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Connection

Terence Flotte to alpha 1-Antitrypsin

This is a "connection" page, showing publications Terence Flotte has written about alpha 1-Antitrypsin.
Connection Strength

8.549
  1. Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther. 2018 08; 29(8):843-844.
    View in: PubMed
    Score: 0.568
  2. Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR. 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. Mol Ther. 2017 06 07; 25(6):1387-1394.
    View in: PubMed
    Score: 0.518
  3. Tang Q, Gruntman AM, Flotte TR. Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA. Methods Mol Biol. 2017; 1639:211-216.
    View in: PubMed
    Score: 0.509
  4. Loring HS, Flotte TR. Current status of gene therapy for a-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36.
    View in: PubMed
    Score: 0.438
  5. 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. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest. 2013 Dec; 123(12):5310-8.
    View in: PubMed
    Score: 0.409
  6. Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9.
    View in: PubMed
    Score: 0.390
  7. Mueller C, Tang Q, Gruntman A, Blomenkamp K, Teckman J, Song L, Zamore PD, Flotte TR. Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. Mol Ther. 2012 03; 20(3):590-600.
    View in: PubMed
    Score: 0.361
  8. 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. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing a1-antitrypsin: interim results. Hum Gene Ther. 2011 Oct; 22(10):1239-47.
    View in: PubMed
    Score: 0.351
  9. Chulay JD, Ye GJ, Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther. 2011 Feb; 22(2):155-65.
    View in: PubMed
    Score: 0.334
  10. 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. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A. 2009 Sep 22; 106(38):16363-8.
    View in: PubMed
    Score: 0.305
  11. Liqun Wang R, McLaughlin T, Cossette T, Tang Q, Foust K, Campbell-Thompson M, Martino A, Cruz P, Loiler S, Mueller C, Flotte TR. Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery. Mol Ther. 2009 Jan; 17(1):81-7.
    View in: PubMed
    Score: 0.288
  12. Flotte TR, Goetzmann J, Caridi J, Paolillo J, Conlon TJ, Potter M, Mueller C, Byrne BJ. Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. Hum Gene Ther. 2008 Jul; 19(7):681-9.
    View in: PubMed
    Score: 0.282
  13. Cruz PE, Mueller C, Cossette TL, Golant A, Tang Q, Beattie SG, Brantly M, Campbell-Thompson M, Blomenkamp KS, Teckman JH, Flotte TR. In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest. 2007 Sep; 87(9):893-902.
    View in: PubMed
    Score: 0.263
  14. Flotte TR, Conlon TJ, Poirier A, Campbell-Thompson M, Byrne BJ. Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum Gene Ther. 2007 Mar; 18(3):245-56.
    View in: PubMed
    Score: 0.257
  15. 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. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther. 2006 Dec; 17(12):1177-86.
    View in: PubMed
    Score: 0.253
  16. Conlon TJ, Cossette T, Erger K, Choi YK, Clarke T, Scott-Jorgensen M, Song S, Campbell-Thompson M, Crawford J, Flotte TR. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther. 2005 Nov; 12(5):867-75.
    View in: PubMed
    Score: 0.231
  17. Pires-Ferreira D, Reil D, Tang Q, Blackwood M, Gallagher T, Keeler AM, Chichester JA, Vyhnal KK, Lindborg JA, Benson J, Fu D, Flotte TR, Gruntman AM. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy. Genes (Basel). 2024 Sep 10; 15(9).
    View in: PubMed
    Score: 0.217
  18. Song S, Goudy K, Campbell-Thompson M, Wasserfall C, Scott-Jorgensen M, Wang J, Tang Q, Crawford JM, Ellis TM, Atkinson MA, Flotte TR. Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice. Gene Ther. 2004 Jan; 11(2):181-6.
    View in: PubMed
    Score: 0.207
  19. Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ, Humphries M. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther. 2004 Jan; 15(1):93-128.
    View in: PubMed
    Score: 0.207
  20. Pires Ferreira D, Gruntman AM, Flotte TR. Gene therapy for alpha-1 antitrypsin deficiency: an update. Expert Opin Biol Ther. 2023 03; 23(3):283-291.
    View in: PubMed
    Score: 0.195
  21. Song S, Scott-Jorgensen M, Wang J, Poirier A, Crawford J, Campbell-Thompson M, Flotte TR. Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. Mol Ther. 2002 Sep; 6(3):329-35.
    View in: PubMed
    Score: 0.188
  22. Ke H, Guay KP, Flotte TR, Gierasch LM, Gershenson A, Hebert DN. Secretion of functional a1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy. Proc Natl Acad Sci U S A. 2022 08 02; 119(31):e2206103119.
    View in: PubMed
    Score: 0.187
  23. Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2001 Sep; 8(17):1299-306.
    View in: PubMed
    Score: 0.176
  24. Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998 Nov 24; 95(24):14384-8.
    View in: PubMed
    Score: 0.145
  25. Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther. 2018 08; 29(8):853-860.
    View in: PubMed
    Score: 0.140
  26. Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C. Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema. Proc Natl Acad Sci U S A. 2018 03 13; 115(11):2788-2793.
    View in: PubMed
    Score: 0.137
  27. Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of a-1 Antitrypsin Deficiency. Mol Ther. 2017 11 01; 25(11):2477-2489.
    View in: PubMed
    Score: 0.134
  28. Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, Vonderheide RH, Mueller C, Flotte TR, Wilson JM. Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for a-1-antitrypsin deficiency. Proc Natl Acad Sci U S A. 2017 02 14; 114(7):1655-1659.
    View in: PubMed
    Score: 0.128
  29. Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
    View in: PubMed
    Score: 0.127
  30. Bouchecareilh M, Hutt DM, Szajner P, Flotte TR, Balch WE. Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of a1-antitrypsin deficiency. J Biol Chem. 2012 Nov 02; 287(45):38265-78.
    View in: PubMed
    Score: 0.095
  31. Petrache I, Fijalkowska I, Medler TR, Skirball J, Cruz P, Zhen L, Petrache HI, Flotte TR, Tuder RM. alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis. Am J Pathol. 2006 Oct; 169(4):1155-66.
    View in: PubMed
    Score: 0.062
  32. Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, Flotte TR, Song S. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med. 2006 Jun; 8(6):730-5.
    View in: PubMed
    Score: 0.061
  33. Petrache I, Fijalkowska I, Zhen L, Medler TR, Brown E, Cruz P, Choe KH, Taraseviciene-Stewart L, Scerbavicius R, Shapiro L, Zhang B, Song S, Hicklin D, Voelkel NF, Flotte T, Tuder RM. A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. Am J Respir Crit Care Med. 2006 Jun 01; 173(11):1222-8.
    View in: PubMed
    Score: 0.060
  34. Loiler SA, Tang Q, Clarke T, Campbell-Thompson ML, Chiodo V, Hauswirth W, Cruz P, Perret-Gentil M, Atkinson MA, Ramiya VK, Flotte TR. Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Mol Ther. 2005 Sep; 12(3):519-27.
    View in: PubMed
    Score: 0.058
  35. Virella-Lowell I, Zusman B, Foust K, Loiler S, Conlon T, Song S, Chesnut KA, Ferkol T, Flotte TR. Enhancing rAAV vector expression in the lung. J Gene Med. 2005 Jul; 7(7):842-50.
    View in: PubMed
    Score: 0.057
  36. Barbour KW, Wei F, Brannan C, Flotte TR, Baumann H, Berger FG. The murine alpha(1)-proteinase inhibitor gene family: polymorphism, chromosomal location, and structure. Genomics. 2002 Nov; 80(5):515-22.
    View in: PubMed
    Score: 0.048
  37. Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S.
    View in: PubMed
    Score: 0.045
  38. Virella-Lowell I, Poirier A, Chesnut KA, Brantly M, Flotte TR. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther. 2000 Oct; 7(20):1783-9.
    View in: PubMed
    Score: 0.041
  39. Tang Q, Gernoux G, Cheng Y, Flotte T, Mueller C. Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. Methods Mol Biol. 2020; 2164:75-85.
    View in: PubMed
    Score: 0.039
  40. Chen S, Kapturczak M, Loiler SA, Zolotukhin S, Glushakova OY, Madsen KM, Samulski RJ, Hauswirth WW, Campbell-Thompson M, Berns KI, Flotte TR, Atkinson MA, Tisher CC, Agarwal A. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther. 2005 Feb; 16(2):235-47.
    View in: PubMed
    Score: 0.014
  41. Song S, Witek RP, Lu Y, Choi YK, Zheng D, Jorgensen M, Li C, Flotte TR, Petersen BE. Ex vivo transduced liver progenitor cells as a platform for gene therapy in mice. Hepatology. 2004 Oct; 40(4):918-24.
    View in: PubMed
    Score: 0.014
  42. Mah C, Fraites TJ, Zolotukhin I, Song S, Flotte TR, Dobson J, Batich C, Byrne BJ. Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther. 2002 Jul; 6(1):106-12.
    View in: PubMed
    Score: 0.012
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