UMass Chan Affiliations
Department of PediatricsDocument Type
Journal ArticlePublication Date
2008-12-01Keywords
AnimalsCystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator
*Gene Therapy
Genes, Recessive
Genetic Predisposition to Disease
Genetic Vectors
Humans
Immunity, Cellular
Mutagenesis, Site-Directed
Protein Transport
Signal Transduction
Stem Cells
Targeted Gene Repair
Allergy and Immunology
Genetics and Genomics
Pediatrics
Respiratory Tract Diseases
UMCCTS funding
Metadata
Show full item recordAbstract
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane conductance regulator (CFTR) gene that lead to defective ion transport in the conducting pulmonary airways and exocrine glands. Through a process that is not fully understood, CFTR defects predispose affected patients to chronic endobronchial infections with organisms such as Pseudomonas aeruginosa and Staphylococcus aureus. Following the discovery of the CFTR gene in 1989, CF became one of the primary targets for gene therapy research. Early enthusiasm surrounded the new field of gene therapy during most of the 1990s and it led academics and clinicians on a big effort to apply gene therapy for cystic fibrosis. Clinical studies have been pursued using recombinant adenovirus, recombinant adeno-associated virus, cationic liposomes, and cationic polymer vectors. Although to this date no dramatic therapeutic benefits have been observed, a lot of information has been gained from the pre-clinical and clinical studies that were performed. This learning curve has led to the optimization of vector technology and an appreciation of immune and mechanical barriers that have to be overcome for successful delivery.Source
Clin Rev Allergy Immunol. 2008 Dec;35(3):164-78. Link to article on publisher's site
DOI
10.1007/s12016-008-8080-3Permanent Link to this Item
http://hdl.handle.net/20.500.14038/43819PubMed ID
18600482Related Resources
ae974a485f413a2113503eed53cd6c53
10.1007/s12016-008-8080-3