Gene editing of DNAH11 restores normal cilia motility in primary ciliary dyskinesia

Journal of Medical Genetics

Volumn 53, Issue 4, 2016, Pages 242-249

  Lai, Michele ^a   Pifferi, Massimo ^b   Bush, Andrew ^c   Piras, Martina ^b   Michelucci, Angela ^b   Di Cicco, Maria ^b   del Grosso, Ambra ^a   Quaranta, Paola ^a,d   Cursi, Chiara ^a   Tantillo, Elena ^a   Franceschi, Sara ^e   Mazzanti, Maria Chiara ^e   Simi, Paolo ^b   Saggese, Giuseppe ^b   Boner, Attilio ^f   Pistello, Mauro ^a  

^a UNIVERSITY OF PISA   (Italy)

^b UNIVERSITY HOSPITAL OF PISA   (Italy)

^c ROYAL BROMPTON HOSPITAL   (United Kingdom)

^d ARPA Foundation   (Italy)

^e Mass Spectrometry and Proteomics   (Italy)

^f UNIVERSITY OF VERONA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

DYNEIN ADENOSINE TRIPHOSPHATASE; DYNEIN HEAVY CHAIN 11; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; AXONEMAL DYNEIN; DNAH11 PROTEIN, HUMAN;

ARTICLE; CASE REPORT; CILIARY DYSKINESIA; CILIARY MOTILITY; CILIATED EPITHELIUM CELL; CONTROLLED STUDY; EX VIVO STUDY; GENE FUNCTION; GENE MUTATION; GENE SEQUENCE; GENE TARGETING; GENETIC DISORDER; GENETIC RECOMBINATION; HUMAN; HUMAN CELL; NONSENSE MUTATION; PRIORITY JOURNAL; PROTEIN CLEAVAGE; ADOLESCENT; CELL LINE; CELL MOTION; EPITHELIUM CELL; EUKARYOTIC FLAGELLUM; GENE EDITING; GENE THERAPY; GENETICS; GENOTYPE; KARTAGENER SYNDROME; LENTIVIRINAE; MALE; METABOLISM; PATHOLOGY; PHENOTYPE; TWINS;

ADOLESCENT; AXONEMAL DYNEINS; CELL LINE; CELL MOVEMENT; CILIA; EPITHELIAL CELLS; GENE EDITING; GENETIC THERAPY; GENOTYPE; HUMANS; KARTAGENER SYNDROME; LENTIVIRUS; MALE; PHENOTYPE; TWINS;

EID: 84962881975     PISSN: 00222593     EISSN: 14686244     Source Type: Journal    
DOI: 10.1136/jmedgenet-2015-103539     Document Type: Article

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