Stop Progression of the 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
• Repurposed drugs that result in proliferation
• iPSC cell replacement

Correct the Gene Defect

Gene therapy 

• CRISPR cas9
• AAV WT WFS1 rescue

View Treatment pipeline

Treatment strategies applied along the pathway
from DNA to Cellular Dysfunction