Multiple Approaches to Novel GSD Ia Therapies

<p>Glycogen storage disease type Ia is an autosomal recessive disorder caused by a mutation in the glucose-6-phosphatase (G6Pase) catalytic subunit, encoded in humans by G6PC. G6Pase dephosphorylates glucose-6-phosphate (G6P) in the liver to generate glucose that can be shuttled to the bloodst...

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Bibliographic Details
Main Author: Landau, Dustin James
Other Authors: Koeberl, Dwight D
Published: 2016
Subjects:
AAV
ZFN
Online Access:http://hdl.handle.net/10161/13358