Recent advances and future challenges in gene therapy for hearing loss

Hearing loss is the most common sensory deficit experienced by humans and represents one of the largest chronic health conditions worldwide. It is expected that around 10% of the world's population will be affected by disabling hearing impairment by 2050. Hereditary hearing loss accounts for mo...

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Main Authors: Ana E. Amariutei, Jing-Yi Jeng, Saaid Safieddine, Walter Marcotti
Format: Article
Language:English
Published: The Royal Society 2023-06-01
Series:Royal Society Open Science
Subjects:
Online Access:https://royalsocietypublishing.org/doi/10.1098/rsos.230644
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author Ana E. Amariutei
Jing-Yi Jeng
Saaid Safieddine
Walter Marcotti
author_facet Ana E. Amariutei
Jing-Yi Jeng
Saaid Safieddine
Walter Marcotti
author_sort Ana E. Amariutei
collection DOAJ
description Hearing loss is the most common sensory deficit experienced by humans and represents one of the largest chronic health conditions worldwide. It is expected that around 10% of the world's population will be affected by disabling hearing impairment by 2050. Hereditary hearing loss accounts for most of the known forms of congenital deafness, and over 25% of adult-onset or progressive hearing loss. Despite the identification of well over 130 genes associated with deafness, there is currently no curative treatment for inherited deafness. Recently, several pre-clinical studies in mice that exhibit key features of human deafness have shown promising hearing recovery through gene therapy involving the replacement of the defective gene with a functional one. Although the potential application of this therapeutic approach to humans is closer than ever, substantial further challenges need to be overcome, including testing the safety and longevity of the treatment, identifying critical therapeutic time windows and improving the efficiency of the treatment. Herein, we provide an overview of the recent advances in gene therapy and highlight the current hurdles that the scientific community need to overcome to ensure a safe and secure implementation of this therapeutic approach in clinical trials.
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spelling doaj.art-3d8b373fd34b4f61a61ddbf341419d2f2023-06-14T07:05:28ZengThe Royal SocietyRoyal Society Open Science2054-57032023-06-0110610.1098/rsos.230644Recent advances and future challenges in gene therapy for hearing lossAna E. Amariutei0Jing-Yi Jeng1Saaid Safieddine2Walter Marcotti3School of Biomedical Science, University of Sheffield, Sheffield S10 2TN, UKSchool of Biomedical Science, University of Sheffield, Sheffield S10 2TN, UKInstitut Pasteur, Université Paris Cité, Inserm, Institut de l'Audition, F-75012 Paris, FranceSchool of Biomedical Science, University of Sheffield, Sheffield S10 2TN, UKHearing loss is the most common sensory deficit experienced by humans and represents one of the largest chronic health conditions worldwide. It is expected that around 10% of the world's population will be affected by disabling hearing impairment by 2050. Hereditary hearing loss accounts for most of the known forms of congenital deafness, and over 25% of adult-onset or progressive hearing loss. Despite the identification of well over 130 genes associated with deafness, there is currently no curative treatment for inherited deafness. Recently, several pre-clinical studies in mice that exhibit key features of human deafness have shown promising hearing recovery through gene therapy involving the replacement of the defective gene with a functional one. Although the potential application of this therapeutic approach to humans is closer than ever, substantial further challenges need to be overcome, including testing the safety and longevity of the treatment, identifying critical therapeutic time windows and improving the efficiency of the treatment. Herein, we provide an overview of the recent advances in gene therapy and highlight the current hurdles that the scientific community need to overcome to ensure a safe and secure implementation of this therapeutic approach in clinical trials.https://royalsocietypublishing.org/doi/10.1098/rsos.230644hearinghearing lossdeafnesscochleahair cellgene therapy
spellingShingle Ana E. Amariutei
Jing-Yi Jeng
Saaid Safieddine
Walter Marcotti
Recent advances and future challenges in gene therapy for hearing loss
Royal Society Open Science
hearing
hearing loss
deafness
cochlea
hair cell
gene therapy
title Recent advances and future challenges in gene therapy for hearing loss
title_full Recent advances and future challenges in gene therapy for hearing loss
title_fullStr Recent advances and future challenges in gene therapy for hearing loss
title_full_unstemmed Recent advances and future challenges in gene therapy for hearing loss
title_short Recent advances and future challenges in gene therapy for hearing loss
title_sort recent advances and future challenges in gene therapy for hearing loss
topic hearing
hearing loss
deafness
cochlea
hair cell
gene therapy
url https://royalsocietypublishing.org/doi/10.1098/rsos.230644
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