A review article led by researchers from the B·ARGO group at the Germans Trias i Pujol Research Institute (IGTP) and from the ...

A treatment that is effective against hearing loss caused by the 'MPZL2 gene' mutation has been developed. A research team ...

Developed by Novartis, ITVISMA is a one-time therapy for patients aged 2 and older with a confirmed SMN1 gene mutation. The ...

A single microscopic change in our DNA can permanently alter how the brain is built, locking in developmental problems that ...

Imagine getting only a few hours of sleep and being raring to go the next day. A new study published in the journal Proceedings of the National Academy of Sciences shows the discovery of a gene ...

Researchers at Weill Cornell Medicine report that a rare gene mutation that delays Alzheimer’s disease does so by damping inflammatory signaling in brain-resident immune cells in a preclinical study.

By editing thousands of genes in mouse stem cells, the scientists identified a list of over 300 that are crucial for neural differentiation.

In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare ...

Two children from the same family are among seven confirmed cases worldwide with an incurable ultra-rare disease. In November 2022, Mel Dixon, 46, and her husband Charlie, 47, received “the ...