Most gene-editing tools are blind to context. Point them at a DNA sequence and they cut, whether that sequence sits inside a ...

Scientists have taken an important step toward a gene therapy that could one day turn off the extra genetic material that ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

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 ...

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 ...

A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

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 ...

The CRISPR-Cas gene-editing system has long been the focus of research as a promising tool in genome editing. However, the ...