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

• Main Authors: Juliette Pulman (Author), Catherine Botto (Author), Hugo Malki (Author), Duohao Ren (Author), Paul Oudin (Author), Anne De Cian (Author), Marie As (Author), Charlotte Izabelle (Author), Bruno Saubamea (Author), Valerie Forster (Author), Stéphane Fouquet (Author), Camille Robert (Author), Céline Portal (Author), Aziz El-Amraoui (Author), Sylvain Fisson (Author), Jean-Paul Concordet (Author), Deniz Dalkara (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: 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...