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Showing 1–4 of 4 results
Advanced filters: Author: Sajeev Kohli Clear advanced filters

  • Two approaches using an adenine base editor and a Cas9 nuclease prevented the development of hypertrophic cardiomyopathy in mice carrying a pathogenic mutation on the Myh6 gene, highlighting the potential of single-dose genetic therapies for the treatment of cardiac disease.

    • Daniel Reichart
    • Gregory A. Newby
    • Christine Seidman
    ResearchOpen Access
    Nature Medicine
    Volume: 29, P: 412-421

  • Gene editing strategies for cystic fibrosis are challenging. Here the authors improve on their previously reported shuttle peptide noncovalently combined with Cas ribonucleoprotein (RNP), and derive the S315 peptide for delivery: they show base editing in the respiratory tract of the rhesus macaques.

    • Katarina Kulhankova
    • Soumba Traore
    • Paul B. McCray Jr.
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-16