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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...

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Detalles Bibliográficos
Autores principales: Drolet, Daniel W., Green, Louis S., Gold, Larry, Janjic, Nebojsa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2016
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.
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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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