Skoltech, MIT, UMass researchers develop gene-editing solution to liver diseases

Abstract

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Researchers from Skoltech, the Massachusetts Institute of Technology (MIT) and the University of Massachusetts (UMass) have developed novel nanoparticles that can deliver the CRISPR-Cas9 genome editing system and specifically modify genes directly within the liver of a mouse. Researchers were able to remove target genes in approximately 80% of hepatocytes – the best rate ever achieved with CRISPR in adult animals.