scholarly journals Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia

Gene Therapy ◽  
2011 ◽  
Vol 19 (4) ◽  
pp. 443-452 ◽  
Author(s):  
B Crane ◽  
X Luo ◽  
A Demaster ◽  
K D Williams ◽  
D M Kozink ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Adenovirus Vector ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Term Efficacy ◽  
Type Ia

scholarly journals Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

2012 ◽  
Vol 23 (4) ◽  
pp. 407-418 ◽  
Author(s):  
Amanda Demaster ◽  
Xiaoyan Luo ◽  
Sarah Curtis ◽  
Kyha D. Williams ◽  
Dustin J. Landau ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Virus Vector ◽  
Adeno Associated Virus ◽  
Term Efficacy ◽  
Type Ia

scholarly journals Glycogen Storage Disease Type Ia: Current Management Options, Burden and Unmet Needs

Nutrients ◽  
2021 ◽  
Vol 13 (11) ◽  
pp. 3828
Author(s):  
Terry G. J. Derks ◽  
David F. Rodriguez-Buritica ◽  
Ayesha Ahmad ◽  
Foekje de Boer ◽  
María L. Couce ◽  
...  
Keyword(s):  
Metabolic Control ◽  
Glycogen Storage Disease ◽  
Psychosocial Development ◽  
Storage Disease ◽  
Dietary Treatment ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Type Ia

Glycogen storage disease type Ia (GSDIa) is caused by defective glucose-6-phosphatase, a key enzyme in carbohydrate metabolism. Affected individuals cannot release glucose during fasting and accumulate excess glycogen and fat in the liver and kidney, putting them at risk of severe hypoglycaemia and secondary metabolic perturbations. Good glycaemic/metabolic control through strict dietary treatment and regular doses of uncooked cornstarch (UCCS) is essential for preventing hypoglycaemia and long-term complications. Dietary treatment has improved the prognosis for patients with GSDIa; however, the disease itself, its management and monitoring have significant physical, psychological and psychosocial burden on individuals and parents/caregivers. Hypoglycaemia risk persists if a single dose of UCCS is delayed/missed or in cases of gastrointestinal intolerance. UCCS therapy is imprecise, does not treat the cause of disease, may trigger secondary metabolic manifestations and may not prevent long-term complications. We review the importance of and challenges associated with achieving good glycaemic/metabolic control in individuals with GSDIa and how this should be balanced with age-specific psychosocial development towards independence, management of anxiety and preservation of quality of life (QoL). The unmet need for treatment strategies that address the cause of disease, restore glucose homeostasis, reduce the risk of hypoglycaemia/secondary metabolic perturbations and improve QoL is also discussed.

scholarly journals Corrigendum to “Efficacy of Helper-dependent Adenovirus Vector-mediated Gene Therapy in Murine Glycogen Storage Disease Type Ia”

2007 ◽  
Vol 15 (11) ◽  
pp. 2051
Author(s):  
Dwight D Koeberl ◽  
Baodong Sun ◽  
Andrew Bird ◽  
Yuan-Tsong Chen ◽  
Kazuhiro Oka ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Adenovirus Vector ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Type Ia
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