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Retinal disease in ciliopathies: Recent advances with a focus on stem cell-based therapies

Ciliopathies display extensive genetic and clinical heterogeneity, varying in severity, age of onset, disease progression and organ systems affected. Retinal involvement, as demonstrated by photoreceptor dysfunction or death, is a highly penetrant phenotype among a vast majority of ciliopathies. Pho...

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Détails bibliographiques
Publié dans:Transl Sci Rare Dis
Auteurs principaux: Chen, Holly Yu, Welby, Emily, Li, Tiansen, Swaroop, Anand
Format: Artigo
Langue:Inglês
Publié: IOS Press 2019
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6839492/
https://ncbi.nlm.nih.gov/pubmed/31763178
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3233/TRD-190038
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