Researchers modified AlphaFold AI to identify and alter key areas of the Cas9 protein in gene-editing systems, significantly reducing off-target effects from 28% to 5% through targeted amino acid swaps.
Starlet sea anemones use specialized mobile nematosomes and the cJun gene to selectively identify and destroy harmful bacteria while preserving beneficial microbes, challenging the belief that targeted immunity is exclusive to vertebrates' adaptive immune systems and pushing back its evolutionary timeline.
Researchers from Heinrich Heine University Düsseldorf and Kiel University discovered that the starlet sea anemone, Nematostella vectensis, uses specialized mobile cell structures called nematosomes to selectively identify and destroy harmful bacteria while preserving beneficial ones. This challenges the long-held assumption that targeted immune discrimination requires the complex adaptive immune system of vertebrates, pushing back the evolutionary timeline for such capabilities by hundreds of millions of years.
Scientists at ShanghaiTech University have developed CS-101, a base-editing gene therapy that successfully cured five Chinese patients with beta-thalassaemia. The treatment restored haematopoietic function, raising total haemoglobin and fetal haemoglobin levels, freeing patients from lifelong blood transfusions. China has 30 million thalassaemia gene carriers while the global total reaches 350 million.
Intellia Therapeutics' CRISPR-based treatment for hereditary angioedema met its goals in a Phase 3 trial, reducing attacks by 87% compared with placebo. The one-time infusion therapy edits genes directly in the liver and achieved 62% of patients being attack-free six months after treatment. The company has started a rolling FDA application and plans to launch in the first half of next year if approved.
