Article
Rapid quantitation of gene therapy specific CFTR expression using the amplification refractory mutation system.
BioTechniques - 1 Jul 1999
Thorpe P H, Porteous D J
Abstract excerpt
Gene therapy offers the potential of correcting genetic disorders such as cystic fibrosis (CF). By complementing the non-functional endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene with a functional transgene, we anticipate it may alleviate the disease phenotype. All approaches to CF gene therapy rely upon sensitive assays to monitor delivery, expression and maintenance of CFTR vectors....
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