Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches
Inherited optic neuropathies share visual impairment due to the degeneration of retinal ganglion cells (RGCs) as the hallmark of the disease. This group of genetic disorders are caused by mutations in nuclear genes or in the mitochondrial DNA (mtDNA). An impaired mitochondrial function is the underl...
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doaj-a0ab6114df304406bedd01369af6f1652021-01-19T00:04:38ZengMDPI AGGenes2073-44252021-01-011211211210.3390/genes12010112Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D ApproachesMarta García-López0Joaquín Arenas1M. Esther Gallardo2Grupo de Investigación Traslacional con Células iPS, Instituto de Investigación Sanitaria Hospital 12 de Octubre (i+12), 28041 Madrid, SpainGrupo de Enfermedades Raras, Mitocondriales y Neuromusculares, Instituto de Investigación Sanitaria Hospital 12 de Octubre (i+12), 28041 Madrid, SpainGrupo de Investigación Traslacional con Células iPS, Instituto de Investigación Sanitaria Hospital 12 de Octubre (i+12), 28041 Madrid, SpainInherited optic neuropathies share visual impairment due to the degeneration of retinal ganglion cells (RGCs) as the hallmark of the disease. This group of genetic disorders are caused by mutations in nuclear genes or in the mitochondrial DNA (mtDNA). An impaired mitochondrial function is the underlying mechanism of these diseases. Currently, optic neuropathies lack an effective treatment, and the implementation of induced pluripotent stem cell (iPSC) technology would entail a huge step forward. The generation of iPSC-derived RGCs would allow faithfully modeling these disorders, and these RGCs would represent an appealing platform for drug screening as well, paving the way for a proper therapy. Here, we review the ongoing two-dimensional (2D) and three-dimensional (3D) approaches based on iPSCs and their applications, taking into account the more innovative technologies, which include tissue engineering or microfluidics.https://www.mdpi.com/2073-4425/12/1/112induced pluripotent stem cellsiPSCsiPSoptic neuropathiesoptic atrophymitochondriopathy |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Marta García-López Joaquín Arenas M. Esther Gallardo |
spellingShingle |
Marta García-López Joaquín Arenas M. Esther Gallardo Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches Genes induced pluripotent stem cells iPSCs iPS optic neuropathies optic atrophy mitochondriopathy |
author_facet |
Marta García-López Joaquín Arenas M. Esther Gallardo |
author_sort |
Marta García-López |
title |
Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches |
title_short |
Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches |
title_full |
Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches |
title_fullStr |
Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches |
title_full_unstemmed |
Hereditary Optic Neuropathies: Induced Pluripotent Stem Cell-Based 2D/3D Approaches |
title_sort |
hereditary optic neuropathies: induced pluripotent stem cell-based 2d/3d approaches |
publisher |
MDPI AG |
series |
Genes |
issn |
2073-4425 |
publishDate |
2021-01-01 |
description |
Inherited optic neuropathies share visual impairment due to the degeneration of retinal ganglion cells (RGCs) as the hallmark of the disease. This group of genetic disorders are caused by mutations in nuclear genes or in the mitochondrial DNA (mtDNA). An impaired mitochondrial function is the underlying mechanism of these diseases. Currently, optic neuropathies lack an effective treatment, and the implementation of induced pluripotent stem cell (iPSC) technology would entail a huge step forward. The generation of iPSC-derived RGCs would allow faithfully modeling these disorders, and these RGCs would represent an appealing platform for drug screening as well, paving the way for a proper therapy. Here, we review the ongoing two-dimensional (2D) and three-dimensional (3D) approaches based on iPSCs and their applications, taking into account the more innovative technologies, which include tissue engineering or microfluidics. |
topic |
induced pluripotent stem cells iPSCs iPS optic neuropathies optic atrophy mitochondriopathy |
url |
https://www.mdpi.com/2073-4425/12/1/112 |
work_keys_str_mv |
AT martagarcialopez hereditaryopticneuropathiesinducedpluripotentstemcellbased2d3dapproaches AT joaquinarenas hereditaryopticneuropathiesinducedpluripotentstemcellbased2d3dapproaches AT mesthergallardo hereditaryopticneuropathiesinducedpluripotentstemcellbased2d3dapproaches |
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1724332634531692544 |