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Journal: Molecular therapy. Methods & clinical development
Article Title: Design and validation of cell-based potency assays for frataxin supplementation treatments.
doi: 10.1016/j.omtm.2024.101347
Figure Lengend Snippet: Figure 3. Stable expression of exogenous human FXN in MUT MEFs (A) A schematic of the lentivirus vector pLenti hygro FXN encoding a human miniFXN gene. The miniFXN gene used in experiments contains fragments of the endogenous FXN 50UTR, promoter and intron 1, and all five exons of the gene. (B) Representative western blot demonstrating expression of exogenous FXN. The miniFXN encodes a C-terminal HA tag that results in a slightly greater molecular weight, as detected by western blot. (C) Quantitation of endogenous Fxn (*) and exogenous FXN by western blot. Gapdh was used as a normalization control. MUT+FXN designates cells expressing exogenous, human FXN. The experiment was performed three independent times. Results are plotted as mean values with standard deviations; (**p < 0.001, ***p < 0.0001).
Article Snippet: Cloning and packaging of pLenti hygro FXN-L A miniFXN construct expressing human FXN under the control of endogenous regulatory elements was cloned into the
Techniques: Expressing, Plasmid Preparation, Western Blot, Molecular Weight, Quantitation Assay, Control