Disease modeling and lentiviral gene transfer in patient-specific induced pluripotent stem cells from late-onset Pompe disease patient
Pompe disease is an autosomal recessive inherited metabolic disease caused by deficiency of acid α-glucosidase (GAA). Glycogen accumulation is seen in the affected organ such as skeletal muscle, heart, and liver. Hypertrophic cardiomyopathy is frequently seen in the infantile onset Pompe disease. O...
Main Authors: | Yohei Sato, Hiroshi Kobayashi, Takashi Higuchi, Yohta Shimada, Takumi Era, Shigemi Kimura, Yoshikatsu Eto, Hiroyuki Ida, Toya Ohashi |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2015-01-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050116300353 |
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