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Items: 1 to 20 of 126


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.


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.


Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase.

Conlon TJ, Erger K, Porvasnik S, Cossette T, Roberts C, Combee L, Islam S, Kelley J, Cloutier D, Clément N, Abernathy CR, Byrne BJ.

Hum Gene Ther Clin Dev. 2013 Sep;24(3):127-33. doi: 10.1089/humc.2013.147.


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.


Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males.

Arruda VR, Fields PA, Milner R, Wainwright L, De Miguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel JA, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, High KA.

Mol Ther. 2001 Dec;4(6):586-92.


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.


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.


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.


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.


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;123(12):5310-8. doi: 10.1172/JCI70314. Epub 2013 Nov 15.


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.


Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.

Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.

Mol Ther. 2008 Jul;16(7):1291-9. doi: 10.1038/mt.2008.87. Epub 2008 May 6.


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.


Preclinical safety evaluation of a recombinant AAV8 vector for X-linked retinoschisis after intravitreal administration in rabbits.

Marangoni D, Wu Z, Wiley HE, Zeiss CJ, Vijayasarathy C, Zeng Y, Hiriyanna S, Bush RA, Wei LL, Colosi P, Sieving PA.

Hum Gene Ther Clin Dev. 2014 Dec;25(4):202-11. doi: 10.1089/humc.2014.067.


Intrapleural administration of an AAVrh.10 vector coding for human α1-antitrypsin for the treatment of α1-antitrypsin deficiency.

Chiuchiolo MJ, Kaminsky SM, Sondhi D, Hackett NR, Rosenberg JB, Frenk EZ, Hwang Y, Van de Graaf BG, Hutt JA, Wang G, Benson J, Crystal RG.

Hum Gene Ther Clin Dev. 2013 Dec;24(4):161-73. doi: 10.1089/humc.2013.168.


Ovine adenovirus vectors mediate efficient gene transfer to skeletal muscle.

Löser P, Hillgenberg M, Arnold W, Both GW, Hofmann C.

Gene Ther. 2000 Sep;7(17):1491-8.


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.


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