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Fit for the Eye: Aptamers in Ocular Disorders
For any new class of therapeutics, there are certain types of indications that represent a natural fit. For nucleic acid ligands in general, and aptamers in particular, the eye has historically been an attractive site for therapeutic intervention. In this review, we recount the discovery and early d...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc.
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4900223/ https://www.ncbi.nlm.nih.gov/pubmed/26757406 http://dx.doi.org/10.1089/nat.2015.0573 |
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author | Drolet, Daniel W. Green, Louis S. Gold, Larry Janjic, Nebojsa |
author_facet | Drolet, Daniel W. Green, Louis S. Gold, Larry Janjic, Nebojsa |
author_sort | Drolet, Daniel W. |
collection | PubMed |
description | For any new class of therapeutics, there are certain types of indications that represent a natural fit. For nucleic acid ligands in general, and aptamers in particular, the eye has historically been an attractive site for therapeutic intervention. In this review, we recount the discovery and early development of three aptamers designated for use in ophthalmology, one approved (Macugen), and two in late-stage development (Fovista and Zimura). Every one of these molecules was originally intended for other indications. Key improvements in technology, specifically with regard to libraries used for in vitro selection and subsequent chemical optimization of aptamers, have played an important role in allowing the identification of development candidates with suitable properties. The lessons learned from the selection of these molecules are valuable for informing us about the many remaining opportunities for aptamer-based therapeutics in ophthalmology as well as for identifying additional indications for which aptamers as a class of therapeutics have distinct advantages. |
format | Online Article Text |
id | pubmed-4900223 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | Mary Ann Liebert, Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-49002232016-06-14 Fit for the Eye: Aptamers in Ocular Disorders Drolet, Daniel W. Green, Louis S. Gold, Larry Janjic, Nebojsa Nucleic Acid Ther Reviews For any new class of therapeutics, there are certain types of indications that represent a natural fit. For nucleic acid ligands in general, and aptamers in particular, the eye has historically been an attractive site for therapeutic intervention. In this review, we recount the discovery and early development of three aptamers designated for use in ophthalmology, one approved (Macugen), and two in late-stage development (Fovista and Zimura). Every one of these molecules was originally intended for other indications. Key improvements in technology, specifically with regard to libraries used for in vitro selection and subsequent chemical optimization of aptamers, have played an important role in allowing the identification of development candidates with suitable properties. The lessons learned from the selection of these molecules are valuable for informing us about the many remaining opportunities for aptamer-based therapeutics in ophthalmology as well as for identifying additional indications for which aptamers as a class of therapeutics have distinct advantages. Mary Ann Liebert, Inc. 2016-06-01 /pmc/articles/PMC4900223/ /pubmed/26757406 http://dx.doi.org/10.1089/nat.2015.0573 Text en © Daniel W. Drolet, et al., 2016; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. |
spellingShingle | Reviews Drolet, Daniel W. Green, Louis S. Gold, Larry Janjic, Nebojsa Fit for the Eye: Aptamers in Ocular Disorders |
title | Fit for the Eye: Aptamers in Ocular Disorders |
title_full | Fit for the Eye: Aptamers in Ocular Disorders |
title_fullStr | Fit for the Eye: Aptamers in Ocular Disorders |
title_full_unstemmed | Fit for the Eye: Aptamers in Ocular Disorders |
title_short | Fit for the Eye: Aptamers in Ocular Disorders |
title_sort | fit for the eye: aptamers in ocular disorders |
topic | Reviews |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4900223/ https://www.ncbi.nlm.nih.gov/pubmed/26757406 http://dx.doi.org/10.1089/nat.2015.0573 |
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