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Highly sensitive measurements of disease progression in rare disorders: Developing and validating a multimodal model of retinal degeneration in Stargardt disease

BACKGROUND: Each inherited retinal disorder is rare, but together, they affect millions of people worldwide. No treatment is currently available for these blinding diseases, but promising new options—including gene therapy—are emerging. Arguably, the most prevalent retinal dystrophy is Stargardt dis...

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Bibliographic Details
Main Authors: Lambertus, Stanley, Bax, Nathalie M., Fakin, Ana, Groenewoud, Joannes M. M., Klevering, B. Jeroen, Moore, Anthony T., Michaelides, Michel, Webster, Andrew R., van der Wilt, Gert Jan, Hoyng, Carel B.
Format: Online Article Text
Language:English
Published: Public Library of Science 2017
Subjects:
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5371327/
https://www.ncbi.nlm.nih.gov/pubmed/28355279
http://dx.doi.org/10.1371/journal.pone.0174020