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Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques.

Fischer AC, Beck SE, Smith CI, Laube BL, Askin FB, Guggino SE, Adams RJ, Flotte TR, Guggino WB.

Mol Ther. 2003 Dec;8(6):918-26.


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.

Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, Conrad CK, Brass-Ernst L, Humphries M, Sullivan K, Wetzel R, Taylor G, Carter BJ, Guggino WB.

Hum Gene Ther. 2003 Jul 20;14(11):1079-88.


Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques.

Beck SE, Laube BL, Barberena CI, Fischer AC, Adams RJ, Chesnut K, Flotte TR, Guggino WB.

Mol Ther. 2002 Oct;6(4):546-54.


Repeated delivery of adeno-associated virus vectors to the rabbit airway.

Beck SE, Jones LA, Chesnut K, Walsh SM, Reynolds TC, Carter BJ, Askin FB, Flotte TR, Guggino WB.

J Virol. 1999 Nov;73(11):9446-55.


Adeno-associated virus serotype 2-mediated gene transfer to the parotid glands of nonhuman primates.

Voutetakis A, Zheng C, Mineshiba F, Cotrim AP, Goldsmith CM, Schmidt M, Afione S, Roescher N, Metzger M, Eckhaus MA, Chiorini JA, Dunbar CE, Donahue RE, Baum BJ.

Hum Gene Ther. 2007 Feb;18(2):142-50.


Noninvasive Imaging Reveals Stable Transgene Expression in Mouse Airways After Delivery of a Nonintegrating Recombinant Adeno-Associated Viral Vector.

Vidović D, Gijsbers R, Quiles Jimenez A, Dooley J, Van den Haute C, Van der Perren A, Liston A, Baekelandt V, Debyser Z, Carlon MS.

Hum Gene Ther. 2016 Jan;27(1):60-71. doi: 10.1089/hum.2015.109. Epub 2015 Dec 21.


Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented.

Peden CS, Manfredsson FP, Reimsnider SK, Poirier AE, Burger C, Muzyczka N, Mandel RJ.

Mol Ther. 2009 Mar;17(3):524-37. doi: 10.1038/mt.2008.284. Epub 2009 Jan 13.


Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.

Ciron C, Cressant A, Roux F, Raoul S, Cherel Y, Hantraye P, Déglon N, Schwartz B, Barkats M, Heard JM, Tardieu M, Moullier P, Colle MA.

Hum Gene Ther. 2009 Apr;20(4):350-60. doi: 10.1089/hum.2008.155.


Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.

Unzu C, Hervás-Stubbs S, Sampedro A, Mauleón I, Mancheño U, Alfaro C, de Salamanca RE, Benito A, Beattie SG, Petry H, Prieto J, Melero I, Fontanellas A.

J Transl Med. 2012 Jun 15;10:122. doi: 10.1186/1479-5876-10-122.


In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates.

Garrett DJ, Larson JE, Dunn D, Marrero L, Cohen JC.

BMC Biotechnol. 2003 Sep 30;3:16.


Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.

Loring HS, ElMallah MK, Flotte TR.

Hum Gene Ther Methods. 2016 Apr;27(2):49-58. doi: 10.1089/hgtb.2015.150. Epub 2016 Feb 19. Review.


Gene therapy in cystic fibrosis.

Flotte TR, Laube BL.

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


AAV5-mediated gene transfer to the parotid glands of non-human primates.

Voutetakis A, Zheng C, Cotrim AP, Mineshiba F, Afione S, Roescher N, Swaim WD, Metzger M, Eckhaus MA, Donahue RE, Dunbar CE, Chiorini JA, Baum BJ.

Gene Ther. 2010 Jan;17(1):50-60. doi: 10.1038/gt.2009.123. Epub 2009 Sep 17.


Aerosol-mediated delivery of AAV2/6-IκBα attenuates lipopolysaccharide-induced acute lung injury in rats.

MacLoughlin RJ, Higgins BD, Devaney J, O'Toole D, Laffey JG, O'Brien T.

Hum Gene Ther. 2015 Jan;26(1):36-46. doi: 10.1089/hum.2014.053.


Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.

Rolling F, Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Péréon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F.

Bull Mem Acad R Med Belg. 2006;161(10-12):497-508; discussion 508-9.


Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways.

Sumner-Jones SG, Davies LA, Varathalingam A, Gill DR, Hyde SC.

Gene Ther. 2006 Dec;13(24):1703-13. Epub 2006 Jul 20.


Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors.

Chen S, Agarwal A, Glushakova OY, Jorgensen MS, Salgar SK, Poirier A, Flotte TR, Croker BP, Madsen KM, Atkinson MA, Hauswirth WW, Berns KI, Tisher CC.

J Am Soc Nephrol. 2003 Apr;14(4):947-58.


Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat.

Reimsnider S, Manfredsson FP, Muzyczka N, Mandel RJ.

Mol Ther. 2007 Aug;15(8):1504-11. Epub 2007 Jun 12.


Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: implications for preclinical gene therapy trials.

Minella AL, Mowat FM, Willett KL, Sledge D, Bartoe JT, Bennett J, Petersen-Jones SM.

Gene Ther. 2014 Oct;21(10):913-20. doi: 10.1038/gt.2014.65. Epub 2014 Jul 24.


Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping.

Ni W, Le Guiner C, Gernoux G, Penaud-Budloo M, Moullier P, Snyder RO.

Gene Ther. 2011 Jul;18(7):709-18. doi: 10.1038/gt.2011.19. Epub 2011 Mar 10.


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