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 ...

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 ...

Gene-editing techniques such as CRISPR-Cas9 have many uses in the area of food and agriculture. They can combat persistent drought and disease, and improve the colour and nutritional content of food ...

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 ...

Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...

For some diseases, gene therapies offer the potential for lifelong disease amelioration and even cure. And these immensely important novel biotechnologies may be on the cusp of a boom. That is in part ...

Researchers have identified critical spots on the genome where gene editing could cause an unwanted response, and they provide recommendations for safer approaches. CRISPR/Cas9 is a commonly used, ...

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 ...

NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...

A powerful form of DNA-editing machinery discovered in bacteria might allow us to make much bigger changes to genomes than is currently possible with CRISPR-based techniques. However, it isn’t yet ...