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Relevant bibliographies by topics / Cystic fibrosis Gene therapy

Academic literature on the topic 'Cystic fibrosis Gene therapy'

Author: Grafiati

Published: 4 June 2021

Last updated: 11 February 2022

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Contents

Journal articles
Dissertations / Theses
Books
Book chapters
Conference papers

Journal articles on the topic "Cystic fibrosis Gene therapy":

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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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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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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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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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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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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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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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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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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Dissertations / Theses on the topic "Cystic fibrosis Gene therapy":

1

Davies, Gwyneth. "Outcome measures for cystic fibrosis gene therapy clinical trials." Thesis, Imperial College London, 2013. http://hdl.handle.net/10044/1/28414.

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Abstract:

Background: Cystic fibrosis (CF) is a life-shortening, chronic respiratory disease caused by mutations in the CFTR gene. Novel therapeutic agents such as gene therapy aim to correct CFTR and to demonstrate evidence of molecular, functional and (ultimately) clinical efficacy. It was hypothesised that currently used methods to detect these changes may be optimised to enhance sensitivity and allow quantification, and facilitate an understanding of geographical effects within the airway. Methods: Outcome measures were investigated within two UK CF Gene Therapy Consortium studies; a longitudinal observational study ('Run-In'), and a single dose gene therapy study ('Pilot') which investigated safety and functional efficacy. Measurement of airway function with spirometry and lung clearance index in the Run-In study allowed investigation of variability and change over time and comparison with other outcomes. Development of methodology and data analysis from measurements of potential difference (PD) in the nose and lung in the Pilot study allowed investigation of these as measures of functional efficacy. Results: In the Run-In study, the choice of external reference source was crucial for interpretation of spirometry outcomes. Airway physiology outcomes correlated with structural changes on chest CT however were limited in their ability to detect site of airway abnormality. There was some evidence that disease severity was associated with intra-subject variability and affected rate of change over time. In the Pilot study, airway PD was shown to change post gene therapy within individuals but the responses were not universal and depended on the definitions used. A novel method of nasal PD quantification did not improve an ability to quantify change. Conclusions: There is no single universal outcome measure in CF, but it is important to take account of the patient population in terms of disease severity. Whilst it would be inappropriate to relate PD outcomes with clinical outcomes in the Pilot study; this will be an important relationship to understand in the future in order to allow rational design of CF gene therapy clinical trials.

2

Rose, Andrew C. "Studies on the expression of the murine CFTR gene : implications for gene therapy." Thesis, University of Oxford, 2001. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.365354.

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3

Dragomir, Anca. "Approaches to Pharmacological Treatment and Gene Therapy of Cystic Fibrosis." Doctoral thesis, Uppsala : Acta Universitatis Upsaliensis : Univ.-bibl. [distributör], 2004. http://urn.kb.se/resolve?urn=urn:nbn:se:uu:diva-3845.

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McKay, Tristan Rowntree. "Investigations toward gene therapy for hepatobiliary disease in cystic fibrosis." Thesis, Imperial College London, 2000. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.392184.

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Cooney, Ashley L. "Integrating viral vectors as a gene therapy approach for cystic fibrosis." Diss., University of Iowa, 2018. https://ir.uiowa.edu/etd/6083.

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Abstract:

Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasian populations. CF affects multiple organ systems including pancreas, liver, intestines, sweat glands, and male reproductive organs, however the leading cause of morbidity and mortality in CF patients is chronic lung disease. CF is caused by a mutant cystic fibrosis transmembrane conductance regulator (CFTR) gene which leads to chloride (Cl-) and bicarbonate (HCO3-) anion dysregulation at the airway surface. Without adequate anion exchange, thick, viscous mucus accumulates at the airway surface allowing bacterial colonization to occur. Complementing CFTR in the appropriate airway cells restores the anion channel activity in CFTR-deficient cells. The ultimate goal for CF gene therapy is to design an integrating vector that would lead to persistent and efficient expression of CFTR in the airways. Performing gene therapy experiments is dependent upon a relevant animal model. The CF pig is a large animal model similar in size, anatomy, and physiology to humans. Importantly, the CF pig recapitulates human lung disease. From the CF pig, we have learned much about CF lung disease and have developed relevant assays to measure anion channel correction. We have learned that loss of CFTR leads to a decreased airway surface ASL pH, bacterial killing ability, and increased mucus viscosity. Standardized assays have been developed to evaluate the change in current by Ussing chambers, ASL pH, bacterial killing in vivo and ASL pH and viscosity on primary airway cultures in vitro. Ultimately, these metrics allow us to make conclusions about the efficiency of CFTR restoration. Viral vectors are promising candidates for CF gene therapy. Viral vectors such as adenovirus (Ad), adeno-associated virus (AAV), and pseudotyped lentiviral vectors such as feline immunodeficiency virus (FIV) or human immunodeficiency virus (HIV) can efficiently transduce airway cells and express CFTR. Ad and AAV have both been tested in CF clinical trials, but CFTR expression was transient, if detected at all. Understanding vector biology and overcoming barriers in the lung have allowed us to improve vector delivery to the airways. However, the next major hurdle was achieving persistent expression. Ad and AAV are both transiently expressing vectors, and vector readministration is implausible due to the presence of neutralizing antibodies that develop against the vector. Creating a hybrid nonviral/viral vector in which the integrating nonviral piggyBac transposon system is delivered by an Ad or AAV vector has allowed us to achieve persistent expression in mice. In a third integrating vector system, lentiviral vectors have historically been challenging to work with due to low titer levels. However, improvement in vector purification methods have allowed us to validate a lentiviral vector as a viable gene therapy option. In total, we have validated three integrating vector systems by restoring CFTR to CF pigs to correct the phenotypic defect.

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Scott, Emily Siân. "Improving the efficiency of liposome-mediated gene transfer for cystic fibrosis gene therapy." Thesis, University of Cambridge, 1999. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.624332.

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Jannetta, Evelyn Elena. "Qualitative study of cystic fibrosis (CF) patients' expectations of gene therapy." Thesis, University of Edinburgh, 2009. http://hdl.handle.net/1842/8745.

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Abstract:

Introduction: Gene therapy is currently being developed for people with cystic fibrosis (CF), a life-threatening condition for which there is no cure. The UK CF Gene Therapy Consortium are preparing for a multi-dose gene therapy trial of sufficient duration that clinical benefit may be seen. Aims: The current study aimed to explore the expectations and beliefs of cystic fibrosis (CF) patients involved in the preparatory phase of the gene therapy trial (the Run-in study), from which participants will be selected for the multi-dose actual gene therapy trial. Method: Twelve participants (six with mild and six with moderate CF) were interviewed using a semi-structured interview. Interviews were recorded, transcribed verbatim and then analysed using a Constructivist Grounded Theory approach. Results: Since entering the Run-in study, half of the patients had increased their expectations of gene therapy being an effective future treatment. Most of the participants hoped to derive clinical benefit from the trial itself though half were unsure of what to expect. Whilst half of the participants expressed the hope of a future cure for CF, the remainder saw gene therapy only in terms of an improved treatment. Participants used several strategies to manage their expectations including not thinking too far ahead and trusting the research team. Discussion: The findings indicate that participants in the Run-in trial are generally eager to be involved in the gene therapy trial and have developed a strong sense of trust in the research team conducting the trials. The levels of optimism expressed for personal benefit from trial were higher than those from earlier studies. Some of the positive expectations were unlikely to be met by the gene therapy trial and participants risk disappointment. However other patients participated with apparently realistic expectations and it seems likely that some patients would have participated even without prospect for personal benefit. Possible areas of psychological support are discussed e.g. a standard clinical interview for all those not accepted for the gene therapy trial; screening for anxiety pre-, during and post-participation.

8

Jaffe, Adam. "Assessment and feasibility of gene therapy for cystic fibrosis in children." Thesis, Imperial College London, 2000. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.589769.

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Middleton, Peter Gordon. "Cystic fibrosis ion transport and the effect of CFTR gene transfer." Thesis, Imperial College London, 1995. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.307399.

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Kwilas, Anna R. "Respiratory Syncytial Virus Based Vectors for the Treatment of Cystic Fibrosis." The Ohio State University, 2010. http://rave.ohiolink.edu/etdc/view?acc_num=osu1284384649.

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Books on the topic "Cystic fibrosis Gene therapy":

1

Love, Cynthia B. Genetic testing for cystic fibrosis: January 1989 through February 1997 : 1224 citations. Bethesda, Md. (8600 Rockville Pike, Bethesda 20894): U.S. Dept. of Health and Human Services, Public Health Service, National Institutes of Health, National Library of Medicine, Reference Section, 1997.

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United States. Congress. Senate. Committee on Small Business. Research on childhood diseases by entrepreneurs: Hearing before the Committee on Small Business, United States Senate, One Hundred Third Congress, second session ... Thursday, May 26, 1994. Washington: U.S. G.P.O., 1995.

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United States. Congress. Senate. Committee on Small Business. Research on childhood diseases by entrepreneurs: Hearing before the Committee on Small Business, United States Senate, One Hundred Third Congress, second session ... Thursday, May 26, 1994. Washington: U.S. G.P.O., 1995.

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4

Skypala, Isabel. Eating well with cystic fibrosis. Bromley: Cystic Fibrosis Trust, 1994.

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5

Tsui, Lap-Chee, Giovanni Romeo, Rainer Greger, and Sergio Gorini, eds. The Identification of the CF (Cystic Fibrosis) Gene. Boston, MA: Springer US, 1991. http://dx.doi.org/10.1007/978-1-4684-5934-0.

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6

Ferguson, Kate. The physical treatment of cystic fibrosis. Bromley: Cystic Fibrosis Trust, 1995.

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7

Mofford, K. A. Preclinical studies in DNA/liposome gene transfer for cystic fibrosis. Oxford: Oxford Brookes University, 1996.

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8

MacDonald, Anita. Eating well with cystic fibrosis: A guide for children and parents. Bromley: Cystic Fibrosis Trust, 1996.

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9

Genetic Analysis Workshop (6th 1988 Long Beach, Miss.). Multipoint mapping and linkage based upon affected pedigree members: Genetic Analysis Workshop 6, proceedings of a workshop held at Gulf Park, Long Beach, Mississippi, October 10-12, 1988. Edited by Elston Robert C. 1932-. New York: Liss, 1988.

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10

Duguay, François. Use of the Xenopus oocyte as a potential expression system for the cystic fibrosis gene. Ottawa: National Library of Canada = Bibliothèque nationale du Canada, 1991.

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Book chapters on the topic "Cystic fibrosis Gene therapy":

1

Porteous, D. J., and J. A. Innes. "Gene Therapy for Cystic Fibrosis." In Gene Therapy, 137–49. Basel: Birkhäuser Basel, 1999. http://dx.doi.org/10.1007/978-3-0348-7011-5_10.

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Boyd, A. Christopher. "Gene and Stem Cell Therapy." In Cystic Fibrosis in the 21st Century, 221–29. Basel: KARGER, 2005. http://dx.doi.org/10.1159/000088601.

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3

Sumner-Jones, Stephanie G., Deborah R. Gill, and Stephen C. Hyde. "Gene therapy for cystic fibrosis lung disease." In Gene Therapy for Autoimmune and Inflammatory Diseases, 47–64. Basel: Springer Basel, 2010. http://dx.doi.org/10.1007/978-3-0346-0165-8_4.

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4

Trapnell, Bruce C. "Gene Therapy for Cystic Fibrosis Lung Disease." In The Pediatric Lung, 229–58. Basel: Birkhäuser Basel, 1997. http://dx.doi.org/10.1007/978-3-0348-8960-5_10.

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5

Alton, Eric W. F. W. "Gene Delivery and Therapy: The Case for Cystic Fibrosis." In Targeting of Drugs 5, 15–19. Boston, MA: Springer US, 1996. http://dx.doi.org/10.1007/978-1-4615-6405-8_2.

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6

Carter, B. J., and T. R. Flotte. "Development of Adeno-associated Virus Vectors for Gene Therapy of Cystic Fibrosis." In Adeno-Associated Virus (AAV) Vectors in Gene Therapy, 119–44. Berlin, Heidelberg: Springer Berlin Heidelberg, 1996. http://dx.doi.org/10.1007/978-3-642-80207-2_8.

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Bruzzese, Eugenia, Vittoria Buccigrossi, Giusy Ranucci, and Alfredo Guarino. "Microbial therapy for cystic fibrosis." In The Human Microbiota and Chronic Disease, 497–506. Hoboken, NJ, USA: John Wiley & Sons, Inc., 2016. http://dx.doi.org/10.1002/9781118982907.ch32.

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Wadsworth, Samuel C., and Alan E. Smith. "Cystic fibrosis and lung diseases." In Molecular and Cell Biology of Human Gene Therapeutics, 237–51. Dordrecht: Springer Netherlands, 1995. http://dx.doi.org/10.1007/978-94-011-0547-7_12.

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Schnabel, Dirk. "Cystic Fibrosis – Growth Hormone Treatment." In Growth Hormone Therapy in Pediatrics - 20 Years of KIGS, 296–303. Basel: KARGER, 2007. http://dx.doi.org/10.1159/000101859.

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Gajbhiye, Rahul, and Avinash Gaikwad. "Cystic Fibrosis, CFTR Gene, and Male Infertility." In Male Infertility: Understanding, Causes and Treatment, 131–50. Singapore: Springer Singapore, 2017. http://dx.doi.org/10.1007/978-981-10-4017-7_9.

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Conference papers on the topic "Cystic fibrosis Gene therapy":

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Sinadinos, AJ, A. Sergijenko, AD Saleh, NAM Nafchi, JW Hickmott, T. Gamlen, DR Gill, SC Hyde, EWFW Alton, and U. Griesenbach. "S82 Quantification of mRNA and protein from single cells for cystic fibrosis gene therapy." In British Thoracic Society Winter Meeting, Wednesday 17 to Friday 19 February 2021, Programme and Abstracts. BMJ Publishing Group Ltd and British Thoracic Society, 2021. http://dx.doi.org/10.1136/thorax-2020-btsabstracts.87.

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Saleh, AD, NK Clarke, C. Meng, MR Jacobson, JC Davies, SR Durham, EWFW Alton, and U. Griesenbach. "S94 Development of assays to assess safety and efficacy of lentiviral gene therapy for cystic fibrosis." In British Thoracic Society Winter Meeting 2017, QEII Centre Broad Sanctuary Westminster London SW1P 3EE, 6 to 8 December 2017, Programme and Abstracts. BMJ Publishing Group Ltd and British Thoracic Society, 2017. http://dx.doi.org/10.1136/thoraxjnl-2017-210983.100.

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Collie, David, Peter Tennant, Catherine Gordon, Christina Vrettou, Alison Baker, Eilidh Baker, David Porteous, et al. "The Uk Cystic Fibrosis Gene Therapy Consortium: Normal Values And Reproducibility Of Forced Expiratory Flow Volume Curves In Sheep." In American Thoracic Society 2011 International Conference, May 13-18, 2011 • Denver Colorado. American Thoracic Society, 2011. http://dx.doi.org/10.1164/ajrccm-conference.2011.183.1_meetingabstracts.a2176.

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Saleh, AD, SR Durham, MH Shamji, U. Griesenbach, and EWFW Alton. "S19 Peak nasal inspiratory flow and nasal cytokines are useful biomarkers of nasal inflammation in cystic fibrosis gene therapy." In British Thoracic Society Winter Meeting 2019, QEII Centre, Broad Sanctuary, Westminster, London SW1P 3EE, 4 to 6 December 2019, Programme and Abstracts. BMJ Publishing Group Ltd and British Thoracic Society, 2019. http://dx.doi.org/10.1136/thorax-2019-btsabstracts2019.25.

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Coleman, Phoebe, and Bill Elder. "The journey of the Cystic Fibrosis gene." In SIGGRAPH '09: Posters. New York, New York, USA: ACM Press, 2009. http://dx.doi.org/10.1145/1599301.1599395.

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Nguyen, J. P., M. Bianca, R. D. Huff, N. Tiessen, Y. Kim, V. Hou, M. Heller, M. D. Inman, and J. A. Hirota. "Development of a Novel Combinatorial Therapy for Cystic Fibrosis." In American Thoracic Society 2019 International Conference, May 17-22, 2019 - Dallas, TX. American Thoracic Society, 2019. http://dx.doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2580.

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Tymchuk, I. V., M. A. Panas, Yu T. Konechnyi, O. P. Korniychuk, and V. V. Danyleichenko. "Pseudomonadaceae as vancomycin resistance gene reservoir in patients with cystic fibrosis." In NEW TRENDS AND UNRESOLVED ISSUES OF PREVENTIVE AND CLINICAL MEDICINE. Baltija Publishing, 2020. http://dx.doi.org/10.30525/978-9934-588-81-5-2.40.

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Yoshimura, K., C. Anzai, A. Miyamoto, H. Uruga, Y. Beika, N. Morokawa, and K. Kishi. "Analysis of CFTR Gene Mutations in Japanese Individuals with Cystic Fibrosis." In American Thoracic Society 2009 International Conference, May 15-20, 2009 • San Diego, California. American Thoracic Society, 2009. http://dx.doi.org/10.1164/ajrccm-conference.2009.179.1_meetingabstracts.a1201.

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Einhorn, Klaus, and Manfred Ballmann. "Pseudomonas aeruginosa eradication therapy on Cystic Fibrosis- Guideline and clinical routine." In ERS International Congress 2018 abstracts. European Respiratory Society, 2018. http://dx.doi.org/10.1183/13993003.congress-2018.pa1330.

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Mitchelmore, Philip, Louise Anning, Victoria Carnell, Sarah Jephcote, Pia Charters, Tim Crowe, Christopher Dean, Anna Lowdon, Christopher Sheldon, and Nicholas Withers. "Azithromycin therapy andpseudomonas aeruginosaisolation in a non-cystic fibrosis bronchiectasis cohort." In Annual Congress 2015. European Respiratory Society, 2015. http://dx.doi.org/10.1183/13993003.congress-2015.oa470.

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