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

Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients ...

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

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

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 ...

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 ...

Please provide your email address to receive an email when new articles are posted on . The expression of GSDMB was high in children with a 17q21 genetic variant. These children did not have ...

CNBC anchor Becky Quick opens up about daughter’s rare genetic disease - Quick’s daughter was diagnosed with SYNGAP1 when she ...

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, ...

The Iowa State University Extension and Outreach Dairy Team monthly webinar series continues from noon to 1 p.m. April 2. The program will help Holstein breeders understand the muscle weakness genetic ...

Researchers have identified a gene that causes heart defects in Down syndrome, a condition that results from an additional copy of chromosome 21. Reducing the overactivity of this gene partially ...