RAB3B for treatment and prevention of Parkinson's disease
The invention features methods and compositions for the treatment and prevention of Parkinson's Disease.
1. A method for increasing dopaminergic neuron content in the brain of a human patient affected with Parkinson's Disease, said method comprising administering with a stereotactic injection into the substantia nigra and/or ventral tegmental region comprising A9 neurons of the patient's brain an adeno associated virus vector comprising a polynucleotide encoding human RAB3B having the sequence set forth in SEQ ID NO: 6, wherein the human RAB3B is expressed and wherein the expression results in increase of dopaminergic neuron content.
2. The method of claim 1 , wherein said substantia nigra region is substantia nigra A9 region comprising A9 neurons.
3. The method of claim 1 , wherein said polynucleotide encoding human RAB3B is operably linked to at least one regulatory element.
4. The method of claim 1 , wherein the stereotactic injection is performed as intracerebroventricular injection.
5. The method of claim 1 , wherein the stereotactic injection is performed as intranigral injection.
6. The method of claim 3 , wherein the adeno-associated virus is AAV2.