Antisense oligonucleotides for treatment of neurological disorders
AssigneesAUM LifeTech, Inc, The Trustees of the University of Pennsylvania
InventorsVeenu Aishwarya, Anna Caputo, Kelvin Cheuk Wai Luk
Life of this patent
Estimating the term…
Abstract
Parkinson's Disease (PD) is the second most common neurodegenerative disorder. Essentially all PD patients accumulate misfolded forms of alpha-synuclein (α-Syn) in their neurons, while mutations in the α-synuclein gene (SNCA) cause familial PD, suggesting that abnormal α-synuclein plays a central role in PD. The present invention is based on the seminal discovery that FANA antisense oligonucleotides targeting α-synuclein are effective at treating and/or preventing Parkinson's Disease. Specifically. FANA antisense oligonucleotides targeting α-synuclein decrease the expression of α-synuclein in neurons and decrease Lewy body and Lewy neurite pathology.
Claims17
A composition comprising an α-synuclein targeting FANA-ASO oligonucleotide.
A pharmaceutical composition comprising an α-synuclein targeting FANA-ASO oligonucleotide and a pharmaceutically acceptable carrier.
A method of decreasing α-synuclein expression comprising administering an α-synuclein targeting FANA-ASO oligonucleotide to a subject in need thereof, thereby reducing α-synuclein expression.
17-35. (canceled)
Dependencies are inferred from the claim text.