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Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration.
Wiley LA, Burnight ER, Drack AV, Banach BB, Ochoa D, Cranston CM, Madumba RA, East JS, Mullins RF, Stone EM, Tucker BA. Wiley LA, et al. Among authors: east js. Hum Gene Ther. 2016 Oct;27(10):835-846. doi: 10.1089/hum.2016.049. Epub 2016 Jul 11. Hum Gene Ther. 2016. PMID: 27400765 Free PMC article.