Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery

by Biotech Newsroom


  • Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-Thalassemia. N Engl J Med. 2021;384:252–60.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, et al. Patient-specific in vivo gene editing to treat a rare genetic disease. N Engl J Med. 2025;392:2235–43.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Jinek M,…



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