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Establishing the Therapeutic Potential of Engineered Neural Circuits in a Preclinical Model of Parkinson’s Disease

Study Rationale: Parkinson’s disease (PD) is caused by the death of specific brain cells that connect two different regions of the brain, disrupting a neural circuit that is key for motor function. We have developed tissue-engineering techniques that allow us to grow cells in the lab that reconnect this circuitry and, after transplantation, reestablish a pathway between the two brain regions. If successful, this approach may provide a groundbreaking treatment for people with PD.

Hypothesis: We hypothesize that transplanting our lab-grown neural circuits into preclinical models for PD will demonstrate significant therapeutic potential that may translate into a treatment for individuals with PD.

Study Design: We plan to test how thoroughly and efficiently our lab-grown neural circuits integrate into the brain and assess how fully this treatment restores dopamine levels compared to other cell transplant approaches. We will also evaluate the therapeutic effects of our lab-grown neural circuits on behaviors related to the motor symptoms of PD.

Impact on Diagnosis/Treatment of Parkinson’s disease: If successful, our engineered neural transplants could revolutionize the treatment of PD. This treatment approach could alleviate many symptoms of PD and potentially alter the course of the disease.

Next Steps for Development: The proposed studies are important for advancing our engineered circuits from the bench to the bedside. The data we gather will be critical to understanding the benefits of our circuit-replacement approach as well as interacting with regulatory agencies to pave the way for clinical studies in people with PD.


Researchers

  • D. Kacy Cullen, PhD

    Philadelphia, PA United States


  • John Duda, MD

    Philadelphia, PA United States


  • Han-Chiao Isaac Chen, MD

    Philadephia, PA United States


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