Gene therapy is no longer the future of medicine — it's reality. Treatments that replace, silence or edit genes are already changing clinical ...

Korro and Tevard outlined approaches designed to expand the therapeutic use of RNA medicines beyond protein silencing, highlighting programs that aim to repair genetic defects or restore production of ...

She had just diagnosed our 11-year-old daughter, Maya, with Nicolaides-Baraitser syndrome, a genetic condition so rare there ...

Researchers report a previously unrecorded de novo GLI3 frameshift variant in a Palestinian infant with Pallister-Hall ...

A massive genetic analysis reveals that while men and women share much of the underlying risk for anxiety, they also possess ...

The majority of rare diseases have a genetic cause. The underlying genetic alteration can be found more and more easily, for example by means of exome sequencing (ES), leading to a molecular genetic ...

Myelodysplastic syndromes (MDS) are a group of disorders that typically arise in adulthood, especially after the age of 70, and their five-year survival rate is around 30%. MDS are characterized by ...

In a landmark discovery, researchers from the University of Antioquia have identified a new genetic syndrome that bridges the gap between neurodevelopmental disorders and neurodegenerative diseases.

Population cohorts combining genetics with multi-organ imaging reveal that brain disorders share a distributed genetic ...

Children with rare genetic disorders often face years of uncertainty before receiving a diagnosis, leaving families without clear information about disease progression, treatment options, prognosis, ...

“Ultimately, we'll help the people we discriminate against if we try to understand more about them; genetics will lead to a world where there is a sympathy for the underdog.”—James D. Watson A recent ...