173
Views
1
CrossRef citations to date
0
Altmetric
Original Research

Perceptions of airway gene therapy for cystic fibrosis

ORCID Icon, ORCID Icon & ORCID Icon
Pages 103-113 | Received 03 Aug 2022, Accepted 18 Nov 2022, Published online: 06 Dec 2022

References

  • Boucher RC. An overview of the pathogenesis of cystic fibrosis lung disease. Adv Drug Deliv Rev. 2002 Dec 5;54(11):1359–1371. DOI:10.1016/s0169-409x(02)00144-8.
  • Lopes-Pacheco M. CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine. Front Pharmacol. 2019;10:1662. DOI:10.3389/fphar.2019.01662.
  • Cooney AL, McCray PB Jr., Sinn PL. Cystic fibrosis gene therapy: looking back, looking forward. Genes (Basel). 2018 Nov 7;9(11). DOI:10.3390/genes9110538.
  • Alton E, Armstrong DK, Ashby D, et al. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. Lancet Respir Med. 2015 Sep;3(9):684–691. DOI:10.1016/S2213-2600(15)00245-3.
  • Pranke I, Golec A, Hinzpeter A, et al. Emerging therapeutic approaches for cystic fibrosis. from gene editing to personalized medicine. Front Pharmacol. 2019;10:121. DOI:10.3389/fphar.2019.00121.
  • Guggino WB, Cebotaru L. Gene therapy for cystic fibrosis paved the way for the use of Adeno-Associated virus in gene therapy. Hum Gene Ther. 2020 May;31(9–10):538–541. DOI:10.1089/hum.2020.046.
  • Yan Z, Feng Z, Sun X, et al. Human bocavirus Type-1 capsid facilitates the transduction of ferret airways by Adeno-Associated virus genomes. Hum Gene Ther. 2017 Aug;28(8):612–625. DOI:10.1089/hum.2017.060.
  • Cmielewski P, Donnelley M, Parsons DW. Long-term therapeutic and reporter gene expression in lentiviral vector treated cystic fibrosis mice. J Gene Med. 2014 Sep-Oct;16(9–10):291–299. DOI:10.1002/jgm.2778.
  • Cooney AL, Abou Alaiwa MH, Shah VS, et al. Lentiviral-mediated phenotypic correction of cystic fibrosis pigs. JCI Insight. 2016 Sep 8;1(14). DOI:10.1172/jci.insight.88730.
  • Donnelley M, Parsons DW. Gene therapy for cystic fibrosis lung disease: overcoming the barriers to translation to the clinic. Front Pharmacol. 2018;9:1381. DOI:10.3389/fphar.2018.01381.
  • Cao H, Ouyang H, Grasemann H, et al. Transducing airway basal cells with a helper-dependent adenoviral vector for lung gene therapy. Hum Gene Ther. 2018 Jun;29(6):643–652. DOI:10.1089/hum.2017.201.
  • Cooney AL, Singh BK, Loza LM, et al. Widespread airway distribution and short-term phenotypic correction of cystic fibrosis pigs following aerosol delivery of piggyBac/adenovirus. Nucleic Acids Res. 2018 Oct 12;46(18):9591–9600. DOI:10.1093/nar/gky773.
  • Delhove J, Osenk I, Prichard I, et al. Public acceptability of gene therapy and gene editing for human use: a systematic review. Hum Gene Ther. 2020 Jan;31(1–2):20–46. DOI:10.1089/hum.2019.197.
  • Deakin CT, Alexander IE, Kerridge I. Accepting risk in clinical research: is the gene therapy field becoming too risk-averse? Mol Ther. 2009 Nov;17(11):1842–1848. DOI:10.1038/mt.2009.223.
  • Jaffe A. Gene therapy for children with cystic fibrosis--who has the right to choose? J Med Ethics. 2006 Jun;32(6):361–364. DOI:10.1136/jme.2005.012740.
  • Iredale R, Dolan G, McDonald K, et al. Public attitudes to human gene therapy: a pilot study in Wales. Community Genet. 2003;6(3):139–146. DOI:10.1159/000078161.
  • Hoberman A, Shaikh N, Bhatnagar S, et al. Factors that influence parental decisions to participate in clinical research: consenters vs nonconsenters. JAMA Pediatr. 2013 Jun;167(6):561–566. DOI:10.1001/jamapediatrics.2013.1050.
  • Moorcraft SY, Marriott C, Peckitt C, et al. Patients’ willingness to participate in clinical trials and their views on aspects of cancer research: results of a prospective patient survey. Trials J. 2016 Jan 9;17(1):17. DOI:10.1186/s13063-015-1105-3.
  • Robillard JM, Roskams-Edris D, Kuzeljevic B, et al. Prevailing public perceptions of the ethics of gene therapy,” (in English). Hum Gene Ther. 2014 Aug;25(8):740–746. DOI:10.1089/hum.2014.030.
  • Chirumbolo S. Vaccination hesitancy and the “myth” on mRNA-based vaccines in Italy in the COVID-19 era: does urgency meet major safety criteria? J Med Virol. 2021 Jul;93(7):4049–4053. DOI:10.1002/jmv.26922.
  • Hamilton LC, Safford TG. Elite cues and the rapid decline in trust in science agencies on COVID-19. Soc Perspect. 2021;64(5):988–1011. DOI:10.1177/07311214211022391.
  • Jennings W, Stoker G, Willis H, et al. Lack of trust, conspiracy beliefs, and social media use predict COVID-19 vaccine hesitancy. Vaccines (Basel). 2021 Jun 3;9(6). DOI:10.3390/vaccines9060593.
  • Damase TR, Sukhovershin R, Boada C, et al. The limitless future of RNA therapeutics. Front Bioeng Biotechnol. 2021;9:628137.
  • Healey N. The gender gap in cystic fibrosis. Nature. 2020;583(7818):S10–S11. DOI:10.3389/fbioe.2021.628137.
  • Approved cellular and gene therapy products. U.S. Food & Drug Administration. [cited 2021 Jul 29]. Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
  • Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849–860. DOI:10.1016/S0140-6736(17)31868-8.
  • McMillan HJ, Proud CM, Farrar MA, et al. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy. Expert Opin Biol Ther. 2022 Sep;22(9):1075–1090. DOI:10.1080/14712598.2022.2066471.
  • Shahryari A, Saghaeian Jazi M, Mohammadi S, et al. Development and clinical translation of approved gene therapy products for genetic disorders. Front Genet. 2019;10:868. DOI:10.3389/fgene.2019.00868.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.