Priority 12 from the Liver Glycogen Storage Disease (International) PSP

UNCERTAINTY: What are the risks and benefits of gene therapy for patients with liver Glycogen Storage Disease?  (JLA PSP Priority 12)
Overall ranking 12
JLA question ID 0090/12
Explanatory note

A gene therapy trial for GSD Ia is currently being performed and gene therapy is being developped for other types of liver GSD. This question stresses that it is important to evaluate short-term and long-term risks and benefits of this new treatment option. 


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 What side effects will gene therapy have? ~ Long-term studies - stem cells / 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