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Bovine AAV transcytosis inhibition by tannic acid results in functional expression of CFTR in vitro and altered biodistribution in vivo.

Di Pasquale G, Ostedgaard L, Vermeer D, Swaim WD, Karp P, Chiorini JA.

Gene Ther. 2012 May;19(5):576-81. doi: 10.1038/gt.2011.138. Epub 2011 Oct 20.


Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ.

Proc Natl Acad Sci U S A. 1993 Nov 15;90(22):10613-7.


Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, Manley S, Norbash AM, Conrad CK, Friborg S, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P.

Laryngoscope. 1999 Feb;109(2 Pt 1):266-74.


Adenovirus 5-fiber 35 chimeric vector mediates efficient apical correction of the cystic fibrosis transmembrane conductance regulator defect in cystic fibrosis primary airway epithelia.

Granio O, Ashbourne Excoffon KJ, Henning P, Melin P, Norez C, Gonzalez G, Karp PH, Magnusson MK, Habib N, Lindholm L, Becq F, Boulanger P, Zabner J, Hong SS.

Hum Gene Ther. 2010 Mar;21(3):251-69. doi: 10.1089/hum.2009.056.


Species-specific differences in mouse and human airway epithelial biology of recombinant adeno-associated virus transduction.

Liu X, Yan Z, Luo M, Engelhardt JF.

Am J Respir Cell Mol Biol. 2006 Jan;34(1):56-64. Epub 2005 Sep 29.


Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells.

White AF, Mazur M, Sorscher EJ, Zinn KR, Ponnazhagan S.

Hum Gene Ther. 2008 Dec;19(12):1407-14. doi: 10.1089/hgt.2008.117.


Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter.

Flotte TR, Afione SA, Solow R, Drumm ML, Markakis D, Guggino WB, Zeitlin PL, Carter BJ.

J Biol Chem. 1993 Feb 15;268(5):3781-90.


Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes.

Zhang L, Wang D, Fischer H, Fan PD, Widdicombe JH, Kan YW, Dong JY.

Proc Natl Acad Sci U S A. 1998 Aug 18;95(17):10158-63.


Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector.

Sirninger J, Muller C, Braag S, Tang Q, Yue H, Detrisac C, Ferkol T, Guggino WB, Flotte TR.

Hum Gene Ther. 2004 Sep;15(9):832-41.


A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.

Ostedgaard LS, Rokhlina T, Karp PH, Lashmit P, Afione S, Schmidt M, Zabner J, Stinski MF, Chiorini JA, Welsh MJ.

Proc Natl Acad Sci U S A. 2005 Feb 22;102(8):2952-7. Epub 2005 Feb 9.


Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice.

Ostedgaard LS, Meyerholz DK, Vermeer DW, Karp PH, Schneider L, Sigmund CD, Welsh MJ.

Proc Natl Acad Sci U S A. 2011 Feb 15;108(7):2921-6. doi: 10.1073/pnas.1019752108. Epub 2011 Feb 1.


Single-dose lentiviral gene transfer for lifetime airway gene expression.

Stocker AG, Kremer KL, Koldej R, Miller DS, Anson DS, Parsons DW.

J Gene Med. 2009 Oct;11(10):861-7. doi: 10.1002/jgm.1368.


Efficient CFTR expression from AAV vectors packaged with promoters--the second generation.

Wang D, Fischer H, Zhang L, Fan P, Ding RX, Dong J.

Gene Ther. 1999 Apr;6(4):667-75.


Cloning and characterization of a bovine adeno-associated virus.

Schmidt M, Katano H, Bossis I, Chiorini JA.

J Virol. 2004 Jun;78(12):6509-16.


Gene therapy in cystic fibrosis.

Flotte TR, Laube BL.

Chest. 2001 Sep;120(3 Suppl):124S-131S. Review.


Regulation of cystic fibrosis transmembrane conductance regulator by microRNA-145, -223, and -494 is altered in ΔF508 cystic fibrosis airway epithelium.

Oglesby IK, Chotirmall SH, McElvaney NG, Greene CM.

J Immunol. 2013 Apr 1;190(7):3354-62. doi: 10.4049/jimmunol.1202960. Epub 2013 Feb 22.


Adeno-associated virus vectors for gene therapy of cystic fibrosis.

Flotte TR, Carter BJ.

Methods Enzymol. 1998;292:717-32. No abstract available.


Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers.

Yan Z, Sun X, Feng Z, Li G, Fisher JT, Stewart ZA, Engelhardt JF.

Hum Gene Ther. 2015 Jun;26(6):334-46. doi: 10.1089/hum.2015.001. Epub 2015 Apr 20.

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