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New and emerging targeted therapies for cystic fibrosis.

Quon BS, Rowe SM.

BMJ. 2016 Mar 30;352:i859. doi: 10.1136/bmj.i859. Review.


Testing gene therapy vectors in human primary nasal epithelial cultures.

Cao H, Ouyang H, Ip W, Du K, Duan W, Avolio J, Wu J, Duan C, Yeger H, Bear CE, Gonska T, Hu J, Moraes TJ.

Mol Ther Methods Clin Dev. 2015 Sep 9;2:15034. doi: 10.1038/mtm.2015.34. eCollection 2015.


Cystic Fibrosis Gene Therapy in the UK and Elsewhere.

Griesenbach U, Pytel KM, Alton EW.

Hum Gene Ther. 2015 May;26(5):266-75. doi: 10.1089/hum.2015.027. Review.


Ferret and pig models of cystic fibrosis: prospects and promise for gene therapy.

Yan Z, Stewart ZA, Sinn PL, Olsen JC, Hu J, McCray PB Jr, Engelhardt JF.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):38-49. doi: 10.1089/humc.2014.154. Epub 2015 Feb 12. Review.


Gene therapy for the treatment of cystic fibrosis.

Burney TJ, Davies JC.

Appl Clin Genet. 2012 May 29;5:29-36. doi: 10.2147/TACG.S8873. Print 2012.


Adenoviral gene transfer corrects the ion transport defect in the sinus epithelia of a porcine CF model.

Potash AE, Wallen TJ, Karp PH, Ernst S, Moninger TO, Gansemer ND, Stoltz DA, Zabner J, Chang EH.

Mol Ther. 2013 May;21(5):947-53. doi: 10.1038/mt.2013.49. Epub 2013 Mar 19.


Advances in cell and gene-based therapies for cystic fibrosis lung disease.

Oakland M, Sinn PL, McCray PB Jr.

Mol Ther. 2012 Jun;20(6):1108-15. doi: 10.1038/mt.2012.32. Epub 2012 Feb 28. Review.


Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: effect of acyl chain length.

Cmielewski P, Anson DS, Parsons DW.

Respir Res. 2010 Jun 23;11:84. doi: 10.1186/1465-9921-11-84.


Cystic fibrosis pigs develop lung disease and exhibit defective bacterial eradication at birth.

Stoltz DA, Meyerholz DK, Pezzulo AA, Ramachandran S, Rogan MP, Davis GJ, Hanfland RA, Wohlford-Lenane C, Dohrn CL, Bartlett JA, Nelson GA 4th, Chang EH, Taft PJ, Ludwig PS, Estin M, Hornick EE, Launspach JL, Samuel M, Rokhlina T, Karp PH, Ostedgaard LS, Uc A, Starner TD, Horswill AR, Brogden KA, Prather RS, Richter SS, Shilyansky J, McCray PB Jr, Zabner J, Welsh MJ.

Sci Transl Med. 2010 Apr 28;2(29):29ra31. doi: 10.1126/scitranslmed.3000928.


Concordant activity of transgene expression cassettes inserted into E1, E3 and E4 cloning sites in the adenovirus genome.

Pham L, Nakamura T, Gabriela Rosales A, Carlson SK, Bailey KR, Peng KW, Russell SJ.

J Gene Med. 2009 Mar;11(3):197-206. doi: 10.1002/jgm.1289.


Activation of a dendritic cell-T cell axis by Ad5 immune complexes creates an improved environment for replication of HIV in T cells.

Perreau M, Pantaleo G, Kremer EJ.

J Exp Med. 2008 Nov 24;205(12):2717-25. doi: 10.1084/jem.20081786. Epub 2008 Nov 3.


Lentivirus vector can be readministered to nasal epithelia without blocking immune responses.

Sinn PL, Arias AC, Brogden KA, McCray PB Jr.

J Virol. 2008 Nov;82(21):10684-92. doi: 10.1128/JVI.00227-08. Epub 2008 Sep 3.


Protective immunity against botulism provided by a single dose vaccination with an adenovirus-vectored vaccine.

Zeng M, Xu Q, Elias M, Pichichero ME, Simpson LL, Smith LA.

Vaccine. 2007 Oct 23;25(43):7540-8. Epub 2007 Sep 5.


Detection of cystic fibrosis transmembrane conductance regulator activity in early-phase clinical trials.

Rowe SM, Accurso F, Clancy JP.

Proc Am Thorac Soc. 2007 Aug 1;4(4):387-98. Review.


Recombinant adenoviral vectors can induce expression of p73 via the E4-orf6/7 protein.

Shapiro GS, Van Peursem C, Ornelles DA, Schaack J, DeGregori J.

J Virol. 2006 Jun;80(11):5349-60.


Replicating rather than nonreplicating adenovirus-human immunodeficiency virus recombinant vaccines are better at eliciting potent cellular immunity and priming high-titer antibodies.

Peng B, Wang LR, Gómez-Román VR, Davis-Warren A, Montefiori DC, Kalyanaraman VS, Venzon D, Zhao J, Kan E, Rowell TJ, Murthy KK, Srivastava I, Barnett SW, Robert-Guroff M.

J Virol. 2005 Aug;79(16):10200-9.


Immunological hurdles to lung gene therapy.

Ferrari S, Griesenbach U, Geddes DM, Alton E.

Clin Exp Immunol. 2003 Apr;132(1):1-8. Review.


Current status of gene therapy for inherited lung diseases.

Driskell RA, Engelhardt JF.

Annu Rev Physiol. 2003;65:585-612. Epub 2002 May 1. Review.


Bronchoalveolar fluid is not a major hindrance to virus-mediated gene therapy in cystic fibrosis.

Rooney CP, Denning GM, Davis BP, Flaherty DM, Chiorini JA, Zabner J.

J Virol. 2002 Oct;76(20):10437-43.

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