one-and-done” treatment for a rare kidney condition that its CEO says can address concerns patients ...