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Статті в журналах з теми "Cystic fibrosis; gene therapy; lentivirus"

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Автор: Grafiati

Опубліковано: 10 грудня 2022

Оновлено: 28 січня 2023

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1

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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2

Goldman, Mitchell J., Po-Shun Lee, Joo-Sung Yang, and James M. Wilson. "Lentiviral Vectors for Gene Therapy of Cystic Fibrosis." Human Gene Therapy 8, no. 18 (December 10, 1997): 2261–68. http://dx.doi.org/10.1089/hum.1997.8.18-2261.

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3

Mitomo, Katsuyuki, Uta Griesenbach, Makoto Inoue, Lucinda Somerton, Cuixiang Meng, Eiji Akiba, Toshiaki Tabata, et al. "Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes." Molecular Therapy 18, no. 6 (June 2010): 1173–82. http://dx.doi.org/10.1038/mt.2010.13.

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4

Griesenbach, Uta, and Eric WFW Alton. "Cystic Fibrosis Gene Therapy – Not Low-hanging Fruit." European Respiratory & Pulmonary Diseases 02, no. 02 (2016): 48. http://dx.doi.org/10.17925/erpd.2016.02.02.48.

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The last 25 years have shown that it has been comparatively slow and difficult to develop cystic fibrosis (CF) gene therapy; the lung is a complex target organ. However, research has steadily progressed and recently it was shown that non-viral gene therapy can stabilise CF lung disease. These data, in addition to the development of potent lentiviral vectors, have renewed interest in CF gene therapy within academia and industry.

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5

Copreni, E., M. Penzo, S. Carrabino, and M. Conese. "Lentivirus-mediated gene transfer to the respiratory epithelium: a promising approach to gene therapy of cystic fibrosis." Gene Therapy 11, S1 (September 29, 2004): S67—S75. http://dx.doi.org/10.1038/sj.gt.3302372.

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6

Sinn, Patrick L., Melissa A. Hickey, Patrick D. Staber, Douglas E. Dylla, Scott A. Jeffers, Beverly L. Davidson, David A. Sanders, and Paul B. McCray. "Lentivirus Vectors Pseudotyped with Filoviral Envelope Glycoproteins Transduce Airway Epithelia from the Apical Surface Independently of Folate Receptor Alpha." Journal of Virology 77, no. 10 (May 15, 2003): 5902–10. http://dx.doi.org/10.1128/jvi.77.10.5902-5910.2003.

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ABSTRACT The practical application of gene therapy as a treatment for cystic fibrosis is limited by poor gene transfer efficiency with vectors applied to the apical surface of airway epithelia. Recently, folate receptor alpha (FRα), a glycosylphosphatidylinositol-linked surface protein, was reported to be a cellular receptor for the filoviruses. We found that polarized human airway epithelia expressed abundant FRα on their apical surface. In an attempt to target these apical receptors, we pseudotyped feline immunodeficiency virus (FIV)-based vectors by using envelope glycoproteins (GPs) from the filoviruses Marburg virus and Ebola virus. Importantly, primary cultures of well-differentiated human airway epithelia were transduced when filovirus GP-pseudotyped FIV was applied to the apical surface. Furthermore, by deleting a heavily O-glycosylated extracellular domain of the Ebola GP, we improved the titer of concentrated vector severalfold. To investigate the folate receptor dependence of gene transfer with the filovirus pseudotypes, we compared gene transfer efficiency in immortalized airway epithelium cell lines and primary cultures. By utilizing phosphatidylinositol-specific phospholipase C (PI-PLC) treatment and FRα-blocking antibodies, we demonstrated FRα-dependent and -independent entry by filovirus glycoprotein-pseudotyped FIV-based vectors in airway epithelia. Of particular interest, entry independent of FRα was observed in primary cultures of human airway epithelia. Understanding viral vector binding and entry pathways is fundamental for developing cystic fibrosis gene therapy applications.

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7

Bradbury, Jane. "Detergent–lentiviral combination gives gene therapy hope for cystic fibrosis." Lancet 360, no. 9342 (October 2002): 1306. http://dx.doi.org/10.1016/s0140-6736(02)11359-6.

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8

Marquez Loza, Laura, Eric Yuen, and Paul McCray. "Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease." Genes 10, no. 3 (March 14, 2019): 218. http://dx.doi.org/10.3390/genes10030218.

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Despite the continued development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs for the treatment of cystic fibrosis (CF), the need for mutation agnostic treatments remains. In a sub-group of CF individuals with mutations that may not respond to modulators, such as those with nonsense mutations, CFTR gene transfer to airway epithelia offers the potential for an effective treatment. Lentiviral vectors are well-suited for this purpose because they transduce nondividing cells, and provide long-term transgene expression. Studies in primary cultures of human CF airway epithelia and CF animal models demonstrate the long-term correction of CF phenotypes and low immunogenicity using lentiviral vectors. Further development of CF gene therapy requires the investigation of optimal CFTR expression in the airways. Lentiviral vectors with improved safety features have minimized insertional mutagenesis safety concerns raised in early clinical trials for severe combined immunodeficiency using γ-retroviral vectors. Recent clinical trials using improved lentiviral vectors support the feasibility and safety of lentiviral gene therapy for monogenetic diseases. While work remains to be done before CF gene therapy reaches the bedside, recent advances in lentiviral vector development reviewed here are encouraging and suggest it could be tested in clinical studies in the near future.

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9

Gui, Liqiong, Hong Qian, Kevin A. Rocco, Loreta Grecu, and Laura E. Niklason. "Efficient intratracheal delivery of airway epithelial cells in mice and pigs." American Journal of Physiology-Lung Cellular and Molecular Physiology 308, no. 2 (January 15, 2015): L221—L228. http://dx.doi.org/10.1152/ajplung.00147.2014.

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Cellular therapy via direct intratracheal delivery has gained interest as a novel therapeutic strategy for treating various pulmonary diseases including cystic fibrosis lung disease. However, concerns such as insufficient cell engraftment in lungs and lack of large animal model data remain to be resolved. This study aimed to establish a simple method for evaluating cell retention in lungs and to develop reproducible approaches for efficient cell delivery into mouse and pig lungs. Human lung epithelial cells including normal human bronchial/tracheal epithelial (NHBE) cells and human lung epithelial cell line A549 were infected with pSicoR-green fluorescent protein (GFP) lentivirus. GFP-labeled NHBE cells were delivered via a modified intratracheal cell instillation method into the lungs of C57BL/6J mice. Two days following cell delivery, GFP ELISA-based assay revealed a substantial cell-retention efficiency (10.48 ± 2.86%, n = 7) in mouse lungs preinjured with 2% polidocanol. When GFP-labeled A549 cells were transplanted into Yorkshire pig lungs with a tracheal intubation fiberscope, a robust initial cell attachment (22.32% efficiency) was observed at 24 h. In addition, a lentiviral vector was developed to induce the overexpression and apical localization of cystic fibrosis transmembrane conductance regulator (CFTR)-GFP fusion proteins in NHBE cells as a means of ex vivo CFTR gene transfer in nonprogenitor (relatively differentiated) lung epithelial cells. These results have demonstrated the convenience and efficiency of direct delivery of exogenous epithelial cells to lungs in mouse and pig models and provided important background for future preclinical evaluation of intratracheal cell transplantation to treat lung diseases.

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10

McCarron, Alexandra, Chantelle McIntyre, Martin Donnelley, Patricia Cmielewski, and David Parsons. "711. Development of a Clinically-Acceptable Lentiviral Vector for Cystic Fibrosis Airway Gene Therapy." Molecular Therapy 24 (May 2016): S280—S281. http://dx.doi.org/10.1016/s1525-0016(16)33519-5.

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11

Marquez Loza, Laura I., Ashley L. Cooney, Qian Dong, Christoph O. Randak, Stefano Rivella, Patrick L. Sinn, and Paul B. McCray. "Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy." Molecular Therapy - Methods & Clinical Development 21 (June 2021): 94–106. http://dx.doi.org/10.1016/j.omtm.2021.02.020.

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12

Cmielewski, Patricia, Nigel Farrow, Sharnna Devereux, David Parsons, and Martin Donnelley. "Gene therapy for Cystic Fibrosis: Improved delivery techniques and conditioning with lysophosphatidylcholine enhance lentiviral gene transfer in mouse lung airways." Experimental Lung Research 43, no. 9-10 (November 26, 2017): 426–33. http://dx.doi.org/10.1080/01902148.2017.1395931.

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13

Bañuls, Lucía, Daniel Pellicer, Silvia Castillo, María Mercedes Navarro-García, María Magallón, Cruz González, and Francisco Dasí. "Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?" Journal of Clinical Medicine 9, no. 8 (August 8, 2020): 2577. http://dx.doi.org/10.3390/jcm9082577.

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Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and currently without curative treatment. After five decades of progress, many different vectors and gene editing tools for genetic engineering are now available. However, we are still a long way from achieving a safe and efficient approach to gene therapy application in clinical practice. Here, we review three of the most common rare respiratory conditions—cystic fibrosis (CF), alpha-1 antitrypsin deficiency (AATD), and primary ciliary dyskinesia (PCD)—alongside attempts to develop genetic treatment for these diseases. Since the 1990s, gene augmentation therapy has been applied in multiple clinical trials targeting CF and AATD, especially using adeno-associated viral vectors, resulting in a good safety profile but with low efficacy in protein expression. Other strategies, such as non-viral vectors and more recently gene editing tools, have also been used to address these diseases in pre-clinical studies. The first gene therapy approach in PCD was in 2009 when a lentiviral transduction was performed to restore gene expression in vitro; since then, transcription activator-like effector nucleases (TALEN) technology has also been applied in primary cell culture. Gene therapy is an encouraging alternative treatment for these respiratory diseases; however, more research is needed to ensure treatment safety and efficacy.

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14

Castellani, Stefano, Sante Di Gioia, Teresa Trotta, Angela Bruna Maffione, and Massimo Conese. "Impact of Lentiviral Vector-Mediated Transduction on the Tightness of a Polarized Model of Airway Epithelium and Effect of Cationic Polymer Polyethylenimine." Journal of Biomedicine and Biotechnology 2010 (2010): 1–11. http://dx.doi.org/10.1155/2010/103976.

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Lentiviral (LV) vectors are promising agents for efficient and long-lasting gene transfer into the lung and for gene therapy of genetically determined pulmonary diseases, such as cystic fibrosis, however, they have not been evaluated for cytotoxicity and impact on the tightness of the airway epithelium. In this study, we evaluated the transduction efficiency of a last-generation LV vector bearing Green Fluorescent Protein (GFP) gene as well as cytotoxicity and tight junction (TJ) integrity in a polarized model of airway epithelial cells. High multiplicities of infection (MOI) showed to be cytotoxic, as assessed by increase in propidium iodide staining and decrease in cell viability, and harmful for the epithelial tightness, as demonstrated by the decrease of transepithelial resistance (TER) and delocalization of occludin from the TJs. To increase LV efficiency at low LV:cell ratio, we employed noncovalent association with the polycation branched 25ߙkDa polyethylenimine (PEI). Transduction of cells with PEI/LV particles resulted in 2.5–3.6-fold increase of percentage of GFP-positive cells only at the highest PEI:LV ratios (1×107 PEI molecules/transducing units with 50 MOI LV) as compared to plain LV. At this dose PEI/LV transduction resulted in6.5±2.4% of propidium iodide-positive cells. On the other hand, PEI/LV particles did not determine any alteration of TER and occludin localization. We conclude that PEI may be useful for improving the efficiency of gene transfer mediated by LV vectors in airway epithelial cells, in the absence of high acute cytotoxicity and alteration in epithelial tightness.

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15

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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16

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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17

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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18

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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19

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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20

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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21

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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22

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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23

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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24

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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25

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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26

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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27

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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28

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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29

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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30

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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31

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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32

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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33

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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34

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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35

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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36

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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37

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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38

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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39

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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40

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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41

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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42

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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43

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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44

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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45

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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46

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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47

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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48

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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49

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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50

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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