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.

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

A research team has discovered an enhanced CRISPR gene-editing system that could enable targeted delivery inside the human ...

The US Food and Drug Administration (FDA) today issued a draft guidance for sponsors seeking approval of human gene therapy ...

NIH-funded UT team and colleagues from Metagenomi Thereapeutics discovered highly efficient enzyme could enable targeted gene editing within the human body.

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

CRISPR Cas9 gene therapy explained with DNA scissors, hereditary diseases treatment, and designer babies ethical dilemmas ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing ...

In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...

A multidisciplinary committee of experts studied the scientific underpinnings of human gene-editing technologies, their potential use in biomedical research and medicine -- including human germline ...