Gene correction of the CLN3 c.175G>A variant in patient-derived induced pluripotent stem cells prevents pathological changes in retinal organoids.

Zhang X, Zhang D, Thompson JA, Chen S-C, Huang Z, Jennings L, McLaren TL, Lamey TM, De Roach JN, Chen FK, et al. Gene correction of the CLN3 c.175G>A variant in patient-derived induced pluripotent stem cells prevents pathological changes in retinal organoids. Mol Genet Genomic Med. 2021:e1601.