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Clinical and preclinical therapeutic outcome metrics for USH2A-related disease

USH2A variants are the most common cause of Usher syndrome type 2, characterized by congenital sensorineural hearing loss and retinitis pigmentosa (RP), and also contribute to autosomal recessive non-syndromic RP. Several treatment strategies are under development; however, sensitive clinical trial...

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Detalles Bibliográficos
Autores principales:	Toms, Maria, Dubis, Adam M, de Vrieze, Erik, Tracey-White, Dhani, Mitsios, Andreas, Hayes, Matthew, Broekman, Sanne, Baxendale, Sarah, Utoomprurkporn, Nattawan, Bamiou, Doris, Bitner-Glindzicz, Maria, Webster, Andrew R, Van Wijk, Erwin, Moosajee, Mariya
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2020
Materias:	General Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7372554/ https://www.ncbi.nlm.nih.gov/pubmed/31998945 http://dx.doi.org/10.1093/hmg/ddaa004

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