Article
Gene editing of DNAH11 restores normal cilia motility in primary ciliary dyskinesia.
Journal of medical genetics - 1 Apr 2016
Lai Michele, Pifferi Massimo, Bush Andrew, Piras Martina, Michelucci Angela, Di Cicco Maria, del Grosso Ambra, Quaranta Paola, Cursi Chiara, Tantillo Elena, Franceschi Sara, Mazzanti Maria Chiara, Simi Paolo, Saggese Giuseppe, Boner Attilio, Pistello Mauro
Abstract excerpt
BACKGROUND: Primary ciliary dyskinesia (PCD) is a rare autosomal recessive genetic disorder characterised by dysfunction of motile cilia. Ciliary dysmotility causes poor mucociliary clearance and leads to impairment of pulmonary function and severe respiratory infections. PCD has no specific therapy. With the aim to permanently restore gene function and normalise ciliary motility, we used gene editing to replace...
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