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 ...
NIH-funded UT team and colleagues from Metagenomi Thereapeutics discovered highly efficient enzyme could enable targeted gene editing within the human body.
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 new method relies on methyl groups, small chemical tags attached to DNA that regulate whether genes are on or off. This ...
The CRISPR-Cas gene-editing system has long been the focus of research as a promising tool in genome editing. However, the emphasis has been on its underlying mechanisms and nucleases. In contrast, ...
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...