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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...
Hauptverfasser: | , , , , , , , , , |
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Format: | Online Artikel Text |
Sprache: | English |
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Public Library of Science
2017
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Online Zugang: | 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 |