Envisioning the development of a CRISPR-Cas mediated base editing strategy for a patient with a novel pathogenic CRB1 single nucleotide variant

<p><strong>Background</strong><br> Inherited retinal degeneration (IRD) associated with mutations in the Crumbs homolog 1 (CRB1) gene is associated with a severe, early-onset retinal degeneration for which no therapy currently exists. Base editing, with its capability to prec...

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Bibliographic Details
Main Authors: Bellingrath, J-S, McClements, ME, Shanks, M, Clouston, P, Fischer, MD, MacLaren, RE
Format: Journal article
Language:English
Published: Taylor and Francis 2022