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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea: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

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