Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina

• मुख्य लेखकों: Juliette Pulman (लेखक), Catherine Botto (लेखक), Hugo Malki (लेखक), Duohao Ren (लेखक), Paul Oudin (लेखक), Anne De Cian (लेखक), Marie As (लेखक), Charlotte Izabelle (लेखक), Bruno Saubamea (लेखक), Valerie Forster (लेखक), Stéphane Fouquet (लेखक), Camille Robert (लेखक), Céline Portal (लेखक), Aziz El-Amraoui (लेखक), Sylvain Fisson (लेखक), Jean-Paul Concordet (लेखक), Deniz Dalkara (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2024-12-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Genome editing by CRISPR-Cas holds promise for the treatment of retinal dystrophies. For therapeutic gene editing, transient delivery of CRISPR-Cas9 is preferable to viral delivery which leads to long-term expression with potential adverse consequences. Cas9 protein and its guide RNA, delivered as r...