A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes towards the controversial approach.

Most current approaches rely on adding nuclear localization signal (NLS) motifs to the ends of Cas9 to facilitate nuclear entry. However, this method is inefficient, and much of the Cas9 that is ...

Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

For years, the CRISPR-Cas9 genome technology has been reshaping genetic engineering, a precision tool to transform everything from agriculture to medicine. With its incredible efficiency, this ...

We performed both homology-based and GUIDE-seq nomination of candidate off-target sites, which resulted in the testing of 5002 sites from four healthy donors (not shown). We then nominated additional ...

Researchers corrected mutations that cause cystic fibrosis in cultured human stem cells. They used a technique called prime editing to replace the 'faulty' piece of DNA with a healthy piece. The study ...

Scientists testing CRISPR-Cas9 in zebrafish discovered unforeseen DNA mutations, some inherited by the next generation. These results raise red flags about using the tool in human medicine, where ...

As far back as 2015, scientists have been warning against using CRISPR-Cas9 technology to modify germline genomes. Indeed, a perspective published that year in Science suggested that otherwise ...

Both CRSP and NTLA are advancing in vivo gene editing therapies targeting large markets, with financial stability supporting ...