Early initiation of ERT facilitated by newborn screening improves health outcomes among infantile Pompe disease patients in the US

Eleanor Rodriguez-Rassi, Grace Stafford,Seung-Hye Jung, Stephanie DeArmey,Ankit K. Desai,Priya Kishnani

Molecular Genetics and Metabolism(2023)

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摘要
In this work, Pure ZnO nanoparticles and a nanocomposite of PbO: ZnO were prepared by the flame synthesis method, and was analyzed for structural, morphological, optical, elemental, and biocompatibility studies. The structural analysis revealed a hexagonal structure for ZnO and an Orthorhombic structure for PbO: ZnO nanocomposite. Scanning electron microscopy (SEM) image showed a Nano-Sponge-like surface morphology for PbO: ZnO nanocomposite and energy dispersive spectra (EDS) confirmed the absence of undesired impurities. Transmission electron microscopy (TEM) image showed a particle size of ∼50 nm for ZnO and ∼20 nm for PbO: ZnO. Using Tauc plot the optical band gap was found to be 3.2 eV for ZnO and 2.9 eV for PbO: ZnO. Anticancer studies confirm the excellent cytotoxicity activity of both compounds. PbO: ZnO nanocomposite has demonstrated the highest cytotoxicity against the tumorigenic HEK 293 cell line with the lowest IC50 value of 13.04 μM. Our study shows that the prepared PbO: ZnO nanocomposite has a huge potential in cancer therapy.
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infantile pompe disease patients,newborn screening
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