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Funded Studies

Exosome/Microvesicle Release from LRRK2 Mutant iPSC-derived Neural Cells

Objective/Rationale:             
Exosomes/microvesicles are small vesicles that are shed from the cell membrane and are known to play a role in communication between cells. Exosomes/microvesicles carry cargoes that modulate gene expression or cell function and are shed from many cell types, including those of the brain. They also can be found in blood, cerebral spinal fluid and urine. This project aims to improve methods for identification and isolation of exosomes/microvesicles from neural cells, as they may serve as potential biomarkers for identification of Parkinson's disease.

Project Description:
Researchers will utilize induced pluripotent stem cell lines that were created from Parkinson's disease patients with a mutation in the LRRK2 gene. Cells will be grown in culture and differentiated into neural cells. The conditioned medium from the cells will be collected and examined for the presence of exosomes/microvesicles. Scientists will employ a variety of techniques to identify neural-specific microvesicles.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:                     
The long-term goal of this project is to identify biomarkers of Parkinson's disease that will aide in patient diagnosis and treatment development. In order to identify a biomarker, scientists must first know where they are and how to find them in a bodily fluid such as CSF, blood, or urine that contains many other substances, most of which are unrelated to the affected cells in the brain of the patient.

Anticipated Outcome:
Researchers anticipate new methodologies for the identification of neural-derived microvesicles. Work from these studies can then be performed using patient samples. This will allow progress in the field of biomarker development and eventually lead to improvements in the diagnosis and treatment of Parkinson's disease.


Researchers

  • Kate M. Candelario, PhD

    Gainesville, FL United States


  • Dennis A. Steindler, PhD

    Boston, MA United States


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