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Results: 1 to 20 of 146

1.

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]
2.

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]
3.

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
4.

Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice.

Song S, Goudy K, Campbell-Thompson M, Wasserfall C, Scott-Jorgensen M, Wang J, Tang Q, Crawford JM, Ellis TM, Atkinson MA, Flotte TR.

Gene Ther. 2004 Jan;11(2):181-6.

PMID:
14712302
[PubMed - indexed for MEDLINE]
5.

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
6.

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]
7.
8.

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]
9.

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
10.

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
11.

Enhancing rAAV vector expression in the lung.

Virella-Lowell I, Zusman B, Foust K, Loiler S, Conlon T, Song S, Chesnut KA, Ferkol T, Flotte TR.

J Gene Med. 2005 Jul;7(7):842-50.

PMID:
15838934
[PubMed - indexed for MEDLINE]
12.

Alpha1-antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic mice.

Lu Y, Tang M, Wasserfall C, Kou Z, Campbell-Thompson M, Gardemann T, Crawford J, Atkinson M, Song S.

Hum Gene Ther. 2006 Jun;17(6):625-34.

PMID:
16776571
[PubMed - indexed for MEDLINE]
13.

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects.

Song S, Scott-Jorgensen M, Wang J, Poirier A, Crawford J, Campbell-Thompson M, Flotte TR.

Mol Ther. 2002 Sep;6(3):329-35.

PMID:
12231168
[PubMed - indexed for MEDLINE]
14.

Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice.

Ishiwata A, Mimuro J, Mizukami H, Kashiwakura Y, Takano K, Ohmori T, Madoiwa S, Ozawa K, Sakata Y.

J Gene Med. 2009 Nov;11(11):1020-9. doi: 10.1002/jgm.1391.

PMID:
19757487
[PubMed - indexed for MEDLINE]
15.

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
16.

Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo.

Okuyama T, Huber RM, Bowling W, Pearline R, Kennedy SC, Flye MW, Ponder KP.

Hum Gene Ther. 1996 Mar 20;7(5):637-45.

PMID:
8845389
[PubMed - indexed for MEDLINE]
17.

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors.

Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR.

Proc Natl Acad Sci U S A. 1998 Nov 24;95(24):14384-8.

PMID:
9826709
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin.

De B, Heguy A, Leopold PL, Wasif N, Korst RJ, Hackett NR, Crystal RG.

Mol Ther. 2004 Dec;10(6):1003-10.

PMID:
15564132
[PubMed - indexed for MEDLINE]
19.

An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes.

Kang W, Wang L, Harrell H, Liu J, Thomas DL, Mayfield TL, Scotti MM, Ye GJ, Veres G, Knop DR.

Gene Ther. 2009 Feb;16(2):229-39. doi: 10.1038/gt.2008.158. Epub 2008 Oct 16.

PMID:
18923452
[PubMed - indexed for MEDLINE]
20.

Gene therapy progress and prospects: alpha-1 antitrypsin.

Stecenko AA, Brigham KL.

Gene Ther. 2003 Jan;10(2):95-9. Review.

PMID:
12571637
[PubMed - indexed for MEDLINE]

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