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Modulation of Protein Accumulation and Uses Therefore

总结

Modulation of protein accumulation in Motor Neurone Disease

技术优势

Endogenous method for directly controlling TDP-43 ubiquitylation.
Utilisation of endogenous pathway within neurons (ie low chance of off-target side-effects).
In-vivo data demonstrated that viral-mediated overexpression of cyclin F in mice is safe.
The first group to identify disease-causing mutations in Cyclin F in MND and FTD patients.
Significant expertise, know-how and access to valuable research tools required to develop this technology further.

技术应用

Delivery of Cyclin F to neurons
Manipulating Cyclin F expression in neurons to: correct the ubiquitylation status of TDP43 and; improve the clearance of TDP43

详细技术说明

Identified the first disease-associated E3 ubiquitin ligase (Cyclin F) that directly targets TDP43 for ubiquitylation. Abnormal ubiquitination of TDP43 contributes to its aggregation. Accordingly we find that disease-causing mutations in Cyclin F cause hyper ubiquitylation of TDP-43 and neuronal death.
Therefore we believe that over-expressing Cyclin F can prevent or slow down disease progression by modifying the function of TDP-43.

合作类型

Licensing or
Research Collaboration

申请号码

PCT/2017/051225

ID号码

2016025

国家/地区

澳洲