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Journal articles on the topic 'Cystic fibrosis Gene therapy'

Author: Grafiati

Published: 4 June 2021

Last updated: 11 February 2022

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1

Griesenbach, Uta, Jane C. Davies, and Eric Alton. "Cystic fibrosis gene therapy." Current Opinion in Pulmonary Medicine 22, no. 6 (November 2016): 602–9. http://dx.doi.org/10.1097/mcp.0000000000000327.

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2

Colledge, W. H., and M. J. Evans. "Cystic fibrosis gene therapy." British Medical Bulletin 51, no. 1 (January 1995): 82–90. http://dx.doi.org/10.1093/oxfordjournals.bmb.a072955.

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3

Colledge, William H. "Cystic fibrosis gene therapy." Current Opinion in Genetics & Development 4, no. 3 (June 1994): 466–71. http://dx.doi.org/10.1016/0959-437x(94)90037-x.

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4

Prickett, Michelle, and Manu Jain. "Gene therapy in cystic fibrosis." Translational Research 161, no. 4 (April 2013): 255–64. http://dx.doi.org/10.1016/j.trsl.2012.12.001.

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5

Colledge, WH. "Gene therapy for cystic fibrosis." Lancet 349, no. 9060 (April 1997): 1249. http://dx.doi.org/10.1016/s0140-6736(97)26017-4.

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6

Dodge, J. A. "Gene therapy for cystic fibrosis." Nature Medicine 1, no. 3 (March 1995): 182. http://dx.doi.org/10.1038/nm0395-182a.

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7

Armstrong, D. K., S. Cunningham, J. C. Davies, and E. W. F. Alton. "Gene therapy in cystic fibrosis." Archives of Disease in Childhood 99, no. 5 (January 24, 2014): 465–68. http://dx.doi.org/10.1136/archdischild-2012-302158.

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8

Wagner, MD, PhD, John A., and Phyllis Gardner, MD. "TOWARD CYSTIC FIBROSIS GENE THERAPY." Annual Review of Medicine 48, no. 1 (February 1997): 203–16. http://dx.doi.org/10.1146/annurev.med.48.1.203.

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9

Flotte, Terence R., and Beth L. Laube. "Gene Therapy in Cystic Fibrosis." Chest 120, no. 3 (September 2001): 124S—131S. http://dx.doi.org/10.1378/chest.120.3_suppl.124s.

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10

Alton, Eric, Stephen Smith, and Duncan Geddes. "Gene therapy for cystic fibrosis." Lancet 349, no. 9060 (April 1997): 1249–50. http://dx.doi.org/10.1016/s0140-6736(05)62441-5.

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11

Janet E Larson, Y., Susan L. Morrow, and J. Craig Cohen. "Gene therapy for cystic fibrosis." Lancet 349, no. 9060 (April 1997): 1250. http://dx.doi.org/10.1016/s0140-6736(05)62442-7.

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12

Turner, Gillian. "Gene therapy for cystic fibrosis." Lancet 349, no. 9060 (April 1997): 1250–51. http://dx.doi.org/10.1016/s0140-6736(05)62443-9.

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13

Boyd, A. Christopher. "Gene therapy for cystic fibrosis." Expert Opinion on Therapeutic Patents 11, no. 1 (January 2001): 1–15. http://dx.doi.org/10.1517/13543776.11.1.1.

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14

Boyd, A. Christopher. "Gene therapy for cystic fibrosis." Expert Opinion on Therapeutic Patents 11, no. 2 (February 2001): 1–15. http://dx.doi.org/10.1517/13543776.11.2.1.

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15

Conese, Massimo, Sante Di Gioia, and Stefano Castellani. "Gene therapy for cystic fibrosis." Expert Opinion on Therapeutic Patents 18, no. 8 (August 2008): 929–43. http://dx.doi.org/10.1517/13543776.18.8.929.

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16

Davies, Jane C., Duncan M. Geddes, and Eric W. F. W. Alton. "Gene therapy for cystic fibrosis." Journal of Gene Medicine 3, no. 5 (2001): 409–17. http://dx.doi.org/10.1002/jgm.200.

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17

Bellon, G. "Cystic fibrosis (CF) gene therapy." Pediatric Pulmonology 23, S16 (April 1997): 278–79. http://dx.doi.org/10.1002/ppul.19502308144.

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18

Johnson, Larry G. "Gene Therapy for Cystic Fibrosis." Chest 107, no. 2 (February 1995): 77S—83S. http://dx.doi.org/10.1378/chest.107.2_supplement.77s.

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19

Rosenfeld, Melissa A., and Francis S. Collins. "Gene Therapy for Cystic Fibrosis." Chest 109, no. 1 (January 1996): 241–52. http://dx.doi.org/10.1378/chest.109.1.241.

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20

Coutelle, C., N. Caplen, S. Hart, C. Huxley, and R. Williamson. "Gene therapy for cystic fibrosis." Archives of Disease in Childhood 68, no. 4 (April 1, 1993): 437–40. http://dx.doi.org/10.1136/adc.68.4.437.

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21

Kitson, Chris, and Eric Alton. "Gene therapy for cystic fibrosis." Expert Opinion on Investigational Drugs 9, no. 7 (July 2000): 1523–35. http://dx.doi.org/10.1517/13543784.9.7.1523.

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22

Davies, J. C., and E. W. F. W. Alton. "Gene Therapy for Cystic Fibrosis." Proceedings of the American Thoracic Society 7, no. 6 (October 28, 2010): 408–14. http://dx.doi.org/10.1513/pats.201004-029aw.

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23

Luder, Elisabeth. "Gene Therapy for Cystic Fibrosis." Topics in Clinical Nutrition 14, no. 4 (September 1999): 22–30. http://dx.doi.org/10.1097/00008486-199909000-00004.

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24

Mueller, Christian, and Terence R. Flotte. "Gene Therapy for Cystic Fibrosis." Clinical Reviews in Allergy & Immunology 35, no. 3 (July 4, 2008): 164–78. http://dx.doi.org/10.1007/s12016-008-8080-3.

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25

Alton, E. W. F. W. "Gene therapy for cystic fibrosis." Journal of Inherited Metabolic Disease 18, no. 4 (1995): 501–7. http://dx.doi.org/10.1007/bf00710061.

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26

Geddes, D. "Gene therapy for cystic fibrosis." Netherlands Journal of Medicine 46, no. 6 (June 1995): 306–12. http://dx.doi.org/10.1016/0300-2977(95)00024-h.

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27

Coutelle, Charles. "Gene therapy approaches for cystic fibrosis." Biologicals 23, no. 1 (March 1995): 21–25. http://dx.doi.org/10.1016/1045-1056(95)90006-3.

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28

Brown, Matt. "Gene therapy trials for cystic fibrosis." Drug Discovery Today 7, no. 15 (August 2002): 788–89. http://dx.doi.org/10.1016/s1359-6446(02)02393-0.

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29

Higgins, Christopher F., Stephen C. Hyde, and Deborah R. Gill. "Towards Gene Therapy for Cystic Fibrosis." Biochemical Society Transactions 27, no. 5 (October 1, 1999): A137. http://dx.doi.org/10.1042/bst027a137a.

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30

ALTON, E. W. F. W. "Towards Gene Therapy For Cystic Fibrosis*." Journal of Pharmacy and Pharmacology 47, no. 5 (May 1995): 351–54. http://dx.doi.org/10.1111/j.2042-7158.1995.tb05809.x.

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31

Ezzell, C. "Gene Therapy for Cystic Fibrosis Patients." Science News 142, no. 24 (December 12, 1992): 405. http://dx.doi.org/10.2307/4017870.

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32

O'Neal, WK, and AL Beaudet. "Somatic gene therapy for cystic fibrosis." Human Molecular Genetics 3, suppl_1 (September 1, 1994): 1497–502. http://dx.doi.org/10.1093/hmg/3.suppl_1.1497.

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33

Griesenbach, Uta, Duncan M. Geddes, and Eric W. F. W. Alton. "Advances in cystic fibrosis gene therapy." Current Opinion in Pulmonary Medicine 10, no. 6 (November 2004): 542–46. http://dx.doi.org/10.1097/01.mcp.0000142102.91202.04.

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34

Griesenbach, U., and E. W. F. W. Alton. "Moving forward: cystic fibrosis gene therapy." Human Molecular Genetics 22, R1 (August 4, 2013): R52—R58. http://dx.doi.org/10.1093/hmg/ddt372.

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35

Parsons, DW. "Airway gene therapy and cystic fibrosis." Journal of Paediatrics and Child Health 41, no. 3 (March 2005): 94–96. http://dx.doi.org/10.1111/j.1440-1754.2005.00556.x.

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36

Wilson, James. "Cystic Fibrosis: Strategies for Gene Therapy." Seminars in Respiratory and Critical Care Medicine 15, no. 05 (September 1994): 439–45. http://dx.doi.org/10.1055/s-2007-1006389.

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37

Drittanti, L., MV Masciovecchio, J. Gabbarini, and M. Vega. "Cystic fibrosis: gene therapy or preventive gene transfer?" Gene Therapy 4, no. 10 (October 1997): 1001–3. http://dx.doi.org/10.1038/sj.gt.3300526.

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38

Kutsev, S. I., V. L. Izhevskaya, and E. I. Kondratyeva. "Targeted therapy for cystic fibrosis." PULMONOLOGIYA 31, no. 2 (April 11, 2021): 226–36. http://dx.doi.org/10.18093/0869-0189-2021-31-2-226-236.

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Abstract:

The basic therapy of cystic fibrosis is currently aimed at slowing down the pathological processes associated with a decrease in the CFTR protein activity (cystic fibrosis transmembrane conductance regulator) in the gastrointestinal tract and the respiratory system. The pancreatic insufficiency is well compensated by replacement therapy with microsphere enzyme preparations and a high-calorie diet rich in proteins and fat. Chronic treatment of cystic fibrosis-related lung disease aims to improve the clearance of the bronchial tree, suppressing chronic bacterial infection and local chronic inflammation. However, no therapy was available to correct the defect in the gene or its product until 2012.The aim was to analyze literature on CFTR modulators, including their efficacy and safety, and assess the potential for developing new modulators to treat cystic fibrosis.Materials. The review included literature data (45 publications) on the use of CFTR modulators and international websites’ data.Results. Since the discovery of the CFTR gene in 1989, more than 2000 mutations or variants of the CFTR gene (hereinafter referred to as genetic variants) have been described. They interfere with the synthesis of the CFTR protein, its transport to the apical membrane of the cell, or disrupt its function as a channel for chloride anions. Although it is currently not possible to completely replace the mutant gene with a normal copy, small molecules have been identified that can modify the mutant CFTR protein and amend its function. The potential therapeutic measures are determined by class of the mutation. In clinical practice, pharmacological modeling of ion transport is currently possible only with the use of CFTR modulators: correctors and potentiators. The review defines these groups of drugs and describes 4 licensed CFTR modulators, including molecules of ivacaftor, lumacaftor, tezacaftor, elexacaftor. The data on the promising emerging next generation modulators and the prospects for the personalized selection of drugs using the assays on intestinal organoids are presented.

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39

LEE, Tim W. R., David A. MATTHEWS, and G. Eric BLAIR. "Novel molecular approaches to cystic fibrosis gene therapy." Biochemical Journal 387, no. 1 (March 22, 2005): 1–15. http://dx.doi.org/10.1042/bj20041923.

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Abstract:

Gene therapy holds promise for the treatment of a range of inherited diseases, such as cystic fibrosis. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of cystic fibrosis pulmonary disease has proved elusive. There are many reasons for this lack of progress, both macroscopically in terms of airway defence mechanisms and at the molecular level with regard to effective cDNA delivery. This review of approaches to cystic fibrosis gene therapy covers these areas in detail and highlights recent progress in the field. For gene therapy to be effective in patients with cystic fibrosis, the cDNA encoding the cystic fibrosis transmembrane conductance regulator protein must be delivered effectively to the nucleus of the epithelial cells lining the bronchial tree within the lungs. Expression of the transgene must be maintained at adequate levels for the lifetime of the patient, either by repeat dosage of the vector or by targeting airway stem cells. Clinical trials of gene therapy for cystic fibrosis have demonstrated proof of principle, but gene expression has been limited to 30 days at best. Results suggest that viral vectors such as adenovirus and adeno-associated virus are unsuited to repeat dosing, as the immune response reduces the effectiveness of each subsequent dose. Nonviral approaches, such as cationic liposomes, appear more suited to repeat dosing, but have been less effective. Current work regarding non-viral gene delivery is now focused on understanding the mechanisms involved in cell entry, endosomal escape and nuclear import of the transgene. There is now increasing evidence to suggest that additional ligands that facilitate endosomal escape or contain a nuclear localization signal may enhance liposome-mediated gene delivery. Much progress in this area has been informed by advances in our understanding of the mechanisms by which viruses deliver their genomes to the nuclei of host cells.

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40

Castellani, Stefano, and Massimo Conese. "Lentiviral Vectors and Cystic Fibrosis Gene Therapy." Viruses 2, no. 2 (January 29, 2010): 395–412. http://dx.doi.org/10.3390/v2020395.

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41

&NA;. "Is gene therapy for cystic fibrosis realistic?" Inpharma Weekly &NA;, no. 1137 (May 1998): 6. http://dx.doi.org/10.2165/00128413-199811370-00008.

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42

DAGANI, RON. "Big step toward cystic fibrosis gene therapy." Chemical & Engineering News 70, no. 3 (January 20, 1992): 6. http://dx.doi.org/10.1021/cen-v070n003.p006.

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43

Davies, Jane C. "Gene and cell therapy for cystic fibrosis." Paediatric Respiratory Reviews 7 (January 2006): S163—S165. http://dx.doi.org/10.1016/j.prrv.2006.04.214.

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44

Tizzano, Eduardo F., and Manuel Buchwald. "Cystic fibrosis: Beyond the gene to therapy." Journal of Pediatrics 120, no. 3 (March 1992): 337–49. http://dx.doi.org/10.1016/s0022-3476(05)80895-9.

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45

Jaffe, A., A. Bush, D. M. Geddes, and E. W. F. W. Alton. "Prospects for gene therapy in cystic fibrosis." Archives of Disease in Childhood 80, no. 3 (March 1, 1999): 286–89. http://dx.doi.org/10.1136/adc.80.3.286.

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46

Alton, Eric WFW, and Duncan Geddes. "Gene Therapy: The Case for Cystic Fibrosis." Journal of the Royal Society of Medicine 90, no. 31_suppl (December 1997): 43–46. http://dx.doi.org/10.1177/014107689709031s09.

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47

Caplen, Natasha J. "Cystic fibrosis gene therapy trials and tribulations." Trends in Molecular Medicine 7, no. 11 (November 2001): 488. http://dx.doi.org/10.1016/s1471-4914(01)02168-2.

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48

Davies, Jane C., Duncan M. Geddes, and Eric W. F. W. Alton. "Prospects for gene therapy for cystic fibrosis." Molecular Medicine Today 4, no. 7 (July 1998): 292–99. http://dx.doi.org/10.1016/s1357-4310(98)01265-9.

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49

Fox, Jeffrey L. "NIHRAC okays gene therapy for cystic fibrosis." Nature Biotechnology 11, no. 1 (January 1993): 29. http://dx.doi.org/10.1038/nbt0193-28b.

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50

Griesenbach, U., S. Ferrari, D. M. Geddes, and E. W. F. W. Alton. "Gene Therapy Progress and Prospects: Cystic fibrosis." Gene Therapy 9, no. 20 (October 2002): 1344–50. http://dx.doi.org/10.1038/sj.gt.3301791.

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