A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

Organoids’ ability to replicate aspects of the structure and function of larger tissues and organs makes them effective tools for studying diseases with complex etiologies as well as inherited ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

The history behind the discovery, research, and treatment of hemophilia is as complex and intriguing as the disease itself. A ...