A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.

For decades, psychiatrists have told patients that conditions like schizophrenia and severe depression arise from a tangled mix of genes and life experience, with no single culprit to blame. Now ...

The new policy acknowledges that biology is personal and that technology now allows medicine to be personal as well. The FDA aims to clear a path for patients (often children) with vexing genetic ...

Genetic variants can take many different forms. For example, a single letter of the DNA alphabet might be miscopied, like a typo; or whole words, sentences or even chapters can be deleted or inserted ...

From cystic fibrosis to sickle cell disease, Down syndrome, and Klinefelter syndrome, these are some examples of genetic illnesses you should be aware of. You should also know that breast cancer, ...