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Results: 1 to 20 of 103

1.

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.

Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR.

Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16363-8. doi: 10.1073/pnas.0904514106. Epub 2009 Aug 12. Erratum in: Proc Natl Acad Sci U S A. 2009 Oct 13;106(41):17606.

PMID:
19706466
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.

Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, Campbell-Thompson M, Yachnis AT, Sandhaus RA, McElvaney NG, Mueller C, Messina LM, Wilson JM, Brantly M, Knop DR, Ye GJ, Chulay JD.

Hum Gene Ther. 2011 Oct;22(10):1239-47. doi: 10.1089/hum.2011.053. Epub 2011 Aug 24.

PMID:
21609134
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.

Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR.

Hum Gene Ther. 2006 Dec;17(12):1177-86.

PMID:
17115945
[PubMed - indexed for MEDLINE]
4.

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.

Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR.

J Clin Invest. 2013 Dec 2;123(12):5310-8. doi: 10.1172/JCI70314. Epub 2013 Nov 15.

PMID:
24231351
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells.

Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, Edmonson SA, Hutnick NA, Betts MR, Kastelein JJ, Stroes ES, High KA.

Blood. 2009 Sep 3;114(10):2077-86. doi: 10.1182/blood-2008-07-167510. Epub 2009 Jun 8.

PMID:
19506302
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method.

Chulay JD, Ye GJ, Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR.

Hum Gene Ther. 2011 Feb;22(2):155-65. doi: 10.1089/hum.2010.118. Epub 2010 Dec 12.

PMID:
20812844
[PubMed - indexed for MEDLINE]
Free PMC Article
7.
8.

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults.

Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ, Humphries M.

Hum Gene Ther. 2004 Jan;15(1):93-128.

PMID:
14965381
[PubMed - indexed for MEDLINE]
9.

Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors.

Lu Y, Song S.

Proc Natl Acad Sci U S A. 2009 Oct 6;106(40):17158-62. doi: 10.1073/pnas.0909520106. Epub 2009 Sep 22.

PMID:
19805176
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector.

Conlon TJ, Cossette T, Erger K, Choi YK, Clarke T, Scott-Jorgensen M, Song S, Campbell-Thompson M, Crawford J, Flotte TR.

Mol Ther. 2005 Nov;12(5):867-75. Epub 2005 Aug 8.

PMID:
16085464
[PubMed - indexed for MEDLINE]
11.

Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery.

Liqun Wang R, McLaughlin T, Cossette T, Tang Q, Foust K, Campbell-Thompson M, Martino A, Cruz P, Loiler S, Mueller C, Flotte TR.

Mol Ther. 2009 Jan;17(1):81-7. doi: 10.1038/mt.2008.217. Epub 2008 Oct 21.

PMID:
18941444
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine.

McIntosh JH, Cochrane M, Cobbold S, Waldmann H, Nathwani SA, Davidoff AM, Nathwani AC.

Gene Ther. 2012 Jan;19(1):78-85. doi: 10.1038/gt.2011.64. Epub 2011 Jun 30.

PMID:
21716299
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

Halbert CL, Madtes DK, Vaughan AE, Wang Z, Storb R, Tapscott SJ, Miller AD.

Mol Ther. 2010 Jun;18(6):1165-72. doi: 10.1038/mt.2010.51. Epub 2010 Apr 6.

PMID:
20372105
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Gene-based therapy for alpha-1 antitrypsin deficiency.

Mueller C, Flotte TR.

COPD. 2013 Mar;10 Suppl 1:44-9. doi: 10.3109/15412555.2013.764978. Review.

PMID:
23527792
[PubMed - indexed for MEDLINE]
15.

Adipose tissue-derived mesenchymal stem cell-based liver gene delivery.

Li H, Zhang B, Lu Y, Jorgensen M, Petersen B, Song S.

J Hepatol. 2011 May;54(5):930-8. doi: 10.1016/j.jhep.2010.07.051. Epub 2010 Nov 3.

PMID:
21168381
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.

Cruz PE, Mueller C, Cossette TL, Golant A, Tang Q, Beattie SG, Brantly M, Campbell-Thompson M, Blomenkamp KS, Teckman JH, Flotte TR.

Lab Invest. 2007 Sep;87(9):893-902. Epub 2007 Jun 25.

PMID:
17592477
[PubMed - indexed for MEDLINE]
Free Article
17.

Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes.

Li H, Murphy SL, Giles-Davis W, Edmonson S, Xiang Z, Li Y, Lasaro MO, High KA, Ertl HC.

Mol Ther. 2007 Apr;15(4):792-800. Epub 2007 Jan 23.

PMID:
17245353
[PubMed - indexed for MEDLINE]
18.

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors.

Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR.

Gene Ther. 2001 Sep;8(17):1299-306.

PMID:
11571566
[PubMed - indexed for MEDLINE]
Free Article
19.

Enhanced transduction of mouse bone marrow-derived dendritic cells by repetitive infection with self-complementary adeno-associated virus 6 combined with immunostimulatory ligands.

Aldrich WA, Ren C, White AF, Zhou SZ, Kumar S, Jenkins CB, Shaw DR, Strong TV, Triozzi PL, Ponnazhagan S.

Gene Ther. 2006 Jan;13(1):29-39.

PMID:
16136165
[PubMed - indexed for MEDLINE]
20.

Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector.

Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, Flotte TR, Song S.

J Gene Med. 2006 Jun;8(6):730-5.

PMID:
16518879
[PubMed - indexed for MEDLINE]

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