The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

Inherited retinal degenerations (IRDs) are a diverse group of conditions that are often characterized by the loss of photoreceptors and blindness. Recent innovations in molecular biology and genomics have allowed us to identify the causative defects behind these dystrophies and to design therapeutic...

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Autores principales:	Luis A. Martinez Velazquez, Brian G. Ballios
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	CRISPR gene editing gene therapy inherited retinal disease retina neuroprotection Biology (General) QH301-705.5 Chemistry QD1-999
Acceso en línea:	https://doaj.org/article/57fe87b80315410892a4f5e17fdcc0e3
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spelling	oai:doaj.org-article:57fe87b80315410892a4f5e17fdcc0e32021-11-11T17:00:13ZThe Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease10.3390/ijms2221115421422-00671661-6596https://doaj.org/article/57fe87b80315410892a4f5e17fdcc0e32021-10-01T00:00:00Zhttps://www.mdpi.com/1422-0067/22/21/11542https://doaj.org/toc/1661-6596https://doaj.org/toc/1422-0067Inherited retinal degenerations (IRDs) are a diverse group of conditions that are often characterized by the loss of photoreceptors and blindness. Recent innovations in molecular biology and genomics have allowed us to identify the causative defects behind these dystrophies and to design therapeutics that target specific mechanisms of retinal disease. Recently, the FDA approved the first in vivo gene therapy for one of these hereditary blinding conditions. Current clinical trials are exploring new therapies that could provide treatment for a growing number of retinal dystrophies. While the field has had early success with gene augmentation strategies for treating retinal disease based on loss-of-function mutations, many novel approaches hold the promise of offering therapies that span the full spectrum of causative mutations and mechanisms. Here, we provide a comprehensive review of the approaches currently in development including a discussion of retinal neuroprotection, gene therapies (gene augmentation, gene editing, RNA modification, optogenetics), and regenerative stem or precursor cell-based therapies. Our review focuses on technologies that are being developed for clinical translation or are in active clinical trials and discusses the advantages and limitations for each approach.Luis A. Martinez VelazquezBrian G. BalliosMDPI AGarticleCRISPRgene editinggene therapyinherited retinal diseaseretinaneuroprotectionBiology (General)QH301-705.5ChemistryQD1-999ENInternational Journal of Molecular Sciences, Vol 22, Iss 11542, p 11542 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	CRISPR gene editing gene therapy inherited retinal disease retina neuroprotection Biology (General) QH301-705.5 Chemistry QD1-999
spellingShingle	CRISPR gene editing gene therapy inherited retinal disease retina neuroprotection Biology (General) QH301-705.5 Chemistry QD1-999 Luis A. Martinez Velazquez Brian G. Ballios The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
description	Inherited retinal degenerations (IRDs) are a diverse group of conditions that are often characterized by the loss of photoreceptors and blindness. Recent innovations in molecular biology and genomics have allowed us to identify the causative defects behind these dystrophies and to design therapeutics that target specific mechanisms of retinal disease. Recently, the FDA approved the first in vivo gene therapy for one of these hereditary blinding conditions. Current clinical trials are exploring new therapies that could provide treatment for a growing number of retinal dystrophies. While the field has had early success with gene augmentation strategies for treating retinal disease based on loss-of-function mutations, many novel approaches hold the promise of offering therapies that span the full spectrum of causative mutations and mechanisms. Here, we provide a comprehensive review of the approaches currently in development including a discussion of retinal neuroprotection, gene therapies (gene augmentation, gene editing, RNA modification, optogenetics), and regenerative stem or precursor cell-based therapies. Our review focuses on technologies that are being developed for clinical translation or are in active clinical trials and discusses the advantages and limitations for each approach.
format	article
author	Luis A. Martinez Velazquez Brian G. Ballios
author_facet	Luis A. Martinez Velazquez Brian G. Ballios
author_sort	Luis A. Martinez Velazquez
title	The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
title_short	The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
title_full	The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
title_fullStr	The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
title_full_unstemmed	The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease
title_sort	next generation of molecular and cellular therapeutics for inherited retinal disease
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/57fe87b80315410892a4f5e17fdcc0e3
work_keys_str_mv	AT luisamartinezvelazquez thenextgenerationofmolecularandcellulartherapeuticsforinheritedretinaldisease AT briangballios thenextgenerationofmolecularandcellulartherapeuticsforinheritedretinaldisease AT luisamartinezvelazquez nextgenerationofmolecularandcellulartherapeuticsforinheritedretinaldisease AT briangballios nextgenerationofmolecularandcellulartherapeuticsforinheritedretinaldisease
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The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

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