Attenuation of monocrotaline-induced pulmonary hypertension by luminal adeno-associated virus serotype 9 gene transfer of prostacyclin synthase

Human Gene Therapy

Volumn 25, Issue 6, 2014, Pages 498-505

  Gubrij, Igor B ^a,b   Martin, Sara Rebecca ^a,b   Pangle, Amanda K ^a,b   Kurten, Richard ^c,d   Johnson, Larry G ^a,b  

^a UNIVERSITY OF ARKANSAS FOR MEDICAL SCIENCES   (United States)

^b CENTRAL ARKANSAS VETERANS HEALTHCARE SYSTEM   (United States)

^c ARKANSAS CHILDREN S HOSPITAL   (United States)

^d UNIVERSITY OF ARKANSAS FOR MEDICAL SCIENCES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

6 OXOPROSTAGLANDIN F1 ALPHA; BETA ACTIN; MONOCROTALINE; PROSTACYCLIN SYNTHASE; SODIUM CHLORIDE; CYTOCHROME P450; ISOMERASE;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 9; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ATTENUATION; CONTROLLED STUDY; GENE TRANSFER; HEMODYNAMICS; INTRALUMINAL DRUG ADMINISTRATION; LUNG; LUNG HOMOGENATE; NONHUMAN; NONVIRAL GENE THERAPY; POLYMERASE CHAIN REACTION; PROMOTER REGION; PROSTAGLANDIN BLOOD LEVEL; PROTEIN EXPRESSION; PULMONARY ARTERY; PULMONARY HYPERTENSION; RAT; SEROTYPE; SYSTOLIC BLOOD PRESSURE; THICKNESS; TISSUE SECTION; VIRAL GENE DELIVERY SYSTEM; ANIMAL; BIOSYNTHESIS; CHEMICALLY INDUCED; DEPENDOPARVOVIRUS; ENZYME INDUCTION; FISCHER 344 RAT; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HYPERTENSION, PULMONARY; PATHOPHYSIOLOGY; SPRAGUE DAWLEY RAT; VASCULAR REMODELING;

ANIMALS; CYTOCHROME P-450 ENZYME SYSTEM; DEPENDOVIRUS; ENZYME INDUCTION; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HYPERTENSION, PULMONARY; INTRAMOLECULAR OXIDOREDUCTASES; MONOCROTALINE; PROMOTER REGIONS, GENETIC; RATS, INBRED F344; RATS, SPRAGUE-DAWLEY; TRANSDUCTION, GENETIC; VASCULAR REMODELING;

EID: 84902977193     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.187     Document Type: Article

References (38)

1. Auricchio, A., O'connor, E., Weiner, D., et al. (2002). Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J. Clin. Invest. 110, 499-504.
2. Bal, E., Ilgin, S., Atli, O., et al. (2013). The effects of gender difference on monocrotaline-induced pulmonary hypertension in rats. Hum. Exp. Toxicol. 32, 766-774.
3. Campbell, A.I., Kuliszewski, M.A., and Stewart, D.J. (1999). Cell-based gene transfer to the pulmonary vasculature: endothelial nitric oxide synthase overexpression inhibits monocrotaline-induced pulmonary hypertension. Am. J. Respir. Cell Mol. Biol. 21, 567-575. (Pubitemid 129586592)
4. Campbell, A.I., Zhao, Y., Sandhu, R., and Stewart, D.J. (2001). Cell-based gene transfer of vascular endothelial growth factor attenuates monocrotaline-induced pulmonary hypertension. Circulation 104, 2242-2248. (Pubitemid 33043307)
5. Chao, H., Liu, Y., Rabinowitz, J., et al. (2000). Several log increase in therapeutic transgene delivery by distinct adenoassociated viral serotype vectors. Mol. Ther. 2, 619-623.
6. Deng, Z., Morse, J.H., Slager, S.L., et al. (2000). Familial primary pulmonary hypertension (gene PPH1) is caused by mutations in the bone morphogenetic protein receptor-II gene. Am. J. Hum. Genet. 67, 737-744. (Pubitemid 30659603)
7. Fisher, K.J., Gao, G.P., Weitzman, M.D., et al. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532. (Pubitemid 126508820)
8. Flotte, T.R., Fischer, A.C., Goetzmann, J., et al. (2010). Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. Mol. Ther.18, 594-600.
9. Flotte, T.R., Trapnell, B.C., Humphries, M., et al. (2011). Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum. Gene Ther. 22, 1239-1247.
10. Galie, N., and Branzi, A. (2005). Pulmonary arterial hypertension: therapeutic algorithm. Ital. Heart J. 6, 856-860.
11. Galie, N., Badesch, D., Oudiz, R., et al. (2005). Ambrisentan therapy for pulmonary arterial hypertension. J. Am. Coll. Cardiol. 46, 529-535. (Pubitemid 41040298)
12. Geraci, M.W., Gao, B., Shepherd, D.C., et al. (1999). Pulmonary prostacyclin synthase overexpression in transgenic mice protects against development of hypoxic pulmonary hypertension. J. Clin. Invest. 103, 1509-1515.
13. Grieger, J.C., Choi, V.W., and Samulski, R.J. (2006). Production and characterization of adeno-associated viral vectors. Nat. Protoc. 1, 1412-1428.
14. Hadri, L., Kratlian, R.G., Benard, L., et al. (2013). Therapeutic efficacy of AAV1.SERCA2a in monocrotaline-induced pulmonary arterial hypertension. Circulation 128, 512-523.
15. Halbert, C.L., Allen, J.M., and Miller, A.D. (2001). Adenoassociated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J. Virol. 75, 6615-6624. (Pubitemid 32578181)
16. International PPH Consortium, Lane, K.B., Machado, R.D., et al. (2000). Heterozygous germline mutations in BMPR2, encoding a TGF-beta receptor, cause familial primary pulmonary hypertension. Nat. Genet. 26, 81-84.
17. Ito, T., Okada, T., Mimuro, J., et al. (2007). Adenoassociated virus-mediated prostacyclin synthase expression prevents pulmonary arterial hypertension in rats. Hypertension 50, 531-536. (Pubitemid 47312240)
18. Johnson, L.G., Vanhook, M.K., Coyne, C.B., et al. (2003). Safety and efficiency of modulating paracellular permeability to enhance airway epithelial gene transfer in vivo. Hum. Gene Ther. 14, 729-747. (Pubitemid 36623511)
19. Kataoka, M., Kawakami, T., Tamura, Y., et al. (2013). Gene transfer therapy by either type 1 or type 2 adeno-associated virus expressing human prostaglandin I2 synthase gene is effective for treatment of pulmonary arterial hypertension. J. Cardiovasc. Pharmacol. Ther. 18, 54-59.
20. Kawakami, T., Kanazawa, H., Satoh, T., et al. (2007). AAVPGIS gene transfer improves hypoxia-induced pulmonary hypertension in mice. Biochem. Biophys. Res. Commun. 363, 656-661. (Pubitemid 47513680)
21. Kido, M., Du, L., Sullivan, C.C., et al. (2005). Gene transfer of a TIE2 receptor antagonist prevents pulmonary hypertension in rodents. J. Thorac. Cardiovasc. Surg. 129, 268-276. (Pubitemid 40169548)
22. Limberis, M.P., and Wilson, J.M. (2006). Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc. Natl. Acad. Sci. USA 103, 12993-12998. (Pubitemid 44338905)
23. Limberis, M.P., Vandenberghe, L.H., Zhang, L., et al. (2009). Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol. Ther. 17, 294-301.
24. Mclaughlin, V. (2013). Managing pulmonary arterial hypertension and optimizing treatment options: prognosis of pulmonary artery hypertension. Am. J. Cardiol. 111, 10C-15C.
25. Mclaughlin, V.V., Shillington, A., and Rich, S. (2002). Survival in primary pulmonary hypertension: the impact of epoprostenol therapy. Circulation 106, 1477-1482.
26. Mclaughlin, V.V., Sitbon, O., Badesch, D.B., et al. (2005). Survival with first-line bosentan in patients with primary pulmonary hypertension. Eur. Respir. J. 25, 244-249. (Pubitemid 40246523)
27. McMurtry, M.S., Archer, S.L., Altieri, D.C., et al. (2005). Gene therapy targeting survivin selectively induces pulmonary vascular apoptosis and reverses pulmonary arterial hypertension. J. Clin. Invest. 115, 1479-1491. (Pubitemid 40814657)
28. Moss, R.B., Rodman, D., Spencer, L.T., et al. (2004). 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 125, 509-521. (Pubitemid 38223630)
29. Nagaya, N., Yokoyama, C., Kyotani, S., et al. (2000). Gene transfer of human prostacyclin synthase ameliorates monocrotaline- induced pulmonary hypertension in rats. Circulation 102, 2005-2010.
30. Ono, M., Sawa, Y., Fukushima, N., et al. (2004). Gene transfer of hepatocyte growth factor with prostacyclin synthase in severe pulmonary hypertension of rats. Eur. J. Cardiothorac. Surg. 26, 1092-1097. (Pubitemid 39487453)
31. Pan, L.C., Wilson, D.W., and Segall, H.J. (1993). Strain differences in the response of Fischer 344 and Sprague-Dawley rats to monocrotaline induced pulmonary vascular disease. Toxicology 79, 21-35.
32. Rubin, L.J., Badesch, D.B., Barst, R.J., et al. (2002). Bosentan therapy for pulmonary arterial hypertension. N. Engl. J. Med. 346, 896-903. (Pubitemid 34984638)
33. Shintani, M., Yagi, H., Nakayama, T., et al. (2009). A new nonsense mutation of SMAD8 associated with pulmonary arterial hypertension. J. Med. Genet. 46, 331-337.
34. Sitbon, O., Mclaughlin, V.V., Badesch, D.B., et al. (2005). Survival in patients with class III idiopathic pulmonary arterial hypertension treated with first line oral bosentan compared with an historical cohort of patients started on intravenous epoprostenol. Thorax 60, 1025-1030. (Pubitemid 41705471)
35. Song, S., Morgan, M., Ellis, T., et al. (1998). Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 95, 14384-14388. (Pubitemid 28549376)
36. Stripp, B.R., Sawaya, P.L., Luse, D.S., et al. (1992). Cis-acting elements that confer lung epithelial cell expression of the CC10 gene. J. Biol. Chem. 267, 14703-14712.
37. Zabner, J., Seiler, M.,Walters, R., et al. (2000). Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J. Virol. 74, 3852-3858. (Pubitemid 30180327)
38. Zhao, Y.D., Courtman, D.W., Deng, Y., et al. (2005). Rescue of monocrotaline-induced pulmonary arterial hypertension using bone marrow-derived endothelial-like progenitor cells: efficacy of combined cell and eNOS gene therapy in established disease. Circ. Res. 96, 442-450. (Pubitemid 40354538)