A Liver Model of Infantile-Onset Pompe Disease Using Patient-Specific Induced Pluripotent Stem Cells

A Liver Model of Infantile-Onset Pompe Disease Using Patient-Specific Induced Pluripotent Stem Cells

Infantile-onset Pompe disease (IOPD) is a life-threatening multi-organ disease caused by an inborn defect of lysosomal acid α-glucosidase (GAA), which can degrade glycogen into glucose. Lack of GAA causes abnormal accumulation of glycogen in the lysosomes, particularly in the skeletal muscle, liver,...

Full description

Bibliographic Details
Main Authors: Takeshi Yoshida, Tatsuya Jonouchi, Kenji Osafune, Junko Takita, Hidetoshi Sakurai
Format: Article
Language:English
Published: Frontiers Media S.A. 2019-11-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
infantile-onset Pompe disease
iPS cell
enzyme replacement therapy
liver
disease modeling
Online Access:https://www.frontiersin.org/article/10.3389/fcell.2019.00316/full

Internet

https://www.frontiersin.org/article/10.3389/fcell.2019.00316/full

Similar Items