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Mingling life imaging and cell subpopulations expression analysis in NHP lungs targeted with exosome associated double AAV vectors

Award Number: R21AI182508
ORGANIZATION: NATIONAL INSTITUTE OF ALLERGY & INFECTIOUS DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 06/25/2025
PERIOD OF PERFORMANCE END DATE: 05/31/2027

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Mingling life imaging and cell subpopulations expression analysis in NHP lungs targeted with exosome associated double AAV vectors - PROJECT SUMMARY Gene therapy provides a universal therapeutic option for patients with genetic or acquired disorders that can potentially benefit a broad patient populations. However, up to date several gene therapy clinical trials for lung- specific diseases, while well addressing safety concerns, have failed to demonstrate clear therapeutic benefits among the participated patients. This dismal reality is most likely attributed to the inability to achieve clinically- relevant gene transfer efficacy in the airway epithelium, and particularly to airway basal stem cell or basal cell. Recently, we have demonstrated the ability of AAV serotype 6 (AAV6) vectors to achieve widespread and efficient transgene expression in the human airway epithelial cell (AEC) in in vitro and in the mouse models in vivo. Importantly, subsets of the lung cell, such as basal cells, were targeting that provides anticipation for possible long-term gene expression in highly renewable layer of airways. Additionally we showed in our most recent published collaborative studies higher transduction efficiency over commonly used AAVs by extracellular vesicles associated AAV (EV-AAV) to the mouse lungs and human AEC cultured in air-liquid interface conditions. The large cargo of average EVs (100-200 nm) allows accumulation of multiple AAV particles (20-25 nm), and, unlike AAV, EVs enter cells multiple by ways that do not depend on receptor abundance. These important findings lead to the development of current proposal that combines non-invasive in vivo imaging with post-necropsy analysis of AAV-mediated gene expression in subpopulations of airway cells in non-human primates (NHP) model. NHP models play a critical role in feasibility and safety modeling for the development of therapeutics. The mechanistic underpinning is expected to provide critical information for the design of next-generation gene therapy vectors targeting lung, which can be used for wide range of pulmonary diseases required reliable expression of therapeutic modalities across airways.


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: 2025 ( Subtotal = $437,250 )
2025	2025	REGENTS OF THE UNIVERSITY OF MINNESOTA	2221 UNIVERSITY AVE SE STE 100	MINNEAPOLIS	MN	55414	HENNEPIN	USA	Allergy and Infectious Diseases Research	000	1	6/25/2025	NEW	$437,250
														Subtotal = $437,250

Grand Total All Awards = $437,250

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Mingling life imaging and cell subpopulations expression analysis in NHP lungs targeted with exosome associated double AAV vectors

Award Number: R21AI182508

ORGANIZATION: NATIONAL INSTITUTE OF ALLERGY & INFECTIOUS DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 06/25/2025

PERIOD OF PERFORMANCE END DATE: 05/31/2027

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