Ontology highlight
ABSTRACT:
SUBMITTER: Mirjalili Mohanna SZ
PROVIDER: S-EPMC7114625 | biostudies-literature | 2020 Jun
REPOSITORIES: biostudies-literature
Mirjalili Mohanna Seyedeh Zeinab SZ Hickmott Jack W JW Lam Siu Ling SL Chiu Nina Y NY Lengyell Tess C TC Tam Beatrice M BM Moritz Orson L OL Simpson Elizabeth M EM
Molecular therapy. Methods & clinical development 20200314
Aniridia is a rare eye disorder, which is caused by mutations in the paired box 6 (<i>PAX6</i>) gene and results in vision loss due to the lack of a long-term vision-saving therapy. One potential approach to treating aniridia is targeted CRISPR-based genome editing. To enable the <i>Pax6</i> small eye (<i>Sey</i>) mouse model of aniridia, which carries the same mutation found in patients, for preclinical testing of CRISPR-based therapeutic approaches, we endogenously tagged the <i>Sey</i> allele ...[more]