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Development of targeted therapies for peroxisome biogenesis disorders
- 负责人：
- 关键词：
- peroxins Molecular Biology peroxisome biogenesis disorders PEX1 high-content screening small molecules induced pluripotent stem cells HepG2 cells Pex1-p.G844D mouse
- DOI：
- doi:10.25549/usctheses-c40-298640
- 摘要：
- Peroxisome biogenesis disorders (PBDs) are a group of genetically heterogeneous rare metabolic diseases caused by defects in peroxins, proteins
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