scholarly journals Letter to the Editors: Concerning “Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia” by Lee et al.

2018 ◽  
Vol 41 (6) ◽  
pp. 913-914 ◽  
Author(s):  
Elizabeth D. Brooks ◽  
Priya S. Kishnani ◽  
Dwight D. Koeberl
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Safety And Efficacy ◽  
Type Ia

Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia

2018 ◽  
Vol 41 (6) ◽  
pp. 977-984 ◽  
Author(s):  
Young Mok Lee ◽  
Thomas J. Conlon ◽  
Andrew Specht ◽  
Kirsten E. Coleman ◽  
Laurie M. Brown ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Safety And Efficacy ◽  
Type Ia

scholarly journals Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia

2020 ◽  
Vol 527 (3) ◽  
pp. 824-830
Author(s):  
Lisa Zhang ◽  
Cheol Lee ◽  
Irina Arnaoutova ◽  
Javier Anduaga ◽  
Matthew F. Starost ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Term Efficacy ◽  
Type Ia

Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia

1999 ◽  
Vol 22 (6) ◽  
pp. 723-732 ◽  
Author(s):  
L. Faivre ◽  
D. Houssin ◽  
J. Valayer ◽  
J. Brouard ◽  
M. Hadchouel ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Type Ia

scholarly journals Downregulation of pathways implicated in liver inflammation and tumorigenesis of glycogen storage disease type Ia mice receiving gene therapy

2017 ◽  
Vol 26 (10) ◽  
pp. 1890-1899 ◽  
Author(s):  
Goo-Young Kim ◽  
Joon Hyun Kwon ◽  
Jun-Ho Cho ◽  
Lisa Zhang ◽  
Brian C. Mansfield ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Liver Inflammation ◽  
Type Ia

scholarly journals Adeno-Associated Virus-Mediated Correction of a Canine Model of Glycogen Storage Disease Type Ia

2010 ◽  
Vol 21 (7) ◽  
pp. 903-910 ◽  
Author(s):  
David A. Weinstein ◽  
Catherine E. Correia ◽  
Thomas Conlon ◽  
Andrew Specht ◽  
John Verstegen ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Adeno Associated Virus ◽  
Type Ia

scholarly journals Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy

Hepatology ◽  
2012 ◽  
Vol 56 (5) ◽  
pp. 1719-1729 ◽  
Author(s):  
Young Mok Lee ◽  
Hyun Sik Jun ◽  
Chi-Jiunn Pan ◽  
Su Ru Lin ◽  
Lane H. Wilson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Hepatocellular Adenoma ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Metabolic Abnormalities ◽  
Type Ia

scholarly journals Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia

Gene Therapy ◽  
2007 ◽  
Vol 14 (3) ◽  
pp. 281-281
Author(s):  
D D Koeberl ◽  
B D Sun ◽  
T V Damodaran ◽  
T Brown ◽  
D S Millington ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Virus Vector ◽  
Adeno Associated Virus ◽  
Sustained 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.

Sign in / Sign up

Export Citation Format

Share Document