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Developing Non-Human Primate Models of Inherited Retinal Diseases

Inherited retinal diseases (IRDs) represent a genetically and clinically heterogenous group of diseases that can eventually lead to blindness. Advances in sequencing technologies have resulted in better molecular characterization and genotype–phenotype correlation of IRDs. This has fueled research i...

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Autores principales: Seah, Ivan, Goh, Debbie, Chan, Hwei Wuen, Su, Xinyi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8872446/
https://www.ncbi.nlm.nih.gov/pubmed/35205388
http://dx.doi.org/10.3390/genes13020344
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author Seah, Ivan
Goh, Debbie
Chan, Hwei Wuen
Su, Xinyi
author_facet Seah, Ivan
Goh, Debbie
Chan, Hwei Wuen
Su, Xinyi
author_sort Seah, Ivan
collection PubMed
description Inherited retinal diseases (IRDs) represent a genetically and clinically heterogenous group of diseases that can eventually lead to blindness. Advances in sequencing technologies have resulted in better molecular characterization and genotype–phenotype correlation of IRDs. This has fueled research into therapeutic development over the recent years. Animal models are required for pre-clinical efficacy assessment. Non-human primates (NHP) are ideal due to the anatomical and genetic similarities shared with humans. However, developing NHP disease to recapitulate the disease phenotype for specific IRDs may be challenging from both technical and cost perspectives. This review discusses the currently available NHP IRD models and the methods used for development, with a particular focus on gene-editing technologies.
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spelling pubmed-88724462022-02-25 Developing Non-Human Primate Models of Inherited Retinal Diseases Seah, Ivan Goh, Debbie Chan, Hwei Wuen Su, Xinyi Genes (Basel) Review Inherited retinal diseases (IRDs) represent a genetically and clinically heterogenous group of diseases that can eventually lead to blindness. Advances in sequencing technologies have resulted in better molecular characterization and genotype–phenotype correlation of IRDs. This has fueled research into therapeutic development over the recent years. Animal models are required for pre-clinical efficacy assessment. Non-human primates (NHP) are ideal due to the anatomical and genetic similarities shared with humans. However, developing NHP disease to recapitulate the disease phenotype for specific IRDs may be challenging from both technical and cost perspectives. This review discusses the currently available NHP IRD models and the methods used for development, with a particular focus on gene-editing technologies. MDPI 2022-02-14 /pmc/articles/PMC8872446/ /pubmed/35205388 http://dx.doi.org/10.3390/genes13020344 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/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 (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Seah, Ivan
Goh, Debbie
Chan, Hwei Wuen
Su, Xinyi
Developing Non-Human Primate Models of Inherited Retinal Diseases
title Developing Non-Human Primate Models of Inherited Retinal Diseases
title_full Developing Non-Human Primate Models of Inherited Retinal Diseases
title_fullStr Developing Non-Human Primate Models of Inherited Retinal Diseases
title_full_unstemmed Developing Non-Human Primate Models of Inherited Retinal Diseases
title_short Developing Non-Human Primate Models of Inherited Retinal Diseases
title_sort developing non-human primate models of inherited retinal diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8872446/
https://www.ncbi.nlm.nih.gov/pubmed/35205388
http://dx.doi.org/10.3390/genes13020344
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