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Antisense Oligonucleotides for Progranulin-deficient Frontotemporal Dementia

Award Number: R56AG064069
ORGANIZATION: NATIONAL INSTITUTE ON AGING
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/30/2024
PERIOD OF PERFORMANCE END DATE: 09/29/2025

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Antisense Oligonucleotides for Progranulin-deficient Frontotemporal Dementia - Project Summary Progranulin is a lysosomal and secreted protein that contains multiple cysteine-rich granulin domains. Although its precise molecular function remains unknown, heterozygous progranulin (GRN) mutations are causal for frontotemporal dementia (FTD). FTD is a devastating disease with a mean survival of 7 years from diagnosis and no approved disease-modifying treatment. Since this is a condition of haploinsufficiency, strategies aimed at increasing progranulin levels are feasible therapeutic approaches. Antisense oligonucleotides (ASOs) are emerging as a promising therapeutic modality for neurological disorders. ASOs are short, single-stranded sequences of RNA that are designed to bind to their target RNAs and enable highly specific modulation of target levels. We have developed a novel strategy for increasing progranulin levels by using ASOs to sterically block microRNA binding sites in the human GRN mRNA. We have identified ASOs that effectively increase progranulin protein levels in the brains of humanized GRN mice. Building on this, we will: 1) test if ASO treatment improves FTD-associated behavioral deficits and neuroinflammation in humanized GRN mice with haploinsufficiency, 2) define the timing window for effective ASO treatment, and 3) assess potential off-target effects for lead ASOs. Completion of these studies will generate important preclinical data that provide insights into the utility of this ASO-based therapeutic approach for progranulin-deficient 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 = -$957 )
2026	2024	SAINT LOUIS UNIVERSITY	221 N GRAND BLVD	SAINT LOUIS	MO	63103	SAINT LOUIS CITY	USA	Aging Research	000	6	3/18/2026	COMPETING CONTINUATION	-$957
														Subtotal = -$957

Issue Date FY: 2024 ( Subtotal = $75,350 )
2024	2024	SAINT LOUIS UNIVERSITY	221 N GRAND BLVD	SAINT LOUIS	MO	63103	SAINT LOUIS CITY	USA	Aging Research	000	6	9/18/2024	COMPETING CONTINUATION	$75,350
														Subtotal = $75,350

Grand Total All Awards = $74,393

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Antisense Oligonucleotides for Progranulin-deficient Frontotemporal Dementia

Award Number: R56AG064069

ORGANIZATION: NATIONAL INSTITUTE ON AGING

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/30/2024

PERIOD OF PERFORMANCE END DATE: 09/29/2025

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