Gene Therapy

Research from Johns Hopkins University, Medical Department has provided new data on gene therapy

Published in Gene Therapy Weekly, October 23rd, 2008

"We previously generated an adenoassociated viral gene therapy vector, rAAV-Delta 264 cystic fibrosis transmembrane conductance regulator (CFTR), missing the first four transmembrane domains of CFTR. When infected into monkey lungs, Delta 264 CFTR increased the levels of endogenous wild type CFTR protein," researchers in the United States report.

"To understand this process, we transfected Delta 264 CFTR plasmid cDNA into COS7 cells, and we noted that protein expression from the truncation mutant is barely detectable when compared with wild type or Delta F508 CFTR. Delta 264 CFTR protein expression increases dramatically when cells are treated with proteasome...

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Source: Gene Therapy Weekly (2008-10-23)

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