Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss

There is growing attention and effort focused on treating the root cause of sensorineural hearing loss rather than managing associated secondary characteristic features. With recent substantial advances in understanding sensorineural hearing-loss mechanisms, gene delivery has emerged as a promising...

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Main Authors:	Kamakshi Bankoti, Charles Generotti, Tiffany Hwa, Lili Wang, Bert W. O’Malley, Jr., Daqing Li
Format:	Article
Language:	English
Published:	Elsevier 2021-06-01
Series:	Molecular Therapy: Methods & Clinical Development
Subjects:	AAV inner ear sensorineural hearing loss
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050121000450

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spelling	doaj-4be1d414966348389c8b1a8d38d231322021-06-13T04:38:46ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012021-06-0121209236Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing lossKamakshi Bankoti0Charles Generotti1Tiffany Hwa2Lili Wang3Bert W. O’Malley, Jr.4Daqing Li5Department of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USADepartment of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USADepartment of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USADepartment of Medicine, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USADepartment of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USADepartment of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Corresponding author: Daqing Li, MD, Department of Otorhinolaryngology, Head and Neck Surgery, Perelman School of Medicine, University of Pennsylvania, 421 Curie Blvd., BRB 1212, Philadelphia, PA 19104, USA.There is growing attention and effort focused on treating the root cause of sensorineural hearing loss rather than managing associated secondary characteristic features. With recent substantial advances in understanding sensorineural hearing-loss mechanisms, gene delivery has emerged as a promising strategy for the biological treatment of hearing loss associated with genetic dysfunction. There are several successful and promising proof-of-principle examples of transgene deliveries in animal models; however, there remains substantial further progress to be made in these avenues before realizing their clinical application in humans. Herein, we review different aspects of development, ongoing preclinical studies, and challenges to the clinical transition of transgene delivery of the inner ear toward the restoration of lost auditory and vestibular function.http://www.sciencedirect.com/science/article/pii/S2329050121000450AAVinner earsensorineural hearing loss
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Kamakshi Bankoti Charles Generotti Tiffany Hwa Lili Wang Bert W. O’Malley, Jr. Daqing Li
spellingShingle	Kamakshi Bankoti Charles Generotti Tiffany Hwa Lili Wang Bert W. O’Malley, Jr. Daqing Li Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss Molecular Therapy: Methods & Clinical Development AAV inner ear sensorineural hearing loss
author_facet	Kamakshi Bankoti Charles Generotti Tiffany Hwa Lili Wang Bert W. O’Malley, Jr. Daqing Li
author_sort	Kamakshi Bankoti
title	Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
title_short	Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
title_full	Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
title_fullStr	Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
title_full_unstemmed	Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
title_sort	advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss
publisher	Elsevier
series	Molecular Therapy: Methods & Clinical Development
issn	2329-0501
publishDate	2021-06-01
description	There is growing attention and effort focused on treating the root cause of sensorineural hearing loss rather than managing associated secondary characteristic features. With recent substantial advances in understanding sensorineural hearing-loss mechanisms, gene delivery has emerged as a promising strategy for the biological treatment of hearing loss associated with genetic dysfunction. There are several successful and promising proof-of-principle examples of transgene deliveries in animal models; however, there remains substantial further progress to be made in these avenues before realizing their clinical application in humans. Herein, we review different aspects of development, ongoing preclinical studies, and challenges to the clinical transition of transgene delivery of the inner ear toward the restoration of lost auditory and vestibular function.
topic	AAV inner ear sensorineural hearing loss
url	http://www.sciencedirect.com/science/article/pii/S2329050121000450
work_keys_str_mv	AT kamakshibankoti advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss AT charlesgenerotti advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss AT tiffanyhwa advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss AT liliwang advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss AT bertwomalleyjr advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss AT daqingli advancesandchallengesinadenoassociatedviralinnereargenetherapyforsensorineuralhearingloss
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Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss

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