A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

Manipulating “thresholds” with mitochondrial gene editing tools. By focusing on the genetic roots of neurodegenerative diseases, mitochondrial DNA editing represents a shift in therapeutic strategies.

For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these mutations cause serious diseases that pass down only through mothers. They ...

The U.S. Food and Drug Administration approved the first cell-based gene therapies for sickle cell disease, including the ...

The energy factories in our cells contain their own genes, and genetic mutations in them can cause deadly inherited diseases. These oblong-shaped organelles, or mitochondria, translate genes into ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Clustered regularly interspaced short palindromic repeats (CRISPR) refer to the small fragments of viral DNA that are stored by the bacteria as a part of their defense mechanism. CRISPR–Cas9 is a ...

A visualization of the A3G editor's crystalline structure, with red arrows indicating locations the engineers modified to change their affinity for binding to DNA. Making the editor more accurate ...