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US 12,564,568 B2Granted
PKC pathway in Parkinson's Disease
AssigneeCedars-Sinai Medical Center
InventorsAlexander Laperle, Samuel Sances, Nur Yucer, Clive N. Svendsen
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AssigneeCedars-Sinai Medical Center
InventorsAlexander Laperle, Samuel Sances, Nur Yucer, Clive N. Svendsen
Estimating the term…
Induced Pluripotent Stem Cell (iPSC) technology enables the generation and study of living brain tissue relevant to Parkinson's disease (PD) ex vivo. Utilizing cell lines from PD patients presents a powerful discovery system that links cellular phenotypes observed in vitro with real clinical data. Differentiating patient derived iPSCs towards a dopaminergic (DA) neural fate revealed that these cells exhibit molecular and functional properties of DA neurons in vitro that are observed to significantly degenerate in the substantia nigra of PD patients. Clinical symptoms that drive the generation of other relevant cell types may also yield novel PD specific phenotypes in vitro that have the potential to lead to new therapeutic avenues for patients with PD. Due to their early onset and nonfamilial origin, differentiated nervous tissue from these patients offer a key opportunity to discover neuron subtype specific pathological mechanisms and importantly interrogate the contribution of their genetic background in susceptibility to PD.
A method of treatment of a subject afflicted with early onset sporadic Parkinson's Disease (EOSPD) by modulating activities of α-synuclein and tyrosine hydroxylase (TH) such that activity of α-synuclein is downregulated and activity of tyrosine hydroxylase (TH) is upregulated in the subject, the method comprising:
A method of reversing or retarding progression of Parkinson's Disease in a subject afflicted with early onset sporadic Parkinson's Disease (EOSPD) by modulating activities of α-synuclein and tyrosine hydroxylase (TH) such that α-synuclein protein level decreases and tyrosine hydroxylase (TH) level increases in dopaminergic neurons of the subject, the method comprising:
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