A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two ...

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 ...

Environmental factors such as lifestyle and the medications you take influence the effects your genes have on your body — and can clarify how diseases develop. When you purchase through links on our ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

Huntington’s disease (HD) is one of those rare conditions that affects not just the patient but the entire family—medically, emotionally and genetically. This inherited brain disorder causes gradual ...

A recent study finds that certain psychiatric disorders have considerable genetic overlap. But these genes aren’t the whole story; you have a say in what they do.

Genetic inheritance may sound straightforward: One gene causes one trait or a specific illness. When doctors use genetics, it’s usually to try to identify a disease-causing gene to help guide ...

Parkinson's disease (PD) is a debilitating and progressive neurodegenerative disorder caused by the loss of ...

State Sen. Danny Burgess and state Rep. Adam Anderson have filed legislation that would address a shortage of genetic ...