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

• Päätekijät: Kai Chen (Tekijä), Na Zhang (Tekijä), Jing-Bo Shao (Tekijä), Hong Li (Tekijä), Jie Li (Tekijä), Jia-Ming Xi (Tekijä), Wu-Hen Xu (Tekijä), Hui Jiang (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: Frontiers Media S.A., 2021-08-01T00:00:00Z.
• Aiheet: article
• Linkit: 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...