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Autosomal Dominant Non-Syndromic Hearing Loss - Its Genetic Diagnosis and Treatment

Award Number: R01DC017955
ORGANIZATION: NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/01/2019
PERIOD OF PERFORMANCE END DATE: 07/31/2025

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Autosomal Dominant Non-Syndromic Hearing Loss - Its Genetic Diagnosis and Treatment - Project Summary Hearing loss affects 15-26% of the world's population. Amongst the elderly, it ranks as the most common neurological disability, impacting ~50% of octogenarians. The majority of inherited late-onset deafness is autosomal dominant and non-syndromic. In this new porposal, we will build on our experience and expertise in human genetics and gene therapy to improve genetic deafness for deafness by leveraging the power of single- cell RNA sequencing and to establish whether targeted allele suppression can prevent or potentially reverse hearing loss in mature animal models of human deafness by using RNA interference (RNAi). To achieve these goals, we propose the following specific aims: • Specific Aim 1. Using single-cell isolation techniques, we propose: o SA1A. To refine our knowledge of individual cell-specific transcription in the organ of Corti and improve comprehensive genetic testing for deafness by identifying and characterizing novel exons and isoforms of genes implicated in both non-syndromic and syndromic forms of hearing loss. o SA1B. To characterize the broader impact of single gene mutations on the transcriptome of individual cells in two murine models of human ADNSHL – the Kncq4+/- mouse, which is a model of human DFNA2-related hearing loss, and the Tmc1Bth/+ mouse (the Beethoven (Bth) mouse), which is a model of human DFNA36-related hearing loss. • Specific Aim 2. Using RNAi, we propose: o SA2A. To establish whether targeted allele suppression can prevent or potentially reverse hearing loss in mature animals. o SA2B. To develop a broadly applicable treatment strategy for all types of deafness caused by a single gene using a novel RNAi construct that suppresses both endogenous alleles with concomitant gene replacement by an exogenous wild-type allele engineered to resist RNAi-mediated silencing. o SA2C. To determine whether systemic neutralizing antibodies (NAbs) to AAV impact the transduction efficiency of AAV-mediated cochlear gene therapy. The successful completion of these aims will advance our understanding of the biology of hearing and deafness, enhance the clinical care of persons with hearing loss, and potentially lay the foundation for clinical trials using RNAi as a treatment option for some types of hearing loss.


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 = $0 )
2026	2023	THE UNIVERSITY OF IOWA	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	5	11/20/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2023 ( Subtotal = $471,223 )
2023	2023	THE UNIVERSITY OF IOWA	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	5	7/26/2023	NON-COMPETING CONTINUATION	$471,223
														Subtotal = $471,223

Issue Date FY: 2022 ( Subtotal = $471,223 )
2022	2022	UNIVERSITY OF IOWA, THE	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	4	6/29/2022	NON-COMPETING CONTINUATION	$471,223
														Subtotal = $471,223

Issue Date FY: 2021 ( Subtotal = $486,171 )
2021	2021	UNIVERSITY OF IOWA, THE	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	3	8/3/2021	NON-COMPETING CONTINUATION	$486,171
														Subtotal = $486,171

Issue Date FY: 2020 ( Subtotal = $606,990 )
2020	2020	UNIVERSITY OF IOWA, THE	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	2	6/23/2020	NON-COMPETING CONTINUATION	$606,990
														Subtotal = $606,990

Issue Date FY: 2019 ( Subtotal = $606,990 )
2019	2019	UNIVERSITY OF IOWA, THE	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Research Related to Deafness and Communication Disorders	000	1	7/25/2019	NEW	$606,990
														Subtotal = $606,990

Grand Total All Awards = $2,642,597

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Autosomal Dominant Non-Syndromic Hearing Loss - Its Genetic Diagnosis and Treatment

Award Number: R01DC017955

ORGANIZATION: NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 08/01/2019

PERIOD OF PERFORMANCE END DATE: 07/31/2025

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