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CRISPR/Cas9-mediated suppression of A4GALT rescues endothelial cell dysfunction in a Fabry disease vasculopathy model derived from human induced pluripotent stem cells

2023-09-12

Abstract excerpt

<title>Abstract</title> <p><bold>Backgrounds</bold>: The objective of this study was to investigate the efficacy of CRISPR/Cas9-mediated <italic>A4GALT</italic> suppression in rescuing endothelial dysfunction in Fabry disease (FD) endothelial cells (FD-ECs) derived from human induced pluripotent stem cells (hiPSCs). <bold>Methods</bold>: We differentiated hiPSCs (WT (wild-type), WTC-11), <italic>GLA</italic>-muta...

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Literature Corpus work
1615a1e5-17b0-5588-9737-5e3183eb7713
DOI
10.21203/rs.3.rs-3264880/v1
Open publication

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CRISPR/Cas9-mediated suppression of A4GALT rescues endothelial cell dysfunction in a Fabry disease vasculopathy model derived from human induced pluripotent stem cellsDOI 10.21203/rs.3.rs-3264880/v1
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