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    • About Wolfram Syndrome
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    • Treatment Pipeline
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    • Published WS Research - 2020
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    • For Clinicians
    • Complete Wolfram Syndrome Clinical Guidelines >
      • Clinical Features of WS
      • Clinical Diagnosis/Baseline Investigation
      • Clinical Management of WS/Treatment Options
      • Genetic Diagnosis of WS-Requisition
    • List of WS Clinicians
    • Add a Clinician
    • Additional Clinician Resources
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    • Patient Information Links
    • What is Wolfram Syndrome
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  • Contact WSRA/More Info
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    • PDF Files for download
WSRA
  • Home
    • About WSRA
    • About Wolfram Syndrome
    • WSRA Advisory Board
    • Upcoming WS Events
  • Research
    • Treatment Strategies
    • Treatment Pipeline
    • WS Research Groups
    • Clinical Trials
    • Patient Registries
    • WU Wolfram Research Clinic
    • Published WS Research - 2020
    • WS Research Library
  • For Researchers
    • For Researchers
    • WS Research Forum
    • WS Research Groups
  • For Clinicians
    • For Clinicians
    • Complete Wolfram Syndrome Clinical Guidelines >
      • Clinical Features of WS
      • Clinical Diagnosis/Baseline Investigation
      • Clinical Management of WS/Treatment Options
      • Genetic Diagnosis of WS-Requisition
    • List of WS Clinicians
    • Add a Clinician
    • Additional Clinician Resources
    • WS Research Library
  • For Patients
    • Patient Information Links
    • What is Wolfram Syndrome
    • WS Foundations
    • Clinicians Treating WS
    • Patient Registries
    • Clinical Trials
  • Donate to WS Research
    • Foundations Supporting WS Research
    • Call for WS Project Proposals
  • Contact WSRA/More Info
    • Contact WSRA
    • PDF Files for download

Treatment Strategies

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
View Treatment pipeline
Treatment strategies applied at various points along the pathway from DNA to cellular dysfunction
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  • Home
    • About WSRA
    • About Wolfram Syndrome
    • WSRA Advisory Board
    • Upcoming WS Events
  • Research
    • Treatment Strategies
    • Treatment Pipeline
    • WS Research Groups
    • Clinical Trials
    • Patient Registries
    • WU Wolfram Research Clinic
    • Published WS Research - 2020
    • WS Research Library
  • For Researchers
    • For Researchers
    • WS Research Forum
    • WS Research Groups
  • For Clinicians
    • For Clinicians
    • Complete Wolfram Syndrome Clinical Guidelines >
      • Clinical Features of WS
      • Clinical Diagnosis/Baseline Investigation
      • Clinical Management of WS/Treatment Options
      • Genetic Diagnosis of WS-Requisition
    • List of WS Clinicians
    • Add a Clinician
    • Additional Clinician Resources
    • WS Research Library
  • For Patients
    • Patient Information Links
    • What is Wolfram Syndrome
    • WS Foundations
    • Clinicians Treating WS
    • Patient Registries
    • Clinical Trials
  • Donate to WS Research
    • Foundations Supporting WS Research
    • Call for WS Project Proposals
  • Contact WSRA/More Info
    • Contact WSRA
    • PDF Files for download