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Funded Studies

Deep Phenotyping of Parkinson's Disease Relevant Mitochondrial and Autophagic Mechanisms in Human iPSC-derived Neural Cells

Study Rationale: The causes of Parkinson’s disease (PD) are not fully understood. However, impairment in the function of mitochondria, the powerhouses of the cell, and in autophagy, a process whereby cells can remove and degrade unwanted or damaged materials, occur in PD. Therefore, determining the precise biological mechanisms underlying mitochondrial and autophagic changes in PD will be important for understanding and treating the disease.

Hypothesis: We hypothesize that neurons derived from individuals with sporadic PD will show specific types of alterations in mitochondria and in autophagy that distinguish them from neurons obtained from healthy individuals.

Study Design: We will carefully examine the mitochondria and autophagy-related processes in neurons generated from people with PD, comparing them to neurons from unaffected people. All neurons will be grown in culture and we will use a variety of methods to assess mitochondrial structure and function, cellular degradation mechanisms and changes in proteins of interest.

Impact on Diagnosis/Treatment of Parkinson’s disease: A major roadblock impeding the development of effective therapeutics has been the inadequate understanding of how PD develops. By studying mechanisms related to mitochondria and autophagy, which are fundamental to PD, our project will reveal important biological changes, and potential biomarkers, that facilitate the early diagnosis and treatment of PD.

Next Steps for Development: The proposed work will lay the foundation for future studies which will use patient-derived cells to identify specific biomarkers relevant to diagnosing PD early, monitoring PD progression and developing a cellular system for testing novel drugs.


  • Lalitha Madhavan, MD, PhD

    Tucson, AZ United States

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