Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in pompe disease: Initial safety and ventilatory outcomes

Human Gene Therapy

Volumn 24, Issue 6, 2013, Pages 630-640

  Smith, Barbara K ^a   Collins, Shelley W ^b   Conlon, Thomas J ^a,b   Mah, Cathryn S ^a,b   Lawson, Lee Ann ^b   Martin, Anatole D ^a   Fuller, David D ^a   Cleaver, Brian D ^a,b   Clément, Nathalie ^a,b   Phillips, Dawn ^b   Islam, Saleem ^b   Dobjia, Nicole ^b   Byrne, Barry J ^a,b  

^a UNIVERSITY OF FLORIDA   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA GLUCOSIDASE; ALPHA GLUCOSIDASE ANTIBODY; ANTIBODY; UNCLASSIFIED DRUG;

ABDOMINAL PAIN; ARTICLE; ARTIFICIAL VENTILATION; BREATHING; CHEST TENDERNESS; CHILD; CHRONIC RESPIRATORY FAILURE; DIAPHRAGM; DRUG ADMINISTRATION ROUTE; DRUG SAFETY; EDEMA; ENZYME REPLACEMENT; EXERCISE; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; INTRADIAPHRAGMATIC DRUG ADMINISTRATION; MAJOR CLINICAL STUDY; MALE; PRESCHOOL CHILD; SCHOOL CHILD; THORAX DISEASE;

ADOLESCENT; ALPHA-GLUCOSIDASES; ANTIBODIES; CHILD, PRESCHOOL; DEPENDOVIRUS; DIAPHRAGM; FEMALE; GENETIC THERAPY; GENETIC VECTORS; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNITY, CELLULAR; INFANT; MALE; POSTOPERATIVE CARE; PREOPERATIVE CARE; PULMONARY VENTILATION; RESISTANCE TRAINING; RESPIRATORY INSUFFICIENCY; T-LYMPHOCYTES; TREATMENT OUTCOME;

ADENO-ASSOCIATED VIRUS; MIRIDAE;

EID: 84879161464     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.250     Document Type: Article

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