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 ...

DNA doesn’t just sit still inside our cells — it folds, loops, and rearranges in ways that shape how genes behave.

Only around two percent of the human genome codes for proteins, and while those proteins carry out many important functions of the cell, the rest of the genome cannot be ignored. However, for decades ...

Identifying and classifying gene mutations—which are the permanent changes in a person's DNA genetic code—are critical in better understanding, and with research, eventually treating or preventing, ...

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 ...

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

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 ...

For 10% of colorectal cancer patients, hereditary factors play a role, with higher percentages among younger patients.