Cargando…
Genetics and therapy for pediatric eye diseases
Ocular morphogenesis in vertebrates is a highly organized process, orchestrated largely by intrinsic genetic programs that exhibit stringent spatiotemporal control. Alternations in these genetic instructions can lead to hereditary or nonhereditary congenital disorders, a major cause of childhood vis...
Autores principales: | , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8122153/ https://www.ncbi.nlm.nih.gov/pubmed/33975254 http://dx.doi.org/10.1016/j.ebiom.2021.103360 |
_version_ | 1783692527673540608 |
---|---|
author | Chen, Holly.Y. Lehmann, Ordan J. Swaroop, Anand |
author_facet | Chen, Holly.Y. Lehmann, Ordan J. Swaroop, Anand |
author_sort | Chen, Holly.Y. |
collection | PubMed |
description | Ocular morphogenesis in vertebrates is a highly organized process, orchestrated largely by intrinsic genetic programs that exhibit stringent spatiotemporal control. Alternations in these genetic instructions can lead to hereditary or nonhereditary congenital disorders, a major cause of childhood visual impairment, and contribute to common late-onset blinding diseases. Currently, limited treatment options exist for clinical phenotypes involving eye development. This review summarizes recent advances in our understanding of early-onset ocular disorders and highlights genetic complexities in development and diseases, specifically focusing on coloboma, congenital glaucoma and Leber congenital amaurosis. We also discuss innovative paradigms for potential therapeutic modalities. |
format | Online Article Text |
id | pubmed-8122153 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Elsevier |
record_format | MEDLINE/PubMed |
spelling | pubmed-81221532021-05-21 Genetics and therapy for pediatric eye diseases Chen, Holly.Y. Lehmann, Ordan J. Swaroop, Anand EBioMedicine Review Ocular morphogenesis in vertebrates is a highly organized process, orchestrated largely by intrinsic genetic programs that exhibit stringent spatiotemporal control. Alternations in these genetic instructions can lead to hereditary or nonhereditary congenital disorders, a major cause of childhood visual impairment, and contribute to common late-onset blinding diseases. Currently, limited treatment options exist for clinical phenotypes involving eye development. This review summarizes recent advances in our understanding of early-onset ocular disorders and highlights genetic complexities in development and diseases, specifically focusing on coloboma, congenital glaucoma and Leber congenital amaurosis. We also discuss innovative paradigms for potential therapeutic modalities. Elsevier 2021-05-08 /pmc/articles/PMC8122153/ /pubmed/33975254 http://dx.doi.org/10.1016/j.ebiom.2021.103360 Text en https://creativecommons.org/licenses/by/4.0/This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Chen, Holly.Y. Lehmann, Ordan J. Swaroop, Anand Genetics and therapy for pediatric eye diseases |
title | Genetics and therapy for pediatric eye diseases |
title_full | Genetics and therapy for pediatric eye diseases |
title_fullStr | Genetics and therapy for pediatric eye diseases |
title_full_unstemmed | Genetics and therapy for pediatric eye diseases |
title_short | Genetics and therapy for pediatric eye diseases |
title_sort | genetics and therapy for pediatric eye diseases |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8122153/ https://www.ncbi.nlm.nih.gov/pubmed/33975254 http://dx.doi.org/10.1016/j.ebiom.2021.103360 |
work_keys_str_mv | AT chenhollyy geneticsandtherapyforpediatriceyediseases AT lehmannordanj geneticsandtherapyforpediatriceyediseases AT swaroopanand geneticsandtherapyforpediatriceyediseases |