New research finds that retinal diseases thought to map one-to-one to genetic mutations are more complicated than that.

Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

Mutations in the tumor suppressor TP53 are a common cause of cancer, making the altered protein an attractive target for ...

Meet the luckiest people on Earth — individuals carrying a rare genetic mutation that prevents wrinkles from forming even as they age well into their 80s. These genetic lottery winners have provided ...

A genetic mutation passed from mother to children in families affected by schizophrenia has now been shown to completely ...

Researchers identified a new genetic mutation that may allow some people to survive, even thrive, on three hours of sleep. The mutation is called SIK3-N783Y. Scientists said mice test subjects ...

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

The domestication of horses is a much more recent even compared to other domesticated species; horses were domesticated about 4,500 years ago, which was about 5,000 years after the domestication of ...

Much of pancreatic cancer’s evasive and deadly nature may come down to low levels of one gene. In A Nutshell Early detection, late impact: Scientists found that a gene called CTDNEP1 shows reduced ...

An affordable new BRCA gene test has hit the market, but who should take it? — -- A new genetic testing kit that hits the market today is the most affordable, and arguably one of the simplest, ...