A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

Genetic diseases are notoriously challenging to treat, especially when each condition requires a tailored approach. With over 10,000 known genetic disorders, developing individual therapies for each ...

A therapy originally developed for HIV now provides new options for patients with rare genetic disorders. It’s now possible to use these drugs to address DNA defects and enhance muscle function. This ...

Mayo Clinic researchers have identified a rare mutation in the MET gene that can directly cause metabolic dysfunction-associated steatotic liver disease. The mutation disrupts the liver’s ability to ...

A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

A closer examination of the APOL1 gene in Black patients with kidney disease can provide more accurate diagnoses than current ...

This Ice Age pair of hunter-gatherers were both shorter than average for their population – one of them markedly so – and now we finally know why. A team of researchers from Europe re-examined the ...

A new study published in Neurology, led by Queen Mary University of London, has revealed that people of South Asian, African ...

Wounded and elderly patients are being evacuated by train from embattled east Ukraine - Copyright AFP Ishara S. KODIKARA Wounded and elderly patients are being ...

Horses are just as much a part of New Mexico’s story as green chile and balloons. One local startup is hoping their research ...