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

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 ...

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

Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.

For too long, the promise of personalized therapies has been tantalizingly close, yet frustratingly out of reach for ...

"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

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

Rapidly testing hundreds of thousands of DNA sequences, scientists identified specific genetic variations contributing to blood pressure, cholesterol, and blood sugar.

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

Domestic cats are some of the most beloved pets in the world. Their mysterious nature, grace and unique character make them special human companions. However, for these pets to remain healthy and live ...