. Flotte, Terence R., and Beth L. Laube. Gene Therapy in Cystic Fibrosis(*). 120 Vol. , 2001.
ABSTRACT. Cystic fibrosis (CF) is a common genetic disorder characterized by defective epithelial chloride transport and progressive lung disease. Although great strides have been made in the treatment of CF, it remains lethal, often by early adulthood. CF is one of the most extensively researched genetic diseases as a.
Gene delivery systems (GDS) play a central role in the development of gene therapy strategies for Cystic Fibrosis (CF). Further, these systems are important tools in studies with cultured cells and in animal models. In this review, we describe the properties of several viral and synthetic gene delivery systems, and evaluate
24 Jan 2014 Since the cloning of the Cystic Fibrosis. Transmembrane Conductance Regulator (CFTR) gene in. 1989, there has been significant interest in the possibility of gene therapy as a treatment for CF. Early studies using viral vectors carrying a healthy CFTR plasmid highlighted the difficulties with overcoming the

Cystic Fibrosis (CF) is an autosomal recessive, life-limiting disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The landscape in cystic fibrosis care has changed markedly with currently more adult patients than children in many countries. Over 2000 different mu- tations in the CFTR gene have been reported and the majority are extremely rare. Understanding how CFTR mu- tations translate to disturbed synthesis or function of the
12 Aug 2016 Acknowledgements: The authors thank Samia Soussi for her help with preparing the manuscript. Our research was supported by the MRC-DPFS programme, the Cystic. Fibrosis Trust, Just Gene Therapy, Medicor Foundation and ERANDA. The studies were supported by the. National Institute for Health
Gene therapy for cystic fibrosis. Get a printable copy (PDF file) of the complete article (1000K), or click on a page image below to browse page by page. Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer.

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February 2018