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

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 SC, Huang Z, Jennings L, McLaren TL, Lamey TM, De Roach JN, Chen FKMcLenachan S.

Mol Genet Genomic Med. 2021 Jan 26:e1601. doi: 10.1002/mgg3.1601. Online ahead of print.

PMID: 33497524 Free article.

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