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Investigating Common Molecular Pathogenic Pathways in Frontotemporal Dementia Patients iPSC-Derived Neurons

Award Number: R01NS057553
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: 12/18/2007
PERIOD OF PERFORMANCE END DATE: 01/31/2031

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Investigating Common Molecular Pathogenic Pathways in Frontotemporal Dementia Patients iPSC-Derived Neurons - ABSTRACT Frontotemporal dementia (FTD) is the most common form of presenile dementia under the age of 60 and about 40% of FTD cases are familial and caused by genetic mutations in a number of genes including C9ORF72. GGGGCC (G4C2) repeat expansion in the first intron of C9ORF72 can be translated into multiple toxic dipeptide repeat (DPR) proteins such as poly(GR) that form aggregates in patient brains but their underlying pathogenic mechanisms in human neurons remain largely unknown. Nuclear depletion of TDP-43 is another major hallmark of both familial and sporadic FTD as well as some other neurodegenerative diseases such as ALS and Alzheimer’s disease. However, common pathogenic mechanisms in familial and sporadic FTD with or without TDP-43 pathology are poorly defined. In this R01 application, we will use patient cortical neurons differentiated from induced pluripotent stem cells (iPSCs) with different genetic mutations including C9ORF72 and TARDBP (that encodes the DNA/RNA-binding protein TDP-43) as well as iPSCs derived from sporadic FTD/ALS patients as a major experimental system. Some supporting experiments will also be carried out in our Drosophila models of FTD. A combination of classical and cutting-edge molecular and cellular approaches (such as CRISPR editing and transcriptomic analysis) will be employed to reveal common pathogenic mechanisms such as DNA damage and modifier pathways in patient neurons and identify potential therapeutic targets for FTD.


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: 2026 ( Subtotal = $637,775 )
2026	2026	UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL	55 LAKE AVE N	WORCESTER	MA	01655	WORCESTER	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	19	2/17/2026	COMPETING CONTINUATION	$637,775
														Subtotal = $637,775

Issue Date FY: 2018 ( Subtotal = $0 )
2018	2017	UNIVERSITY OF MASSACHUSETTS	55 LAKE AVE N	WORCESTER	MA	01655	WORCESTER	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	11	3/29/2018	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2017 ( Subtotal = $366,406 )
2017	2017	UNIVERSITY OF MASSACHUSETTS	55 LAKE AVE N	WORCESTER	MA	01655	WORCESTER	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	11	6/1/2017	NON-COMPETING CONTINUATION	$36,641
2017	2017	UNIVERSITY OF MASSACHUSETTS	55 LAKE AVE N	WORCESTER	MA	01655	WORCESTER	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	11	12/21/2016	NON-COMPETING CONTINUATION	$329,765
														Subtotal = $366,406

Issue Date FY: 2016 ( Subtotal = $329,765 )
2016	2016	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	10	12/17/2015	EXTENSION WITH OR WITHOUT FUNDS	$329,765
														Subtotal = $329,765

Issue Date FY: 2015 ( Subtotal = $366,406 )
2015	2015	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	9	1/6/2015	NON-COMPETING CONTINUATION	$36,641
2015	2015	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	9	12/22/2014	NON-COMPETING CONTINUATION	$329,765
														Subtotal = $366,406

Issue Date FY: 2014 ( Subtotal = $361,480 )
2014	2014	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	8	11/27/2013	NON-COMPETING CONTINUATION	$328,617
2014	2014	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	8	3/10/2014	NON-COMPETING CONTINUATION	$32,863
														Subtotal = $361,480

Issue Date FY: 2013 ( Subtotal = $363,581 )
2013	2013	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	7	11/27/2012	COMPETING CONTINUATION	$363,581
														Subtotal = $363,581

Issue Date FY: 2012 ( Subtotal = $317,383 )
2012	2012	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	6	2/20/2012	NON-COMPETING CONTINUATION	$31,739
2012	2012	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	6	12/5/2011	NON-COMPETING CONTINUATION	$285,644
														Subtotal = $317,383

Issue Date FY: 2011 ( Subtotal = $317,383 )
2011	2011	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	5	6/8/2011	NON-COMPETING CONTINUATION	$25,909
2011	2011	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	11/26/2010	NON-COMPETING CONTINUATION	$291,474
														Subtotal = $317,383

Issue Date FY: 2010 ( Subtotal = $380,067 )
2010	2010	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	11/25/2009	NON-COMPETING CONTINUATION	$345,517
2010	2010	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	004	4	9/10/2010	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$380,067
2010	2010	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	3	9/9/2010	NON-COMPETING CONTINUATION	-$380,067
2010	2010	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	1/13/2010	NON-COMPETING CONTINUATION	$34,550
2010	2009	UNIVERSITY OF MASSACHUSETTS	406 GOODELL BLDG, BOX 33210	AMHERST	MA	01003	HAMPSHIRE	USA	Trans-NIH Recovery Act Research Support	003	4	9/10/2010	SUPPLEMENT FOR EXPANSION	$247,634
2010	2009	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Trans-NIH Recovery Act Research Support	005	2	9/13/2010	SUPPLEMENT FOR EXPANSION	-$247,634
														Subtotal = $380,067

Issue Date FY: 2009 ( Subtotal = $647,336 )
2009	2009	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	2	4/7/2009	NON-COMPETING CONTINUATION	$38,389
2009	2009	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Trans-NIH Recovery Act Research Support	002	2	9/15/2009	SUPPLEMENT FOR EXPANSION	$263,430
2009	2009	J DAVID GLADSTONE INSTITUTES	P.O. BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	11/25/2008	NON-COMPETING CONTINUATION	$345,517
														Subtotal = $647,336

Issue Date FY: 2008 ( Subtotal = $383,906 )
2008	2008	J DAVID GLADSTONE INSTITUTES	POST OFFICE BOX 419100	SAN FRANCISCO	CA	94141	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	12/4/2007	NEW	$383,906
														Subtotal = $383,906

Grand Total All Awards = $4,471,488

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Investigating Common Molecular Pathogenic Pathways in Frontotemporal Dementia Patients iPSC-Derived Neurons

Award Number: R01NS057553

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: 12/18/2007

PERIOD OF PERFORMANCE END DATE: 01/31/2031

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