Gene therapy and genome editing for type I glycogen storage diseases
Type I glycogen storage diseases (GSD-I) consist of two major autosomal recessive disorders, GSD-Ia, caused by a reduction of glucose-6-phosphatase-α (G6Pase-α or G6PC) activity and GSD-Ib, caused by a reduction in the glucose-6-phosphate transporter (G6PT or SLC37A4) activity. The G6Pase-α and G6PT...
Main Authors: | , |
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Format: | Article |
Language: | English |
Published: |
Frontiers Media S.A.
2023-03-01
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Series: | Frontiers in Molecular Medicine |
Subjects: | |
Online Access: | https://www.frontiersin.org/articles/10.3389/fmmed.2023.1167091/full |