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

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

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 ...

A large genetic screen has revealed how stem cells transform into brain cells, exposing hundreds of genes that make this ...

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

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 discovered that aging DNA repeats expand at wildly different speeds—and in some people, the consequences can be devastating. A sweeping genetic study drawing on data from more than 900,000 ...

Rebecca Quick, who is known as Becky Quick, has shared that her daughter Kaylie has a rare genetic disease. The CNBC anchor shared that, for the past 6.5 years, the family has kept Kaylie's condition ...