Priority 1 from the Liver Glycogen Storage Disease PSP

UNCERTAINTY: What are the best options (for example gene therapy or enzyme replacement therapy) for achieving sufficient amount of working enzyme in patients with liver Glycogen Storage Disease?  (JLA PSP Priority 1)
Overall ranking 1
JLA question ID 0090/1
Explanatory note

There are several option for "curing" patients with liver GSD, one of which (gene therapy) is currently under development. This question emphasizes that research into a cure for patients with liver GSD is the top priority. 


For details of the evidence checked, please see the spreadsheet held on the JLA website.

Health Research Classification System category Metabolic and endocrine 
Extra information provided by this PSP
Original uncertainty examples I´d like to know about can crisper cas technolgie could matter possibility of genetherapie for GSD? Who researches in this field? ~ With gene therapy and liver transplantation, are hypoglycaemias avoided and cornstarch dispensed or can they have a diet rich in lactose, fructose and sucrose? ~ realistic alternatives to AAV8 gene therapy. 
Submitted by Patient, carer and healthcare professionals
PSP information
PSP unique ID 0090
PSP name International Glycogen Storage Disease PSP
Total number of uncertainties identified by this PSP 72  (To see a full list of all uncertainties identified, please see the detailed spreadsheet held on the JLA website)
Date of priority setting workshop 24 May 2019