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Genetic Modulation of LAMP-2A in PD Models

Study Rationale:                   
Brain cells use surveillance mechanisms to get rid of toxic proteins, such as mutant alpha-synuclein, that otherwise would accumulate and kill them. We have previously identified that one of such surveillance/cleaning mechanisms fails in brain cells affected by Parkinson's disease. In this study we intend to test whether activating this failing pathway in Parkinson's disease prevents death of brain cells.

Activation of a cellular cleaning mechanism in Parkinson's disease cells may prevent, slow or even reverse pathology and symptoms.

Study Design:
To be able to unequivocally activate this cellular cleaning mechanism and draw conclusions of the possible beneficial effect, we will use a genetic model where we can regulate activation of this process at wish. Although for translation into humans, we will need to use drugs, having genetic proof-of-principle of the beneficial effect will provide support for any future efforts (including those ongoing in our lab) in drugs targeting this pathway.

Impact on Diagnosis/Treatment of Parkinson's Disease:             
We expect that this pre-clinical data will incentive industry to invest efforts and resources to target this pathway as a possible cure for Parkinson's disease.

Next Steps for Development:
If we obtain a positive result we will next: 1) test in vivo some of the molecules that we are developing with the support of MJFF in models of Parkinson's disease to see if we can get comparable results and 2) continue testing/developing molecules with different mechanisms of action but same final effect. 


  • David Sulzer, PhD

    New York, NY United States

  • Ana Maria Cuervo, MD, PhD

    Bronx, NY United States

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