Priority 5 from the Mitochondrial Disease PSP
|UNCERTAINTY: Could gene therapy help people with mitochondrial disease? (JLA PSP Priority 5)
|JLA question ID
|Health Research Classification System category
|Metabolic and Endocrine
|Extra information provided by this PSP
|Original uncertainty examples
|Role of gene therapy ~ I’d like to see treatment that effectively shift heteroplasmy level for mtDNA patients ~ what treatment is there and also we were told of gene removal for going forward if we had p any more plans ~ Development of current treatment options (eg idebenone), other drug treatments, and genetic therapy possibilities. ~ What are the best medicines available. Will gene therapy ever be an option.~ how enzyme replacement therapies, ASO, and gene therapies can help mito patients. ~
There is no coordinated approach to developing new treatments, and more importantly in developing robust evidence for efficacy. If the NHS is to fund any new treatments this evidence base is required. Disease-modifying and disease-correcting therapies are required, and focus on gene therapy/ gene correction treatments should be a priority.
|4 x Healthcare Professionals, 6 x Carers, 3 x Patients
|PSP unique ID
|Total number of uncertainties identified by this PSP.
|42 (To see a full list of all uncertainties identified, please see the detailed spreadsheet held on the JLA website)
|Date of priority setting workshop
|19 January 2020