In Silico Analysis of Pathogenic <i>CRB1</i> Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention

In Silico Analysis of Pathogenic <i>CRB1</i> Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention

Mutations in the <i>Crumbs homolog 1</i> (<i>CRB1</i>) gene cause both autosomal recessive retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). Since three separate CRB1 isoforms are expressed at meaningful levels in the human retina, base editing shows promise as...

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Bibliographic Details
Main Authors: Julia-Sophia Bellingrath, Michelle E. McClements, Maria Kaukonen, Manuel Dominik Fischer, Robert E. MacLaren
Format: Article
Language:English
Published: MDPI AG 2021-11-01
Series:Genes
Subjects:
<i>CRB1</i>
inherited retinal disease
genetics
base editing
CRISPR
Online Access:https://www.mdpi.com/2073-4425/12/12/1908

Internet

https://www.mdpi.com/2073-4425/12/12/1908

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