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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Pubblicato in:PLoS One
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2017
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5371327/
https://ncbi.nlm.nih.gov/pubmed/28355279
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0174020
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