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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 great interest to evaluate gene edi...

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Wedi'i Gadw mewn:
Prif Awduron: Bruna Lopes da Costa, Masha Kolesnikova, Sarah R. Levi, Thiago Cabral, Stephen H. Tsang, Irene H. Maumenee, Peter M. J. Quinn
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: MDPI AG 2023-01-01
Cyfres:Biomedicines
Pynciau:
prime editing
base editing
CRISPR
<i>Crumbs homolog 1</i> (<i>CRB1</i>)
inherited retinal disease (IRD)
maculopathy
Mynediad Ar-lein:https://www.mdpi.com/2227-9059/11/2/385
Tagiau: Ychwanegu Tag
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