A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

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 ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in ...

Sadly, there is no cure for Huntington’s disease. But a couple new research papers suggests this may be about to change.

In an exciting scientific first, researchers at the Allen Institute successfully designed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders (SRD) in mice. These are a ...

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 the Icahn School of Medicine at Mount Sinai and collaborators have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

Affecting 1 in 500 people, hypertrophic cardiomyopathy is a condition in which the walls of the left ventricle, the heart's main pumping chamber, become abnormally thick. "HCM is one of the primary ...

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 ...

The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers at the University of Zurich have now shown that there ...

Potentially more than 90% of Alzheimer's disease cases would not occur without the contribution of a single gene (APOE), ...

Scientists have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect. Scientists at the Icahn School of Medicine at Mount Sinai and ...