Comparative Efficacy of AAV-Tafazzin and AAV-Δ5 Tafazzin in Restoring Mitochondrial Function in Barth Syndrome Induced Pluripotent Stem Cell Derived Cardiomyocytes

Description

We compared efficacy and efficiency of two AAV-Tafazzin variants AAV-CoTAZ (full length) and AAV-Δ5 (exon 5 removed) in Barth syndrome iPSC cardiomyocytes. We performed AAV transduction in iPSC cardiomyocytes and cultured cells for 7 days followed by an array of mitochondrial functional assays and gene expression analysis. The raw data from MTT, ATP, ROS, TMRE, and OCR assays and the qRT-PCR assay for all healthy and BTHS cell lines. Mitochondrial assays are normalized to 1ug of protein for all assays. We hypothesized that the AAV delivery of tafazzin improves mitochondrial function in BTHS iPSC cardiomyocytes and that Δ5 variant performs superior as it is abundant in heart and skeletal muscle.

Institutions

• University of Minnesota

  Minnesota, Minneapolis

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