For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

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.

Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene ...

The US Food and Drug Administration released a draft guidance document on Tuesday with recommendations for using ...

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

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and ...

The ACC has published a scientific statement regarding the use of gene editing therapy for cardiovascular disease.

CRISPR Cas9 genome editing has transformed the way scientists approach gene therapy, acting like precise DNA scissors that can target and repair hereditary diseases at the genetic level. This ...

Scientists are laying the groundwork for treating one of the most common genetic conditions in humans. Research out today ...

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

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