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Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset Pompe disease patient fibroblasts

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NIAID Data Ecosystem2026-03-12 收录
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human Pompe Disease (c.-32-13T>G) fibroblasts were exposed to 10 µM Deferoxamine (DFX) or 0.1% DMSO (control condition), for three days. Three DMSO (CTR01Fib, CTR02Fib, CTR03Fib) and Deferoxamine (DFX04, DFX05, DFX06) independent treatments.

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2021-01-19
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