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Molecular Segregation of Parkinson’s Disease by Patient-Derived Neurons

Award Number: R01NS113763
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 06/01/2020
PERIOD OF PERFORMANCE END DATE: 05/31/2025

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Molecular Segregation of Parkinson’s Disease by Patient-Derived Neurons - Parkinson’s disease (PD) is defined by its hallmark locomotor symptoms including tremor, rigidity, bradykinesia and postural instability, which are caused by a progressive loss of nigral dopaminergic (DA) neurons. A well-recognized categorization of Parkinson’s disease is based on whether rest tremor is present or not at disease onset. PD patients who have rest tremor at onset generally have slower progression and better prognosis than PD patients without rest tremor at onset. Our preliminary study showed that the expression of genes controlling dopamine synthesis, sequestration and degradation was significantly different between midbrain DA neurons derived from induced pluripotent stem cells (iPSC) of normal subjects vs. idiopathic PD patients. Expression of some of these genes was also significantly different between idiopathic PD patients with or without rest tremor at onset. We have developed a series of new technologies including the differentiation of iPSCs to A9 DA neurons and the direct conversion of human skin fibroblasts and urinary track cells (UTCs) to midbrain DA neurons. Using these innovative technologies, the proposal aims to identify molecular signatures that can segregate PD patients and normal subjects, and distinguish PD patients with or without rest tremor at onset. The converging development of stem cell technologies enables this project to identify molecular signatures of idiopathic Parkinson’s disease, which will significantly advance PD diagnosis, research and therapeutic development.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2024 ( Subtotal = $435,515 )
2024	2024	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	6/4/2024	NON-COMPETING CONTINUATION	$435,515
														Subtotal = $435,515

Issue Date FY: 2023 ( Subtotal = $850,235 )
2023	2023	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	4	5/17/2023	NON-COMPETING CONTINUATION	$448,985
2023	2023	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	5/9/2023	SUPPLEMENT FOR EXPANSION	$401,250
														Subtotal = $850,235

Issue Date FY: 2022 ( Subtotal = $448,985 )
2022	2022	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	3	4/25/2022	NON-COMPETING CONTINUATION	$448,985
2022	2021	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	2	10/20/2021	NON-COMPETING CONTINUATION	$0
2022	2020	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	10/20/2021	NEW	$0
														Subtotal = $448,985

Issue Date FY: 2021 ( Subtotal = $448,985 )
2021	2021	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	3/30/2021	NON-COMPETING CONTINUATION	$448,985
														Subtotal = $448,985

Issue Date FY: 2020 ( Subtotal = $448,985 )
2020	2020	RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK, THE	520 LEE ENTRANCE STE 211	AMHERST	NY	14228	ERIE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	5/29/2020	NEW	$448,985
														Subtotal = $448,985

Grand Total All Awards = $2,632,705

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Molecular Segregation of Parkinson’s Disease by Patient-Derived Neurons

Award Number: R01NS113763

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 06/01/2020

PERIOD OF PERFORMANCE END DATE: 05/31/2025

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