Gene therapy rescues cilia defects and restores olfactory function in a mammalian ciliopathy model

Cilia are evolutionarily conserved microtubule-based organelles that are crucial for diverse biological functions, including motility, cell signaling and sensory perception(1). In humans, alterations in the formation and function of cilia manifest clinically as ciliopathies, a growing class of pleio...

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Hauptverfasser: McIntyre, Jeremy C, Davis, Erica E, Joiner, Ariell, Williams, Corey L, Tsai, I-Chun, Jenkins, Paul M, McEwen, Dyke P, Zhang, Lian, Escobado, John, Thomas, Sophie, Szymanska, Katarzyna, Johnson, Colin A, Beales, Philip L, Green, Eric D, Mullikin, James C, Sabo, Aniko, Muzny, Donna M, Gibbs, Richard A, Attié-Bitach, Tania, Yoder, Bradley K, Reed, Randall R, Katsanis, Nicholas, Martens, Jeffrey R
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2012
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3645984/
https://ncbi.nlm.nih.gov/pubmed/22941275
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nm.2860
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