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Funded Studies

Molecular Basis of Neurodegeneration in GBA-associated Parkinson’s Disease

Study Rationale:
Studies in human genetics have implicated the malfunction of a cellular compartment called the lysosome in the pathogenesis of Parkinson’s and other brain diseases. Lysosomes are membrane-bound organelles that reside in our cells and are best known for their ability to degrade various macromolecules and damaged organelles. By properly performing these functions, lysosomes supply the cell with essential nutrients and maintain cellular homeostasis. We aim to determine how lysosomal dysfunction leads to Parkinson’s disease.

We hypothesize that using models of mutated lysosomal genes (GBA) that are known to increase the risk for Parkinson’s disease, we can determine how the lysosomal environment changes and how these changes lead to disease pathogenesis.

Study Design:
We will use a method that we have developed to purify the lysosomes from brain cells of Parkinson’s disease models. The molecular content of these lysosomes will then be characterized using state-of-the-art analytical chemistry technology.

Impact on Diagnosis/Treatment of Parkinson’s Disease:
We believe that our work will provide molecular understanding of lysosomal dysfunction that drives disease pathogenesis. This work will also identify novel pathogenic pathways that can hopefully be targeted to treat Parkinson’s disease.

Next Steps for Development:
In the future we will develop this project in three different directions: 1) evaluate the identified altered pathways as potential drug targets; 2) determine the cell type in the brain whose lysosomes are mostly affected; 3) use this paradigm to study other forms of Parkinson’s disease.


  • Monther Abu-Remaileh, PhD

    Stanford, CA United States

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