Therapy for cystic fibrosis

Anson DS, Smith GJ, Parsons DW. 2006. Gene therapy for cystic fibrosis airway disease - Is clinical success imminent Curr Gene Ther. 6 161-179. 

Boucher RC. 1999. Status of gene therapy for cystic fibrosis lung disease. J Clin Inv. 103 441-445. 

Dodge JA. 1998. Gene therapy for cystic fibrosis What message for the recipient Thorax. 53 157-158. 

Rosenecker J, Huth S, Rudolph C. 2006. Gene therapy for cystic fibrosis lung disease Current status and future perspectives. Curr Opin Mol Ther. 8 439 145. 

White AF, Ponnazhagan S. 2006. Airway epithelium directed gene therapy for cystic fibrosis. Med Chem. 2 499-503. 

Griesenbach U, Geddes DM, Alton EW Gene therapy for cystic fibrosis an example for lung gene therapy. Gene Ther. 2004 ll(suppl 1) S43-S50. 

Prescott WA, Jr, Johnson CE. Antiinflammatory therapies for cystic fibrosis past, present, and future. Pharmacotherapy. 2005 25 555-573. 

Tate S, Elborn S. Progress towards gene therapy for cystic fibrosis. Expert Opin Drug Deliv. 2005 2 269-280. 

Rochat,T.,and Morris, M. A. (2002), Gene therapy for cystic fibrosis by means of aerosol,... 

A number of antibiotics have been used as aerosol therapies. Examples include beta lactam agents, polymycin antimicrobials, neomycin, gentamicin, and tobramycin. Many of the early efforts were reported as case studies, and observations and data regarding safety and efficacy were lacking. Controlled clinical trials were not conducted until the middle of the 1980s. More recent evaluations have focused on the role of inhaled tobramycin used as suppressive therapy for cystic fibrosis patients colonized with Pseudomonas aeruginosa. 

Rubin B. Emerging therapies for cystic fibrosis lung disease. Chest 1999, 115, 1120-1126. 

Kane, R.E. Jennison, K. Wood, C. Black, P.G. Herbst, J.J. Cost savings and economic considerations using home intravenous antibiotic therapy for cystic fibrosis patients. Pediatr. Pulmonol. 1988, 4, 84-89. 

Although aerosols of various forms for treatment of respiratory disorders have been in use since the middle of the twentieth century, the interest in the use of pulmonary route for systemic drug delivery is recent. Interest in this approach has been further stimulated by the demonstration of potential utility of lung as a portal for entry of peptides and the feasibility of gene therapy for cystic fibrosis. It is important to understand the mechanism of macromolecule absorption by the lungs for an effective use of this route. 

Since the 1980s, hundreds of clinical trials of gene therapy for cystic fibrosis, osteogenesis imperfecta, Gaucher s disease, Fanconi s anemia, and several forms of cancer have been attempted. The problems that need to be overcome in developing a successful strategy for gene therapy are ... 

Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer. 

Gene therapy for cystic fibrosis (CF) has focused on correcting electrolyte transport in aiiway epithelia. Success has been limited by the failure of vectors to attach to and enter into airway epithelia, and may require reduecting vectors to targets on the apical mem-... 

Lerondel S, Vecellio None L, Faure L, Sizaret PY, Sene C, Pavirani A, Diot P, Le Pape A et al. Gene therapy for cystic fibrosis with aerosolized adenovirus-CFTR characterization of the aerosol and scintigraphic determination of lung deposition in baboons. J Aerosol Med 2001 14(1) 95-105. 

Boucher, R.C. and Knowles, M.R., Gene therapy for cystic fibrosis using El deleted adenovirus a phase I trial in the nasal cavity. Bethesda, MD, Office of Recombinant DNA Activity, NIH. 

Wilson JM (1995). Gene therapy for cystic fibrosis challenges and future directions. Journal of Clinical Investigation, 96 2547-54. 

K. Herscovitch, N. Dauletbaev, L. C. Lands, Vitamin D as an anti-microbial and antiinflammatory therapy for Cystic Fibrosis, Paediatr. Respir. Rev. 542 (2013) 12-13. 

Flotte TR. Prospects for virus-based gene therapy for cystic fibrosis. J Bioeneig Biomembr 1993 25 37 2. 

Dorin JR, Farley R, Webb S, Smith SN, Farini E, Delaney SJ, Wainwright BJ, Alton EW, Porteous DJ. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther 1996 3 797-801. 

Virella-Lowell I, Mason E, Feikol T, Flotte TR. Anti-inflammatory gene therapy for cystic fibrosis with a RAAV-IL-10 vector. Pediatr Pulmonol 2000 20 (Suppl) 231. 

Kitson C, Alton E. Gene therapy for cystic fibrosis. Exp Opin Invest Drugs 2000 9(7) 1523-1535. 

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