Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

Gene regulation is far more predictable than previously believed, scientists conclude after developing the deep learning ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Despite its strong influence, APOE is not the sole cause of Alzheimer’s or other dementias. Even among people with the ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Scientists have uncovered a surprising genetic shift that may explain how animals with backbones—from fish and frogs to ...

Psoriasis is a common inflammatory skin disease linked to several long-term health conditions, such as heart disease, ...

Gene therapy drugs have the potential to cure some diseases, but some have a price tag of over a million dollars. Who gets access to them and who doesn't?

Modern industry relies heavily on catalysts, which are substances that speed up chemical reactions. They're vital in ...