Viral vector injections of rAAV-∆FosB shRNA significantly reduced abnormal involuntary movements (AIMs) compared to control vector in a rat model of Parkinson’s disease.
Parkinson's disease (PD) is a progressive, debilitating, and currently incurable neurodegenerative disease. Long-term dopamine replacement therapy in PD leads to the development of abnormal involuntary movements known as L-DOPA–induced dyskinesia (LID). Alleviating this dyskinesia can lead to significant improvements in quality of life for patients with PD.
The transcription factor ∆FosB plays an important role in the development of dyskinesias and is a candidate target for gene therapy strategies to control LID. Scientists at Rutgers and Emory University have shown that lowering ∆FosB expression via gene silencing is an effective and feasible therapeutic approach to improve LID. Injection of a rAAV-∆FosB shRNA viral vector significantly reduced dyskinesia in a rodent PD model compared to controls. These effects were achieved while maintaining the antiparkinsonian action of L-DOPA, indicating that this gene therapy can successfully be used to treat dyskinesia in patients with advanced PD.
- Gene therapy
- Treatment for LID/severe dyskinesia
- Highly effective at improving LID
- Can be used in tandem with other PD drugs
Intellectual Property & Development Status:
Patent pending. Available for licensing and/or collaboration.