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Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells.

Xu X, Tay Y, Sim B, Yoon SI, Huang Y, Ooi J, Utami KH, Ziaei A, Ng B, Radulescu C, Low D, Ng AY, Loh M, Venkatesh B, Ginhoux F, Augustine GJ, Pouladi MA
Stem Cell Reports. 2017 Feb 21. pii: S2213-6711(17)30038-3. doi: 10.1016/j.stemcr.2017.01.022. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
87200pX335 HTT sgRNA-bpX335 vector encoding SpCas9n and a chimeric guide RNA targeting 5' UTR of HTT
87201pX335 HTT sgRNA-apX335 vector encoding SpCas9n and a chimeric guide RNA targeting exon 1 of HTT
87228pJOP-HTT-HR18QHTT HR arms with 18 CAG repeats and piggyBac selection cassette

Antibodies from Article