The gene therapy for corneal pathology with novel nonsense cystinosis mouse lines created by CRISPR Gene Editing

Fei Dong,Hassane Amlal,Jhuwala Venkatakrishnan,Jianhua Zhang, Matthew Fry,Yong Yuan, Yu Chia Cheng,Yueh-Chiang Hu, Winston W-Y Kao

The Ocular Surface（2023）

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摘要

Both Ctnsis1 and Ctnsis2 mice exhibit phenotypes resembling human intermediate nephropathic and ocular cystinosis, respectively. scAAV-CTNS viral vectors reduce the corneal cystine crystals and have a great potential as a therapeutic strategy for treating patients suffering from cystinosis.

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关键词

crispr gene,corneal pathology,gene therapy

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