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...
Main Authors: | , , , , , |
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Format: | Journal article |
Language: | English |
Published: |
Taylor and Francis
2022
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