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Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has bee...

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Autores principales: Huang, Di, Fletcher, Sue, Wilton, Steve D., Palmer, Norman, McLenachan, Samuel, Mackey, David A., Chen, Fred K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6836112/
https://www.ncbi.nlm.nih.gov/pubmed/31740647
http://dx.doi.org/10.3390/vision1030022
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author Huang, Di
Fletcher, Sue
Wilton, Steve D.
Palmer, Norman
McLenachan, Samuel
Mackey, David A.
Chen, Fred K.
author_facet Huang, Di
Fletcher, Sue
Wilton, Steve D.
Palmer, Norman
McLenachan, Samuel
Mackey, David A.
Chen, Fred K.
author_sort Huang, Di
collection PubMed
description Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has been made over the last two decades in identifying the many genes implicated in inherited retinal diseases and developing novel therapies to address the underlying genetic defects. Approximately one-quarter of exonic mutations related to human inherited diseases are likely to induce aberrant splicing products, providing opportunities for the development of novel therapeutics that target splicing processes. The feasibility of antisense oligomer mediated splice intervention to treat inherited diseases has been demonstrated in vitro, in vivo and in clinical trials. In this review, we will discuss therapeutic approaches to treat inherited retinal disease, including strategies to correct splicing and modify exon selection at the level of pre-mRNA. The challenges of clinical translation of this class of emerging therapeutics will also be discussed.
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spelling pubmed-68361122019-11-14 Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid Huang, Di Fletcher, Sue Wilton, Steve D. Palmer, Norman McLenachan, Samuel Mackey, David A. Chen, Fred K. Vision (Basel) Review Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has been made over the last two decades in identifying the many genes implicated in inherited retinal diseases and developing novel therapies to address the underlying genetic defects. Approximately one-quarter of exonic mutations related to human inherited diseases are likely to induce aberrant splicing products, providing opportunities for the development of novel therapeutics that target splicing processes. The feasibility of antisense oligomer mediated splice intervention to treat inherited diseases has been demonstrated in vitro, in vivo and in clinical trials. In this review, we will discuss therapeutic approaches to treat inherited retinal disease, including strategies to correct splicing and modify exon selection at the level of pre-mRNA. The challenges of clinical translation of this class of emerging therapeutics will also be discussed. MDPI 2017-09-01 /pmc/articles/PMC6836112/ /pubmed/31740647 http://dx.doi.org/10.3390/vision1030022 Text en © 2017 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Huang, Di
Fletcher, Sue
Wilton, Steve D.
Palmer, Norman
McLenachan, Samuel
Mackey, David A.
Chen, Fred K.
Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title_full Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title_fullStr Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title_full_unstemmed Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title_short Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
title_sort inherited retinal disease therapies targeting precursor messenger ribonucleic acid
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6836112/
https://www.ncbi.nlm.nih.gov/pubmed/31740647
http://dx.doi.org/10.3390/vision1030022
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