Gene therapy cystic fibrosis

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Ferrari, S., Pettenazzo, A., Garbati, N., Zacchello, F., Behr, J.P., Scarpa, M. (1999). Polyethylenimine shows properties of interest for cystic fibrosis gene therapy. Bio-... 

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Schwiebert LM. Cystic fibrosis, gene therapy, and lung inflammation for better or worse Am J Pbysiol Lung Cell Mol Pbysiol 2004 286 L715-L716. 

Lerondel S, Le Pape A, Sene C, et al. (2001). Radioisotopic imaging allows optimization of adenovirus lung deposition for cystic fibrosis gene therapy. Human Gene Ther. 12 1-11. 

Gill, D. R., Southern, K. W., Mofford, K. A. et al. 1997. A placebo-controlled study of hposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Therapy 4 199-209. 

J.Lyon P. Corner Altered Fates Gene Therapy and the Retooling of Human Life W.W. Norton, 1995 K.W. Culver Gene Therapy A Handbook for Physicians Mary Ann Liebert, Inc., Publishers, 1994 R.C. Boucher Current Status of Cystic Fibrosis Gene Therapy Trends Genet. 12 (1996) 81-84]... 

Wagner JA Gardner P. Toward cystic fibrosis gene therapy. Armu Rev Med 1997 48 203-216. 

Griesenbach U, Chonn A, Cassady R, Hannam V, Ackerley C, Post M, Tanswell AK, OlekK, O Brodovich H, Tsui L-C. Comparison between intratracheal and intravenous administration of liposome-DNA complexes for cystic fibrosis gene therapy. Gene Ther 1998 5 181-188. 

Koehler DR, Hannam V, Belcastro R, Steer B, Wen Y, Post M, Downey G, Transwell AK, Hu J. Targeting transgene expression for cystic fibrosis gene therapy. Mol Ther2001 4(l) 58-65. 

Cystic fibrosis Gene therapy Cystic fibrosis gene... 

Griesenbach, U., Geddes, D. M., and Alton, E. W. (2006), Gene therapy progress and prospects Cystic fibrosis, Gene Ther., 13,1061-1067. 

First clinical human gene therapy trials with polyplexes were performed using cancer vaccines based on autologous patient tumor cells. These were modified ex vivo with interleukin-2 pDNA. To obtain high level transfection rates of patient s primary tumor cells, Tf-PLL/pDNA polyplexes linked with inactivated endosomolytic adenovirus particles were applied [221]. Polymer-based in vivo human gene transfer studies were performed with PEGylated PLL polyplexes, delivering CFTR pDNA to the airway epithelium of cystic fibrosis patients [222],... 

Although therapeutic approaches based upon gene therapy (Chapter 14) may well one day cure cystic fibrosis, current therapeutic intervention focuses upon alleviating cystic fibrosis symptoms, particularly those relating to respiratory function. Improved patient care has increased life expectancy of cystic fibrosis patients to well into their 30s. The major elements of cystic fibrosis management include ... 

To date, cellular and gene therapy products submitted to FDA have included clinical studies indicated for bone marrow marking, cancer, cystic fibrosis, AIDS, and inborn errors of metabolism and infectious diseases. Of the current active INDs approximately 78% have been sponsored by individual investigators or academic institutions and 22% have also been industry sponsored. In addition to the variety of clinical indications the cell types have also been varied. Examples include tumor infiltrating lymphocytes (TIL) and lymphocyte activated killer (LAK) cells, selected cells from bone marrow and peripheral blood lymphocytes, for example, stem cells, myoblasts, tumor cells and encapsulated cells (e.g., islet cells and adrenal chromaffin cells). 

Currently, there is stUl a gap for the potential of gene therapy to be fulfilled. Gene therapy clinical trials have been conducted for diseases such as severe combined immunodeficiency disease (SCID, bubble baby syndrome), sickle cell anemia, cystic fibrosis, familial hypercholesterolemia, and Gaucher disease. 

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