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 ...

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.

The practice of genetic modification is as old as humanity. For thousands of years, humans have bred crops, livestock and even pets that possess desirable traits. This selective process, which alters ...

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 ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

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 ...

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 ...