Clinical and Therapeutic Evaluation of the Ten Most Prevalent <i>CRB1</i> Mutations

Clinical and Therapeutic Evaluation of the Ten Most Prevalent <i>CRB1</i> Mutations

Mutations in the <i>Crumbs homolog 1</i> (<i>CRB1</i>) gene lead to severe inherited retinal dystrophies (IRDs), accounting for nearly 80,000 cases worldwide. To date, there is no therapeutic option for patients suffering from <i>CRB1</i>-IRDs. Therefore, it is of...

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Bibliographic Details
Main Authors: Bruna Lopes da Costa, Masha Kolesnikova, Sarah R. Levi, Thiago Cabral, Stephen H. Tsang, Irene H. Maumenee, Peter M. J. Quinn
Format: Article
Language:English
Published: MDPI AG 2023-01-01
Series:Biomedicines
Subjects:
prime editing
base editing
CRISPR
<i>Crumbs homolog 1</i> (<i>CRB1</i>)
inherited retinal disease (IRD)
maculopathy
Online Access:https://www.mdpi.com/2227-9059/11/2/385

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https://www.mdpi.com/2227-9059/11/2/385

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