Article
Gene therapy rescues cilia defects and restores olfactory function in a mammalian ciliopathy model.
Nature medicine - 1 Sept 2012
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
Abstract excerpt
Cilia are evolutionarily conserved microtubule-based organelles that are crucial for diverse biological functions, including motility, cell signaling and sensory perception. In humans, alterations in the formation and function of cilia manifest clinically as ciliopathies, a growing class of pleiotropic genetic disorders. Despite the substantial progress that has been made in identifying genes that cause...
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