More than 9 out of 10 Alzheimer's cases could be driven by specific variations in a single gene and the protein it produces, ...

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

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

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 ...

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.

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 ...

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 ...

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 ...