WDFY3 variant mouse models for neurodegenerative studies

This technology is a mouse strain that contains clinically relevant point mutations in the WDFY3 gene for studies on the role of autophagy in neurodegenerative diseases.

Unmet Need: Therapeutics for neurodegenerative diseases

Though there have been recent advances in therapies for neurodegenerative diseases, very few target the root causes of neurodegeneration and instead focus on symptom management. A major hurdle in the development of these therapeutics is understanding disease progression and protective mechanisms. More precise research tools are needed to elucidate disease mechanisms and identify therapeutic intervention targets.

The Technology: Knock-in mouse model for neuroprotective SNPs

This technology is a knock-in mouse model for a neuroprotective variant of the autophagy-related WDFY3 gene. This mouse model contains a point mutation in the WDFY3 locus, which has been identified in humans to confer neuroprotective effects in several neurodegenerative disorders, including Huntington’s disease, Parkinson’s disease, and tauopathies.

Applications:

  • Research tool for the development of neurodegenerative diseases, including Huntington’s disease, Parkinson’s disease, and tauopathies
  • Research tool for the role of autophagy in neurodegenerative disease progression
  • Research tool for the development of treatments for neurodegenerative diseases

Advantages:

  • Knock-in model
  • Clinically relevant (mutation identified in humans)
  • Relevant for several neurodegenerative diseases

Lead Inventor:

Ai Yamamoto, Ph.D.

Related Publications:

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