Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fibrosis airway

Molecular Therapy

Volumn 10, Issue 6, 2004, Pages 990-1002

  Zhang, Liang N ^a   Karp, Phil ^a   Gerard, Christopher J ^b   Pastor, Eric ^b   Laux, Douglas ^c   Munson, Keith ^b   Yan, Ziying ^a   Liu, Xiaoming ^a   Godwin, Simon ^b   Thomas, Christie P ^a   Zabner, Joseph ^a   Shi, Huidong ^c   Caldwell, Charles W ^c   Peluso, Richard ^b   Carter, Barrie ^b   Engelhardt, John F ^a  

^a UNIVERSITY OF IOWA   (United States)

^b Targeted Genetics Corporation   (United States)

^c University of Missouri   (United States)

Author keywords

AAV; Adeno associated virus; CFTR; Cystic fibrosis; ENaC; Gene therapy

Indexed keywords

AMILORIDE; DOXORUBICIN; MESSENGER RNA; PARVOVIRUS VECTOR; PROTEASOME INHIBITOR; PROTEIN SUBUNIT; SODIUM CHANNEL; SODIUM ION; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG IDENTIFICATION; DRUG MECHANISM; GENE TARGETING; GENE THERAPY; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; HUMAN TISSUE; LUNG FIBROSIS; NONHUMAN; PROMOTER REGION; PROTEIN EXPRESSION; PROTEIN METHYLATION; RESPIRATORY EPITHELIUM; SHORT CIRCUIT CURRENT; VIRAL GENE DELIVERY SYSTEM; VIRUS INFECTION; VIRUS RECOMBINANT;

AMILORIDE; CELL POLARITY; CELLS, CULTURED; CPG ISLANDS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DEPENDOVIRUS; DNA METHYLATION; DOXORUBICIN; EPITHELIAL CELLS; EPITHELIAL SODIUM CHANNEL; GENE THERAPY; GENOME, VIRAL; HUMANS; PROMOTER REGIONS (GENETICS); PROTEASOME ENDOPEPTIDASE COMPLEX; RNA, MESSENGER; SODIUM; SODIUM CHANNELS; TIME FACTORS;

ADENO-ASSOCIATED VIRUS;

EID: 10344225049     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.08.009     Document Type: Article

References (46)

1. Q. Jiang J.F. Engelhardt Cellular heterogeneity of CFTR expression and function in the lung: Implications for gene therapy of cystic fibrosis Eur. J. Hum. Genet. 6 1998 12-31
2. M.J. Stutts B.C. Rossier R.C. Boucher Cystic fibrosis transmembrane conductance regulator inverts protein kinase A-mediated regulation of epithelial sodium channel single channel kinetics J. Biol. Chem. 272 1997 14037-14040
3. S.H. Donaldson E.G. Poligone M.J. Stutts CFTR regulation of ENaC Methods Mol. Med. 70 2002 343-364
4. S.E. Gabriel L.L. Clarke R.C. Boucher M.J. Stutts CFTR and outward rectifying chloride channels are distinct proteins with a regulatory relationship Nature 363 1993 263-268
5. E.M. Schwiebert M.M. Morales S. Devidas M.E. Egan W.B. Guggino Chloride channel and chloride conductance regulator domains of CFTR, the cystic fibrosis transmembrane conductance regulator Proc. Natl. Acad. Sci. USA 95 1998 2674-2679
6. P. Cahill et al. Identification of the cystic fibrosis transmembrane conductance regulator domains that are important for interactions with ROMK2 J. Biol. Chem. 275 2000 16697-16701
7. - conductance of Xenopus oocytes Pflugers Arch. 435 1997 178-181
8. M.R. Knowles R.C. Boucher Mucus clearance as a primary innate defense mechanism for mammalian airways J. Clin. Invest. 109 2002 571-577
9. W.B. Guggino Cystic fibrosis and the salt controversy Cell 96 1999 607-610
10. J.J. Smith S.M. Travis E.P. Greenberg M.J. Welsh Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid Cell 85 1996 229-236
11. R.C. Ahrens Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosis Pediatr. Pulmonol. 33 2002 142-150
12. R.C. Rubenstein P.L. Zeitlin A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: Partial restoration of nasal epithelial CFTR function Am. J. Respir. Crit. Care Med. 157 1998 484-490
13. M.R. Knowles et al. A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis N. Engl. J. Med. 322 1990 1189-1194
14. A. Graham et al. No added benefit from nebulized amiloride in patients with cystic fibrosis Eur. Respir. J. 6 1993 1243-1248
15. G. Pons et al. French multicenter randomized double-blind placebo-controlled trial on nebulized amiloride in cystic fibrosis patients: The Amiloride-AFLM Collaborative Study Group Pediatr. Pulmonol. 30 2000 25-31
16. M.L. Aitken et al. A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease Hum. Gene Ther. 12 2001 1907-1916
17. T.R. Flotte Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study Hum. Gene Ther. 14 2003 1079-1088
18. J.A. Wagner et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies Hum. Gene Ther. 13 2002 1349-1359
19. J.A. Wagner Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus Laryngoscope 109 1999 266-274
20. J. Zabner Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer J. Virol. 74 2000 3852-3858
21. D. Duan Y. Yue Z. Yan P.B. McCray Jr. J.F. Engelhardt Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia Hum. Gene Ther. 9 1998 2761-2776
22. R.W. Walters et al. Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer J. Biol. Chem. 276 2001 20610-20616
23. D. Duan Y. Yue Z. Yan J. Yang J.F. Engelhardt Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus J. Clin. Invest. 105 2000 1573-1587
24. W. Ding et al/. Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia J. Virol. 77 2003 7361-7366
25. T.R. Flotte et al. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter J. Biol. Chem. 268 1993 3781-3790
26. L. Zhang et al. Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes Proc. Natl. Acad. Sci. USA 95 1998 10158-10163
27. L.S. Ostedgaard et al. CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo Proc. Natl. Acad. Sci. USA 99 2002 3093-3098
28. D. Duan Y. Yue Z. Yan J.F. Engelhardt A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation Nat. Med. 6 2000 595-598
29. A. Auricchio et al. Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins J. Clin. Invest. 110 2002 499-504
30. J. Zabner J.J. Smith P.H. Karp J.H. Widdicombe M.J. Welsh Loss of CFTR chloride channels alters salt absorption by cystic fibrosis airway epithelia in vitro Mol. Cell 2 1998 397-403
31. N. Kaludov B. Handelman J.A. Chiorini Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography Hum. Gene Ther. 13 2002 1235-1243
32. C.M. Lynch K.L. Munson T.M. Stepan A.L. Feldhaus Small synthetic promoter elements increase expression of the full-length CFTR cDNA in AAV vectors Pediatr. Pulmonol. Suppl. 19 1999 230
33. J. Zabner et al. Development of cystic fibrosis and noncystic fibrosis airway cell lines Am. J. Physiol. Lung Cell Mol. Physiol. 284 2003 L844-L854
34. P.H. Karp et al. Epithelial Cell Culture Protocols Vol. 188 2002 Humana Press Totowa, NJ
35. C.J. Gerard J. Dell'Aringa K. An Hale W.M. Klump A sensitive, real-time, RNA-specific PCR method for the detection of recombinant AAV-CFTR vector expression Gene. Ther. 10 2003 1744-1753
36. M. Nakayama et al. Hypomethylation status of CpG sites at the promoter region and overexpression of the human MDR1 gene in acute myeloid leukemias Blood 92 1998 4296-4307
37. P.S. Yan et al. Dissecting complex epigenetic alterations in breast cancer using CpG island microarrays Cancer Res. 61 2001 8375-8380
38. Z. Yan et al. Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia J. Virol. 78 2004 2863-2874
39. L.G. Johnson et al. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis Nat. Genet. 2 1992 21-25
40. L.G. Johnson S.E. Boyles J. Wilson R.C. Boucher Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells J. Clin. Invest. 95 1995 1377-1382
41. H. Kusaba Association of 5 CpG demethylation and altered chromatin structure in the promoter region with transcriptional activation of the multidrug resistance 1 gene in human cancer cells Eur. J. Biochem. 262 1999 924-932
42. + channel gamma subunit promoter: Functional analysis and identification of a polypurine-polypyrimidine tract with the potential for triplex DNA formation Biochem. J. 347 Pt. 1 2000 105-114
43. + conductance by the cystic fibrosis transmembrane conductance regulator Pflugers Arch. 440 2000 193-201
44. + channel (ENaC) by ubiquitination EMBO J. 16 1997 6325-6336
45. B. Malik et al. Enac degradation in A6 cells by the ubiquitin-proteasome proteolytic pathway J. Biol. Chem. 276 2001 12903-12910
46. T. Ando M. Nishimura Y. Oka Decitabine (5-aza-2′-deoxycytidine) decreased DNA methylation and expression of MDR-1 gene in K562/ADM cells Leukemia 14 2000 1915-1920