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Direct Delivery of Cas9 or Base Editor Protein and Guide RNA Complex Enables Genome Editing in the Retina

Juliette Pulman,Catherine Botto,Hugo Malki,Duohao Ren, Paul Oudin,Anne De Cian, Marie As,Charlotte Izabelle,Bruno Saubamea,Valerie Forster,Stephane Fouquet,Camille Robert,Aziz El-Amraoui,Sylvain Fisson,Jean-Paul Concordet,Deniz Dalkara

MOLECULAR THERAPY NUCLEIC ACIDS（2024）

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关键词
MT: Delivery Strategies,gene therapy,gene editing,retina,CRISPR/Cas9,base editor,photoreceptor,RPE,inherited retinal dystrophy
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