Chronic kidney disease (CKD) is a public health challenge that affects more than 800 million people worldwide. 1 CKD can be caused by a variety of disease processes. Many causes are difficult to ...

Modifying hematopoietic stem cells to correct the underlying genetic defect, followed by reinfusion of the modified cells ...

The milestone reflects KFSH’s ongoing efforts to advance its gene therapy program, bridging clinical care with translational research. By leveraging patient-specific genetic profiles, the hospital is ...

A new study has answered a question that has puzzled cancer researchers for decades: Does a genetic defect found in nearly ...

Researchers identified biallelic variants in RNU2-2 as the cause of a recessive neurodevelopmental disorder marked by ...

New research from Queen Mary University of London (QMUL) has identified a novel syndrome in patients with kidney and adrenal disease. The research, published in the Journal of Clinical Investigation, ...

Scientists at Sanford Burnham Prebys Medical Discovery Institute and an international team of collaborators have defined a ...

A University of Oklahoma researcher is the first to discover that the sequence of the genetic defect in the neuromuscular disease Friedreich's ataxia isn't always as uniform as previously thought, a ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only minor ...