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 ...

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

Precision gene editing can be achieved by base editing, nucleotide editing, or editing of small or large gene fragments. The specific mechanisms include various DNA repair pathways. Many precision ...

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.

CRISPR-Cas is used broadly in research and medicine to edit, insert, delete or regulate genes in organisms. TnpB is an ancestor of this well-known 'gene scissor' but is much smaller and thus easier to ...

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 ...

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 ...

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.