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Genetic Hearing Loss and Gene Therapy

Genetic hearing loss crosses almost all the categories of hearing loss which includes the following: conductive, sensory, and neural; syndromic and nonsyndromic; congenital, progressive, and adult onset; high-frequency, low-frequency, or mixed frequency; mild or profound; and recessive, dominant, or...

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Detalles Bibliográficos
Autores principales: Carpena, Nathanial T, Lee, Min Young
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Korea Genome Organization 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6440668/
https://www.ncbi.nlm.nih.gov/pubmed/30602081
http://dx.doi.org/10.5808/GI.2018.16.4.e20
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author Carpena, Nathanial T
Lee, Min Young
author_facet Carpena, Nathanial T
Lee, Min Young
author_sort Carpena, Nathanial T
collection PubMed
description Genetic hearing loss crosses almost all the categories of hearing loss which includes the following: conductive, sensory, and neural; syndromic and nonsyndromic; congenital, progressive, and adult onset; high-frequency, low-frequency, or mixed frequency; mild or profound; and recessive, dominant, or sex-linked. Genes play a role in almost half of all cases of hearing loss but effective treatment options are very limited. Genetic hearing loss is considered to be extremely genetically heterogeneous. The advancements in genomics have been instrumental to the identification of more than 6,000 causative variants in more than 150 genes causing hearing loss. Identification of genes for hearing impairment provides an increased insight into the normal development and function of cells in the auditory system. These defective genes will ultimately be important therapeutic targets. However, the auditory system is extremely complex which requires tremendous advances in gene therapy including gene vectors, routes of administration, and therapeutic approaches. This review summarizes and discusses recent advances in elucidating the genomics of genetic hearing loss and technologies aimed at developing a gene therapy that may become a treatment option for in the near future.
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spelling pubmed-64406682019-04-03 Genetic Hearing Loss and Gene Therapy Carpena, Nathanial T Lee, Min Young Genomics Inform Review Article Genetic hearing loss crosses almost all the categories of hearing loss which includes the following: conductive, sensory, and neural; syndromic and nonsyndromic; congenital, progressive, and adult onset; high-frequency, low-frequency, or mixed frequency; mild or profound; and recessive, dominant, or sex-linked. Genes play a role in almost half of all cases of hearing loss but effective treatment options are very limited. Genetic hearing loss is considered to be extremely genetically heterogeneous. The advancements in genomics have been instrumental to the identification of more than 6,000 causative variants in more than 150 genes causing hearing loss. Identification of genes for hearing impairment provides an increased insight into the normal development and function of cells in the auditory system. These defective genes will ultimately be important therapeutic targets. However, the auditory system is extremely complex which requires tremendous advances in gene therapy including gene vectors, routes of administration, and therapeutic approaches. This review summarizes and discusses recent advances in elucidating the genomics of genetic hearing loss and technologies aimed at developing a gene therapy that may become a treatment option for in the near future. Korea Genome Organization 2018-12 2018-12-28 /pmc/articles/PMC6440668/ /pubmed/30602081 http://dx.doi.org/10.5808/GI.2018.16.4.e20 Text en Copyright © 2018 by Korea Genome Organization It is identical to the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/).
spellingShingle Review Article
Carpena, Nathanial T
Lee, Min Young
Genetic Hearing Loss and Gene Therapy
title Genetic Hearing Loss and Gene Therapy
title_full Genetic Hearing Loss and Gene Therapy
title_fullStr Genetic Hearing Loss and Gene Therapy
title_full_unstemmed Genetic Hearing Loss and Gene Therapy
title_short Genetic Hearing Loss and Gene Therapy
title_sort genetic hearing loss and gene therapy
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6440668/
https://www.ncbi.nlm.nih.gov/pubmed/30602081
http://dx.doi.org/10.5808/GI.2018.16.4.e20
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