Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

Thorax

Volumn 72, Issue 2, 2017, Pages 137-147

  Alton, Eric W F W ^a,b   Beekman, Jeffery M ^c   Boyd, A Christopher ^b,d   Brand, June ^d,e   Carlon, Marianne S ^f   Connolly, Mary M ^b,g   Chan, Mario ^a,b   Conlon, Sinead ^a,b   Davidson, Heather E ^b,d   Davies, Jane C ^a,b   Davies, Lee A ^b,g   Dekkers, Johanna F ^c   Doherty, Ann ^b,d   Gea Sorli, Sabrina ^a,b   Gill, Deborah R ^b,g   Griesenbach, Uta ^a,b   Hasegawa, Mamoru ^h   Higgins, Tracy E ^a,b   Hironaka, Takashi ^h   Hyndman, Laura ^b,d   McLachlan, Gerry ^b,i   Inoue, Makoto ^h   Hyde, Stephen C ^b,g   Innes, J Alastair ^b,d   Maher, Toby M ^a   Moran, Caroline ^a,b   Meng, Cuixiang ^a,b   Paul Smith, Michael C ^a,b   Pringle, Ian A ^b,g   Pytel, Kamila M ^a,b   Rodriguez Martinez, Andrea ^a,b   Schmidt, Alexander C ^j   Stevenson, Barbara J ^b,d   Sumner Jones, Stephanie G ^b,g   Toshner, Richard ^a   Tsugumine, Shu ^h   Wasowicz, Marguerite W ^a,b   Zhu, Jie ^e   more..

^a NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^b UK Cystic Fibrosis Gene Therapy Consortium   (United Kingdom)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d UNIVERSITY OF EDINBURGH   (United Kingdom)

^e IMPERIAL COLLEGE LONDON   (United Kingdom)

^f UNIVERSITY OF LEUVEN   (Belgium)

^g JOHN RADCLIFFE HOSPITAL   (United Kingdom)

^h ID Pharme Co Ltd   (Japan)

ⁱ UNIVERSITY OF EDINBURGH   (United Kingdom)

^j NONE   (Belgium)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; ELONGATION FACTOR 1ALPHA; ENHANCED GREEN FLUORESCENT PROTEIN; HUMAN PARAINFLUENZA VIRUS 1 ANTIBODY; IMMUNOGLOBULIN; LENTIVIRUS VECTOR; UNCLASSIFIED DRUG; VIRUS ANTIBODY; ELONGATION FACTOR 1;

ACUTE TOXICITY; ADAPTIVE IMMUNITY; ADULT; AIRWAY EPITHELIUM CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY TITER; ANTIGEN SPECIFICITY; ARTICLE; CHILD; CONTROLLED STUDY; CPG ISLAND; DRUG EFFICACY; EX VIVO STUDY; GENE EXPRESSION REGULATION; HUMAN; HUMAN TISSUE; IN VIVO STUDY; LUNG FIBROSIS; MOUSE; NONHUMAN; RSIV F HN GENE; SINGLE DRUG DOSE; TRANSCRIPTION INITIATION SITE; VIRAL GENE THERAPY; VIRUS TITRATION; ANIMAL; CYSTIC FIBROSIS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; LENTIVIRINAE; PROCEDURES; PROMOTER REGION;

ANIMALS; CYSTIC FIBROSIS; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LENTIVIRUS; MICE; PEPTIDE ELONGATION FACTOR 1; PROMOTER REGIONS, GENETIC;

EID: 84998812292     PISSN: 00406376     EISSN: 14683296     Source Type: Journal    
DOI: 10.1136/thoraxjnl-2016-208406     Document Type: Article

References (48)

1. Kobayashi M, Iida A, Ueda Y, et al. Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus. J Virol 2003;77:2607-14.
2. Griesenbach U, McLachlan G, Owaki T, et al. Validation of recombinant Sendai virus in a non-natural host model. Gene Ther 2011;18:182-8.
3. Mitomo K, Griesenbach U, Inoue M, et al. Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes. Mol Ther 2010;18:1173-82.
4. Griesenbach U, Inoue M, Meng C, et al. Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy. Am J Respir Crit Care Med 2012;186:846-56.
5. Cmielewski P, Anson DS, Parsons DW. Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: effect of acyl chain length. Respir Res 2010;11:84.
6. Sinn PL, Cooney AL, Oakland M, et al. Lentiviral vector gene transfer to porcine airways. Mol Ther Nucleic Acids 2012;1:e56.
7. Hacein-Bey Abina S, Gaspar HB, Blondeau J, et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 2015;313:1550-63.
8. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Gene therapy of primary T cell immunodeficiencies. Gene 2013;525:170-3.
9. Boztug K, Schmidt M, Schwarzer A, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 2010;363:1918-27.
10. Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with wiskott-aldrich syndrome. Science 2013;341: 1233151.
11. Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013;341:1233158.
12. Palfi S, Gurruchaga JM, Ralph GS, et al. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet 2014;383:1138-46.
13. Yáñez-Munõz RJ, Balaggan KS, MacNeil A, et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 2006;12:348-53.
14. Alton EW, 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;3:684-91.
15. Sinn PL, Arias AC, Brogden KA, et al. Lentivirus vector can be readministered to nasal epithelia without blocking immune responses. J Virol 2008;82:10684-92.
16. Gorman WL, Gill DS, Scroggs RA, et al. The hemagglutinin-neuraminidase glycoproteins of human parainfluenza virus type 1 and Sendai virus have high structure-function similarity with limited antigenic cross-reactivity. Virology 1990;175:211-21.
17. Lamb RA, Kolakofsky D. Paramyxoviridae: the viruses and their replication. In: Fields BN, Knipe DM, Howley PM, eds. Fields virology. 3rd edn. Philadelphia, PA: Lippincott-Raven Publishers, 1996:1177-204.
18. Ferrari S, Griesenbach U, Iida A, et al. Sendai virus-mediated CFTR gene transfer to the airway epithelium. Gene Ther 2007;14:1371-9.
19. Griesenbach U, Boyton RJ, Somerton L, et al. Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lung. Gene Ther 2006;13:449-56.
20. Dekkers JF, Wiegerinck CL, de Jonge HR, et al. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat Med 2013;19:939-45.
21. Zhang L, Thrasher AJ, Gaspar HB. Current progress on gene therapy for primary immunodeficiencies. Gene Ther 2013;20:963-9.
22. Stewart HJ, Leroux-Carlucci MA, Sion CJ, et al. Development of inducible EIAV-based lentiviral vector packaging and producer cell lines. Gene Ther 2009;16:805-14.
23. Gill DR, Smyth SE, Goddard CA, et al. Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter. Gene Ther 2001;8:1539-46.
24. Hyde SC, Pringle IA, Abdullah S, et al. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. Nat Biotechnol 2008;26:549-51.
25. Farmen SL, Karp PH, Ng P, et al. Gene transfer of CFTR to airway epithelia: low levels of expression are sufficient to correct Cl-transport and overexpression can generate basolateral CFTR. Am J Physiol Lung Cell Mol Physiol 2005;289: L1123-30.
26. Wilson AA, Murphy GJ, Hamakawa H, et al. Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages. J Clin Invest 2010;120:379-89.
27. Griesenbach U, Inoue M, Hasegawa M, et al. Sendai virus for gene therapy and vaccination. Curr Opin Mol Ther 2005;7:346-52.
28. Nienhuis AW, Dunbar CE, Sorrentino BP. Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 2006;13:1031-49.
29. Bartholomae CC, Arens A, Balaggan KS, et al. Lentiviral vector integration profiles differ in rodent postmitotic tissues. Mol Ther 2011;19:703-10.
30. Lattanzi A, Salvagno C, Maderna C, et al. Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy. Hum Mol Genet 2014;23:3250-68.
31. Yang Y, Li Q, Ertl HC, et al. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 1995;69:2004-15.
32. Moss RB, Milla C, Colombo J, et al. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Hum Gene Ther 2007;18:726-32.
33. Nichols TC, Whitford MH, Arruda VR, et al. Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev 2015;26:5-14.
34. Wilson JM, Limberis M. The challenge of developing animal models of human gene therapy with AAV [abstract]. Mol Ther 2011;19:S1-S361.
35. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 2003;4:346-58.
36. Cichon G, Boeckh-Herwig S, Schmidt HH, et al. Complement activation by recombinant adenoviruses. Gene Ther 2001;8:1794-800.
37. Henrickson KJ. Parainfluenza viruses. Clin Microbiol Rev 2003;16:242-64.
38. Moss RB, Rodman D, Spencer LT, et al. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest 2004;125:509-21.
39. Bastian A, Bewig B. Inhibition of adenovirus-mediated gene transfer by bronchoalveolar lavage fluid. Gene Ther 1999;6:637-42.
40. Griesenbach U, Smith SN, Farley R, et al. Validation of nasal potential difference measurements in gut-corrected CF knockout mice. Am J Respir Cell Mol Biol 2008;39:490-6.
41. Ostrowski LE, Yin W, Diggs PS, et al. Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice. Gene Ther 2007;14:1492-501.
42. Grubb BR, Rogers TD, Boucher RC, et al. Ion transport across CF and normal murine olfactory and ciliated epithelium. Am J Physiol, Cell Physiol 2009;296: C1301-9.
43. Alton EW, Stern M, Farley R, et al. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 1999;353:947-54.
44. Griesenbach U, Sumner-Jones SG, Holder E, et al. Limitations of the murine nose in the development of nonviral airway gene transfer. Am J Respir Cell Mol Biol 2010;43:46-54.
45. Jiang C, O'Connor SP, Fang SL, et al. Efficiency of cationic lipid-mediated transfection of polarized and differentiated airway epithelial cells in vitro and in vivo. Hum Gene Ther 1998;9:1531-42.
46. Caplen NJ, Alton EW, Middleton PG, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995;1:39-46.
47. Alton EW, Middleton PG, Caplen NJ, et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet 1993;5:135-42.
48. Zhang F, Frost AR, Blundell MP, et al. A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors. Mol Ther 2010;18:1640-9.