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2016 ; 26
(3
): 127-46
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Fit for the Eye: Aptamers in Ocular Disorders
#MMPMID26757406
Drolet DW
; Green LS
; Gold L
; Janjic N
Nucleic Acid Ther
2016[Jun]; 26
(3
): 127-46
PMID26757406
show ga
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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