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Novel gene variants in Polish patients with Leber congenital amaurosis (LCA)

BACKGROUND: Leber congenital amaurosis (LCA) is a rare retinal disease that is the most frequent cause of congenital blindness in children and the most severe form of inherited retinal dystrophies. To date, 25 genes have been implicated in the pathogenesis of LCA. As gene therapy is becoming availab...

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Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: Skorczyk-Werner, Anna, Niedziela, Zuzanna, Stopa, Marcin, Krawczyński, Maciej Robert
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2020
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7731562/
https://ncbi.nlm.nih.gov/pubmed/33308271
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01634-y
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