New Era for Gene Therapy: Personalized CRISPR-Cas9 Treatment for CPS1 Deficiency in a Newborn
Discover the first personalized CRISPR-Cas9 gene therapy that precisely corrects CPS1 deficiency in a newborn via base editing A>G. Delivered by liver-targeted lipid nanoparticles, this rapid six-month development achieved durable genomic correction and normalized ammonia levels, opening a new era in safe, agile treatments for ultra-rare metabolic disorders.