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The Development of Novel Adeno-associated Virus Vectors for Intravenous Parkinson's Gene Therapy

Objective/Rationale:
As Parkinson disease progresses, a wide range of brain areas exhibit evidence of inflammation and degeneration, but presently no therapies exist that selectively target these widespread areas of degeneration. Recent epilepsy-focused gene therapy studies showed that by creating a vast array of different adeno-associated viruses, novel vectors were identified that upon intravenous administration, selectively targeted areas of seizure-induced inflammation. By applying the same approach to a model of Parkinson’s disease, the present studies could identify novel vectors that selectively target the entire range of Parkinson degeneration. 

Project Description:
Parkinsonian pre-clinical models will receive a host of adeno-associated virus mutants intravenously and three days later, their effects on areas of the brain where dopamine neurons have degenerated will be observed. 

Relevance to Diagnosis/Treatment of Parkinson’s Disease:
Presently, treatment of Parkinson’s disease involves either electrical stimulation or drug administration which affects the entire brain and has off target side effects. Although site manipulations substantially reduce off-target side effects, Parkinson’s disease affects a range of brain structures. The identification of a viral vector that selectively targets the entire range of Parkinson’s pathology would dramatically advance both the application and the effectiveness of Parkinson’s gene therapy.   

Anticipated Outcome:
Given the success of our epilepsy focused studies, we anticipate recovering a number of adeno-associated virus mutants that after intravenous administration selectively target areas of dopamine neuron degeneration.


Researchers

  • Thomas McCown, PhD

    Chapel Hill, NC United States


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