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 ...

Researchers are working to reveal potential threats to the efficacy of CRISPR/Cas9 gene editing, even when it appears to be working as planned. A Rice University lab is leading the effort to reveal ...

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 ...

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 ...

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 ...