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Genomically rewritten and tailored humanized mouse models for various organ disorders

Award Number: R24OD037800
ORGANIZATION: OFFICE OF THE DIRECTOR, NIH
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2025
PERIOD OF PERFORMANCE END DATE: 02/28/2029

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Genomically rewritten and tailored humanized mouse models for various organ disorders - Summary This proposal leverages Big DNA technology to produce mouse models of a variety of human diseases. The underlying hypothesis tested in our earlier work and continued in this proposal is that changes in human non- coding DNA that underlie human disease susceptibility will translate into appropriate disease phenotypes in the mouse when the entire human genic region of interest is inserted into a predefined location in the mouse genome. We refer to such animals as GREAT-GEMMs (Genomically Rewritten and Tailored Genetically engineered moused models). Furthermore, GREAT-GEMMs will serve as a resource for testing therapeutic interventions against human disease. These animals will be uniquely useful for cutting edge therapies like gene therapies, CRISPR based therapies and allele specific oligonucleotide (ASO) therapies which depend on exact human DNA sequences as targets. Our work is based on our recently developed ability to synthesize and precisely edit 50-300+ kilobase DNA constructs, and then to precisely delivery them to mouse embryonic stem cells (mESCs) and subsequently produce mouse models for human disease.


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 = $642,164 )
2026	2026	NEW YORK UNIVERSITY	550 1ST AVE	NEW YORK	NY	10016	NEW YORK	USA	Research Infrastructure Programs	000	2	2/19/2026	NON-COMPETING CONTINUATION	$642,164
														Subtotal = $642,164

Issue Date FY: 2025 ( Subtotal = $844,954 )
2025	2025	NEW YORK UNIVERSITY	550 1ST AVE	NEW YORK	NY	10016	NEW YORK	USA	Research Infrastructure Programs	000	1	4/25/2025	NEW	$844,954
														Subtotal = $844,954

Grand Total All Awards = $1,487,118

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Genomically rewritten and tailored humanized mouse models for various organ disorders

Award Number: R24OD037800

ORGANIZATION: OFFICE OF THE DIRECTOR, NIH

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 05/01/2025

PERIOD OF PERFORMANCE END DATE: 02/28/2029

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