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

Genetic mutations aren't always a bad thing. A groundbreaking new study suggests that mutations on a single gene may actually be beneficial when it comes to Alzheimer’s disease. For the study, Dr.

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

A new single-cell profiling technique has mapped pre-malignant gene mutations and their effects in solid tissues for the ...

Researchers have discovered new regions of the human genome particularly vulnerable to mutations. These altered stretches of DNA can be passed down to future generations and are important for how we ...

Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism by which brain cells die. A team led by scientists at the German research ...

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 ...

A rare form of diabetes affecting newborn babies and caused by a genetic disorder has been discovered by scientists in the UK ...

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

Scientists have revealed parts of the genome that are especially vulnerable to mutations that occur very early on in development. These areas are in the initial portions of genes, where the cell tends ...

Human cells usually contain two copies of most genes, one of which comes from the mother while the other comes from the father. It's long been thought that usually these two copies, or alleles of ...