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.
Pythia: An AI Tool for Precise and Predictable Genomic Integrations via CRISPR Microhomologies
Predictable AI-guided gene editing: deep learning–optimized microhomologies enable precise transgene integration in human cells, embryos, and adult neurons, surpassing conventional methods.
OpenCRISPR-1: Designing Next-Generation Genome Editors via Artificial Intelligence
An innovative artificial intelligence approach enables the design of more efficient CRISPR–Cas9 editors
EvoCAST: Revolution in Programmed Gene Integration Through the CRISPR-CAST System
A new CRISPR tool, evoCasT, enables the insertion of large DNA fragments into the genome with unprecedented precision, paving the way for major advances in gene therapy and biotechnology.