Litcius/Paper detail

Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia

Seyedeh Zeinab Mirjalili Mohanna, Jack W. Hickmott, Siu Ling Lam, Nina Y. Chiu, Tess C. Lengyell, Beatrice M. Tam, Orson L. Moritz, Elizabeth M. Simpson

2020Molecular Therapy — Methods & Clinical Development31 citationsDOIOpen Access PDF

Abstract

mutation alone rescues the mutant phenotype, and developed an allele-distinguishing CRISPR-based strategy for aniridia.

Topics & Concepts

CRISPRAniridiaGermlineGenome editingGeneticsBiologyCas9PAX6GeneTranscription factorCRISPR and Genetic EngineeringRetinal Development and DisordersGenetics, Aging, and Longevity in Model Organisms