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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Principais autores:	Bruna Lopes da Costa, Masha Kolesnikova, Sarah R. Levi, Thiago Cabral, Stephen H. Tsang, Irene H. Maumenee, Peter M. J. Quinn
格式:	Artigo
语言:	Inglês
出版:	MDPI AG 2023-01-01
丛编:	Biomedicines
主题:	prime editing base editing CRISPR <i>Crumbs homolog 1</i> (<i>CRB1</i>) inherited retinal disease (IRD) maculopathy
在线阅读:	https://www.mdpi.com/2227-9059/11/2/385
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