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

Mutations in the Crumbs homolog 1 (CRB1) 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 a therapeutic approach. This retrospec...

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Detalles Bibliográficos
Autores principales: Bellingrath, Julia-Sophia, McClements, Michelle E., Kaukonen, Maria, Fischer, Manuel Dominik, MacLaren, Robert E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8700976/
https://www.ncbi.nlm.nih.gov/pubmed/34946856
http://dx.doi.org/10.3390/genes12121908

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8700976/
https://www.ncbi.nlm.nih.gov/pubmed/34946856
http://dx.doi.org/10.3390/genes12121908

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