Terence Flotte to alpha 1-Antitrypsin Deficiency
This is a "connection" page, showing publications Terence Flotte has written about alpha 1-Antitrypsin Deficiency.
Connection Strength
9.796
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Gruntman AM, Xue W, Flotte TR. Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:1-7.
Score: 0.787
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Gruntman AM, Xue W, Flotte TR. Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:11-17.
Score: 0.787
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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.
Score: 0.743
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Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther. 2018 08; 29(8):843-844.
Score: 0.541
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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.
Score: 0.494
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Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
Score: 0.485
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Flotte TR. The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy. Hum Gene Ther. 2015 Dec; 26(12):782.
Score: 0.449
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Gruntman AM, Flotte TR. Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency. Hum Gene Ther Methods. 2015 Jun; 26(3):77-81.
Score: 0.434
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Loring HS, Flotte TR. Current status of gene therapy for a-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36.
Score: 0.417
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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.
Score: 0.390
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Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9.
Score: 0.371
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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.
Score: 0.334
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Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 15; 20(R1):R87-92.
Score: 0.326
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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.
Score: 0.318
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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.
Score: 0.290
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Cruz PE, Mueller C, Flotte TR. The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency. Pharmacogenomics. 2007 Sep; 8(9):1191-8.
Score: 0.254
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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.
Score: 0.250
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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.
Score: 0.241
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Bamidele N, Pires-Ferreira D, Zheng C, Destefano A, Cheng H, Tang Q, Cao Y, Xie J, Gao G, Gruntman A, Sontheimer E, Flotte TR, Xue W. A compact base editor rescues AATD-associated liver and lung disease in mouse models. Mol Ther. 2025 Nov 05; 33(11):5817-5828.
Score: 0.221
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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.
Score: 0.220
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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).
Score: 0.206
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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.
Score: 0.197
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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.
Score: 0.178
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Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S.
Score: 0.173
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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.
Score: 0.133
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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.
Score: 0.127
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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.
Score: 0.122
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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.
Score: 0.090
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Lockett AD, Van Demark M, Gu Y, Schweitzer KS, Sigua N, Kamocki K, Fijalkowska I, Garrison J, Fisher AJ, Serban K, Wise RA, Flotte TR, Mueller C, Presson RG, Petrache HI, Tuder RM, Petrache I. Effect of cigarette smoke exposure and structural modifications on the a-1 Antitrypsin interaction with caspases. Mol Med. 2012 May 09; 18:445-54.
Score: 0.088
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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.
Score: 0.061
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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.
Score: 0.052
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Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Yl?-Herttuala S. Progress and prospects: gene therapy clinical trials (part 1). Gene Ther. 2007 Oct; 14(20):1439-47.
Score: 0.016