Current Treatment Strategies
Stop Progression of Disease
Current research strategies include the use of -
- Chemical chaperones/molecular prosthetics
- ER calcium stabilizers
- ER stress reducers
Repair Damaged Tissue
Regenerative treatments
- AAV delivery of MANF
- Repurposed drugs that result in proliferation
- iPSC replacement
Correct the Gene Defect
Gene therapy
- CRISPR cas9
- AAV WT WFS1 rescue
Treatment strategies applied at various points along the pathway from DNA to cellular dysfunction
