MedicinefromNature5 days agoThe baby whose life was saved by the first personalized CRISPR therapyA personalized CRISPR base-editing therapy corrected a newborn's CPS1-deficiency mutation, offering a tailored alternative to liver transplant for an ultra-rare genetic disorder.
Medicinefromwww.nature.com1 month agoGene Editing Helped One BabyCould It Help Thousands?Personalized base-editing therapy cured baby KJ; a clinical trial plans to deliver multiple similarly tailored treatments faster after negotiating simplified regulatory pathways.
SciencefromNature1 month agoDaily briefing: Custom-made gene-editing therapy for children to enter clinical trialPersonalized CRISPR base-editing therapy that rescued an infant will enter a clinical trial for at least five more children.