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Results: 1 to 20 of 100

1.

The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency.

Cruz PE, Mueller C, Flotte TR.

Pharmacogenomics. 2007 Sep;8(9):1191-8. Review.

PMID:
17924834
[PubMed - indexed for MEDLINE]
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.

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]
4.

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]
5.

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
6.

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
7.

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.

PMID:
12524461
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

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]
9.

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]
10.

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
11.

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
12.

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
13.

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]
14.

Gene therapy for alpha-1 antitrypsin deficiency.

Flotte TR, Mueller C.

Hum Mol Genet. 2011 Apr 15;20(R1):R87-92. doi: 10.1093/hmg/ddr156. Epub 2011 Apr 16. Review.

PMID:
21498872
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Gene therapy prospects--intranasal delivery of therapeutic genes.

Podolska K, Stachurska A, Hajdukiewicz K, Małecki M.

Adv Clin Exp Med. 2012 Jul-Aug;21(4):525-34. Review.

PMID:
23240459
[PubMed - indexed for MEDLINE]
Free Article
16.
17.

Alpha-1-antitrypsin expression in the lung is increased by airway delivery of gene-transfected macrophages.

Zhang D, Wu M, Nelson DE, Pasula R, Martin WJ 2nd.

Gene Ther. 2003 Dec;10(26):2148-52.

PMID:
14625570
[PubMed - indexed for MEDLINE]
18.

A review of augmentation therapy for alpha-1 antitrypsin deficiency.

Mohanka M, Khemasuwan D, Stoller JK.

Expert Opin Biol Ther. 2012 Jun;12(6):685-700. doi: 10.1517/14712598.2012.676638. Epub 2012 Apr 14. Review.

PMID:
22500781
[PubMed - indexed for MEDLINE]
19.

Gene therapy for alpha 1-antitrypsin deficiency.

Geddes DM, Beckles MA.

Monaldi Arch Chest Dis. 1995 Oct;50(5):388-93.

PMID:
8541824
[PubMed - indexed for MEDLINE]
20.

Alpha1-antitrypsin deficiency: a report from the 2nd meeting of the Alpha One International Registry, Rapallo (Genoa, Italy), 2001.

Luisetti M, Miravitlles M, Stockley RA.

Eur Respir J. 2002 Oct;20(4):1050-6.

PMID:
12412702
[PubMed - indexed for MEDLINE]
Free Article

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