Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy
AssigneesUcb Biopharma Srl, FundacióN Para La InvestigacióN MéDica Aplicada, Consorcio Centro De InvestigacióN BioméDica En Red
InventorsGloria GONZALEZ ASEGUINOLAZA, José Luis LANCIEGO PEREZ, Ralph Michael LINDEN
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Abstract
The present disclosure relates to viral particles for use in treating synucleinopathies, particularly sporadic Parkinson Diseases by gene therapy. More specifically, the present invention relates to a viral particle for use in treating synucleinopathy by gene therapy in a subject in need thereof, said viral particle comprising a nucleic acid construct including a transgene encoding a glucocerebrosidase.
Claims34
A viral particle comprising a nucleic acid construct comprising a transgene encoding a glucocerebrosidase.
An in vivo dissemination assay comprising the steps:
- a) injecting into the post-commissural putamen of a non-human primate a rAAV comprising a transgene encoding a green fluorescent protein (GFP) by intraparenchymal injection, and,
- b) counting the number of neurons in the cerebral cortex, preferably in the brain areas innervating the caudate putamen nuclei expressing GFP about one month post injection.
Dependencies are inferred from the claim text.