Allogeneic Hematopoietic Stem Cell Transplantation for PEX1-Related Zellweger Spectrum Disorder: A Case Report and Literature Review

• Main Authors: Kai Chen (Author), Na Zhang (Author), Jing-Bo Shao (Author), Hong Li (Author), Jie Li (Author), Jia-Ming Xi (Author), Wu-Hen Xu (Author), Hui Jiang (Author)
• Format: Knjiga
• Izdano: Frontiers Media S.A., 2021-08-01T00:00:00Z.
• Teme: article
• Online dostop: Connect to this object online.

Zellweger spectrum disorder (ZSD) is a heterogeneous group of autosomal recessive disorders characterized by a defect in peroxisome formation and attributable to mutations in the PEX gene family. Patients with ZSD have profound neurologic impairments, including seizures, severe retardation, and dysm...