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Funded Studies

Reprogramming the Brain’s Non-neuronal Support Cells to Generate Functional Dopamine-producing Neurons

Study Rationale:
We have recently demonstrated that a method for converting supportive, non-neuronal brain cells into functional dopamine-producing neurons leads to the complete reversal of symptoms in a pre-clinical model of Parkinson’s disease (PD). This proposal is designed to test our strategy in a pre-clinical model of PD. Given that the pre-clinical brain is more closely related to the human brain, this approach should pave the way for clinical trials in people with PD.

Hypothesis:
We hypothesize that non-neuronal cells in the pre-clinical brain can also be converted into dopamine-producing neurons and that the resulting restoration of dopamine will correct the motor symptoms of PD in the pre-clinical model.

Study Design:
In the first set of experiments, we will apply the same neuronal reprogramming agent we have tested on pre-clinical cells, human cells and pre-clinical models of PD to the pre-clincial brain to determine whether this strategy will generate new dopamine-producing neurons. We will then determine whether this restoration of dopamine signaling can be detected by PET scan. In a second set of experiments, we will improve our reprogramming agent by increasing its specificity in targeting a specific non-neuronal cell type in the pre-clinical brain, which will enhance our ability to characterize newly generated neurons in the pre-clinical brain.

Impact on Diagnosis/Treatment of Parkinson’s Disease:
The proposed study bridges the proof of concept in a preclinical model of PD and clinical trials in humans. If successful, our strategy will provide a therapy ready for testing in people with PD.

Next Steps for Development:
If the proposed experiments are a success, we will finalize the reprogramming agent for a large-scale safety test in pre-clinical models. Once the procedure is proven to be safe, we would like to launch clinical trials as soon as possible.


Researchers

  • Xiang-Dong Fu, PhD

    La Jolla, CA United States


  • William Mobley, MD, PhD

    La Jolla, CA United States


  • Jeffrey H. Kordower, PhD

    Tempe, AZ United States


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