Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges
Hearing impairment is the most frequent sensory deficit in humans of all age groups, from children (1/500) to the elderly (more than 50% of the over-75 s). Over 50% of congenital deafness are hereditary in nature. The other major causes of deafness, which also may have genetic predisposition, are ag...
Main Authors: | , |
---|---|
Format: | Article |
Language: | English |
Published: |
MDPI AG
2020-07-01
|
Series: | Journal of Clinical Medicine |
Subjects: | |
Online Access: | https://www.mdpi.com/2077-0383/9/7/2309 |
id |
doaj-d2346a5e95274f83b740380b06fee13c |
---|---|
record_format |
Article |
spelling |
doaj-d2346a5e95274f83b740380b06fee13c2020-11-25T03:31:04ZengMDPI AGJournal of Clinical Medicine2077-03832020-07-0192309230910.3390/jcm9072309Inner Ear Gene Therapies Take Off: Current Promises and Future ChallengesSedigheh Delmaghani0Aziz El-Amraoui1Progressive Sensory Disorders, Pathophysiology and Therapy Unit, Institut Pasteur, Institut de l’Audition, INSERM-UMRS1120, Sorbonne Université, 63 rue de Charenton, 75012 Paris, FranceProgressive Sensory Disorders, Pathophysiology and Therapy Unit, Institut Pasteur, Institut de l’Audition, INSERM-UMRS1120, Sorbonne Université, 63 rue de Charenton, 75012 Paris, FranceHearing impairment is the most frequent sensory deficit in humans of all age groups, from children (1/500) to the elderly (more than 50% of the over-75 s). Over 50% of congenital deafness are hereditary in nature. The other major causes of deafness, which also may have genetic predisposition, are aging, acoustic trauma, ototoxic drugs such as aminoglycosides, and noise exposure. Over the last two decades, the study of inherited deafness forms and related animal models has been instrumental in deciphering the molecular, cellular, and physiological mechanisms of disease. However, there is still no curative treatment for sensorineural deafness. Hearing loss is currently palliated by rehabilitation methods: conventional hearing aids, and for more severe forms, cochlear implants. Efforts are continuing to improve these devices to help users to understand speech in noisy environments and to appreciate music. However, neither approach can mediate a full recovery of hearing sensitivity and/or restoration of the native inner ear sensory epithelia. New therapeutic approaches based on gene transfer and gene editing tools are being developed in animal models. In this review, we focus on the successful restoration of auditory and vestibular functions in certain inner ear conditions, paving the way for future clinical applications.https://www.mdpi.com/2077-0383/9/7/2309sensory disorderscochleasensorineural hearing lossgene therapyAAVgenome editing |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Sedigheh Delmaghani Aziz El-Amraoui |
spellingShingle |
Sedigheh Delmaghani Aziz El-Amraoui Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges Journal of Clinical Medicine sensory disorders cochlea sensorineural hearing loss gene therapy AAV genome editing |
author_facet |
Sedigheh Delmaghani Aziz El-Amraoui |
author_sort |
Sedigheh Delmaghani |
title |
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges |
title_short |
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges |
title_full |
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges |
title_fullStr |
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges |
title_full_unstemmed |
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges |
title_sort |
inner ear gene therapies take off: current promises and future challenges |
publisher |
MDPI AG |
series |
Journal of Clinical Medicine |
issn |
2077-0383 |
publishDate |
2020-07-01 |
description |
Hearing impairment is the most frequent sensory deficit in humans of all age groups, from children (1/500) to the elderly (more than 50% of the over-75 s). Over 50% of congenital deafness are hereditary in nature. The other major causes of deafness, which also may have genetic predisposition, are aging, acoustic trauma, ototoxic drugs such as aminoglycosides, and noise exposure. Over the last two decades, the study of inherited deafness forms and related animal models has been instrumental in deciphering the molecular, cellular, and physiological mechanisms of disease. However, there is still no curative treatment for sensorineural deafness. Hearing loss is currently palliated by rehabilitation methods: conventional hearing aids, and for more severe forms, cochlear implants. Efforts are continuing to improve these devices to help users to understand speech in noisy environments and to appreciate music. However, neither approach can mediate a full recovery of hearing sensitivity and/or restoration of the native inner ear sensory epithelia. New therapeutic approaches based on gene transfer and gene editing tools are being developed in animal models. In this review, we focus on the successful restoration of auditory and vestibular functions in certain inner ear conditions, paving the way for future clinical applications. |
topic |
sensory disorders cochlea sensorineural hearing loss gene therapy AAV genome editing |
url |
https://www.mdpi.com/2077-0383/9/7/2309 |
work_keys_str_mv |
AT sedighehdelmaghani innereargenetherapiestakeoffcurrentpromisesandfuturechallenges AT azizelamraoui innereargenetherapiestakeoffcurrentpromisesandfuturechallenges |
_version_ |
1724573919873073152 |