Development of targeted therapies for peroxisome biogenesis disorders

负责人：

关键词：

PEX1 Molecular Biology peroxisome biogenesis disorders peroxins high-content screening small molecules induced pluripotent stem cells HepG2 cells Pex1-p.G844D mouse

DOI：

doi:10.25549/usctheses-c40-298640

摘要：

in the characterization of the Pex1-p.G844D mouse which is the first mouse model with hypomorphic PEX alleles and thus better disease model for PBD patients with milder