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Using patient-specific induced pluripotent stem cells to interrogate the pathogenicity of a novel retinal pigment epithelium-specific 65 kDa cryptic splice site mutation and confirm eligibility for enrollment into a clinical gene augmentation trial.
Tucker BA, Cranston CM, Anfinson KA, Shrestha S, Streb LM, Leon A, Mullins RF, Stone EM. Tucker BA, et al. Transl Res. 2015 Dec;166(6):740-749.e1. doi: 10.1016/j.trsl.2015.08.007. Epub 2015 Aug 29. Transl Res. 2015. PMID: 26364624 Free PMC article.
Connective Tissue Growth Factor Promotes Efficient Generation of Human Induced Pluripotent Stem Cell-Derived Choroidal Endothelium.
Songstad AE, Worthington KS, Chirco KR, Giacalone JC, Whitmore SS, Anfinson KR, Ochoa D, Cranston CM, Riker MJ, Neiman M, Stone EM, Mullins RF, Tucker BA. Songstad AE, et al. Among authors: cranston cm. Stem Cells Transl Med. 2017 Jun;6(6):1533-1546. doi: 10.1002/sctm.16-0399. Epub 2017 May 5. Stem Cells Transl Med. 2017. PMID: 28474838 Free PMC article.
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa.
Tucker BA, Burnight ER, Cranston CM, Ulferts MJ, Luse MA, Westfall T, Scott CA, Marsden A, Gibson-Corley K, Wiley LA, Han IC, Slusarski DC, Mullins RF, Stone EM. Tucker BA, et al. Among authors: cranston cm. Gene Ther. 2022 May;29(5):259-288. doi: 10.1038/s41434-021-00291-5. Epub 2021 Sep 14. Gene Ther. 2022. PMID: 34518651 Free PMC article.
39 results