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Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy
Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively inves...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Korean Society for Brain and Neural Sciences
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7344374/ https://www.ncbi.nlm.nih.gov/pubmed/32624504 http://dx.doi.org/10.5607/en20004 |
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author | Thenmozhi, Rajarathinam Lee, Ji-Su Park, Na Young Choi, Byung-Ok Hong, Young Bin |
author_facet | Thenmozhi, Rajarathinam Lee, Ji-Su Park, Na Young Choi, Byung-Ok Hong, Young Bin |
author_sort | Thenmozhi, Rajarathinam |
collection | PubMed |
description | Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively investigated, likely due to the complexities of inheritance in IPN. Indeed, because the majority of the causative mutations of IPN lead to gain-of-function rather than loss-of-function, developing a therapeutic strategy is more difficult, especially considering gene therapy for genetic diseases began with the simple idea of replacing a defective gene with a functional copy. Recent advances in gene manipulation technology have brought novel approaches to gene therapy and its clinical application for IPN treatment. For example, in addition to the classically used gene replacement for mutant genes in recessively inherited IPN, other techniques including gene addition to modify the disease phenotype, modulations of target gene expression, and techniques to edit mutant genes have been developed and evaluated as potent therapeutic strategies for dominantly inherited IPN. In this review, the current status of gene therapy for IPN and future perspectives will be discussed. |
format | Online Article Text |
id | pubmed-7344374 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | The Korean Society for Brain and Neural Sciences |
record_format | MEDLINE/PubMed |
spelling | pubmed-73443742020-07-17 Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy Thenmozhi, Rajarathinam Lee, Ji-Su Park, Na Young Choi, Byung-Ok Hong, Young Bin Exp Neurobiol Review Article Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively investigated, likely due to the complexities of inheritance in IPN. Indeed, because the majority of the causative mutations of IPN lead to gain-of-function rather than loss-of-function, developing a therapeutic strategy is more difficult, especially considering gene therapy for genetic diseases began with the simple idea of replacing a defective gene with a functional copy. Recent advances in gene manipulation technology have brought novel approaches to gene therapy and its clinical application for IPN treatment. For example, in addition to the classically used gene replacement for mutant genes in recessively inherited IPN, other techniques including gene addition to modify the disease phenotype, modulations of target gene expression, and techniques to edit mutant genes have been developed and evaluated as potent therapeutic strategies for dominantly inherited IPN. In this review, the current status of gene therapy for IPN and future perspectives will be discussed. The Korean Society for Brain and Neural Sciences 2020-06-30 2020-06-30 /pmc/articles/PMC7344374/ /pubmed/32624504 http://dx.doi.org/10.5607/en20004 Text en Copyright © Experimental Neurobiology 2020 This is an open-access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Review Article Thenmozhi, Rajarathinam Lee, Ji-Su Park, Na Young Choi, Byung-Ok Hong, Young Bin Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title | Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title_full | Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title_fullStr | Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title_full_unstemmed | Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title_short | Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy |
title_sort | gene therapy options as new treatment for inherited peripheral neuropathy |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7344374/ https://www.ncbi.nlm.nih.gov/pubmed/32624504 http://dx.doi.org/10.5607/en20004 |
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