Article
Analysis of CRB1 Pathogenic Variants Correctable with CRISPR Base and Prime Editing.
Advances in experimental medicine and biology - 1 Jan 2023
da Costa Bruna Lopes, Jenny Laura A, Maumenee Irene H, Tsang Stephen H, Quinn Peter M J
Abstract excerpt
The mouse and human retina contain three major Crumbs homologue-1 (CRB1) isoforms. CRB1-A and CRB1-B have cell-type-specific expression patterns making the choice of gene augmentation strategy unclear. Gene editing may be a viable alternative for the amelioration of CRB1-associated retinal degenerations. To assess the prevalence and spectrum of CRB1-associated pathogenic variants amenable to base and prime...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.