| Clinical gene therapy using recombinant adeno-associated virus vectors C Mueller, TR Flotte Gene therapy 15 (11), 858-863, 2008 | 411 | 2008 |
| Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results TR Flotte, BC Trapnell, M Humphries, B Carey, R Calcedo, F Rouhani, ... Human gene therapy 22 (10), 1239-1247, 2011 | 391 | 2011 |
| Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy ML Brantly, JD Chulay, L Wang, C Mueller, M Humphries, LT Spencer, ... Proceedings of the National Academy of Sciences 106 (38), 16363-16368, 2009 | 383 | 2009 |
| Several rAAV vectors efficiently cross the blood–brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system H Zhang, B Yang, X Mu, SS Ahmed, Q Su, R He, H Wang, C Mueller, ... Molecular Therapy 19 (8), 1440-1448, 2011 | 336 | 2011 |
| Human C9ORF72 hexanucleotide expansion reproduces RNA foci and dipeptide repeat proteins but not neurodegeneration in BAC transgenic mice OM Peters, GT Cabrera, H Tran, TF Gendron, JE McKeon, J Metterville, ... Neuron 88 (5), 902-909, 2015 | 289 | 2015 |
| Hepatocyte‐specific hypoxia‐inducible factor‐1α is a determinant of lipid accumulation and liver injury in alcohol‐induced steatosis in mice B Nath, I Levin, T Csak, J Petrasek, C Mueller, K Kodys, D Catalano, ... Hepatology 53 (5), 1526-1537, 2011 | 216 | 2011 |
| MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression J Xie, Q Xie, H Zhang, SL Ameres, JH Hung, Q Su, R He, X Mu, ... Molecular therapy 19 (3), 526-535, 2011 | 198 | 2011 |
| SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS C Mueller, JD Berry, DM McKenna-Yasek, G Gernoux, MA Owegi, ... New England Journal of Medicine 383 (2), 151-158, 2020 | 196 | 2020 |
| Recombinant AAV as a platform for translating the therapeutic potential of RNA interference F Borel, MA Kay, C Mueller Molecular Therapy 22 (4), 692-701, 2014 | 167 | 2014 |
| A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system JF Alterman, BMDC Godinho, MR Hassler, CM Ferguson, D Echeverria, ... Nature biotechnology 37 (8), 884-894, 2019 | 166 | 2019 |
| Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses C Mueller, SA Braag, A Keeler, C Hodges, M Drumm, TR Flotte American journal of respiratory cell and molecular biology 44 (6), 922-929, 2011 | 163 | 2011 |
| Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression C Mueller, JD Chulay, BC Trapnell, M Humphries, B Carey, RA Sandhaus, ... The Journal of clinical investigation 123 (12), 5310-5318, 2013 | 154 | 2013 |
| Lack of acute xenogeneic graft-versus-host disease, but retention of T-cell function following engraftment of human peripheral blood mononuclear cells in NSG mice deficient in … MA Brehm, LL Kenney, MV Wiles, BE Low, RM Tisch, L Burzenski, ... The FASEB Journal 33 (3), 3137, 2019 | 133 | 2019 |
| Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles C Mueller, Q Tang, A Gruntman, K Blomenkamp, J Teckman, L Song, ... Molecular Therapy 20 (3), 590-600, 2012 | 130 | 2012 |
| Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1G93A Mice and Nonhuman Primates F Borel, G Gernoux, B Cardozo, JP Metterville, G T. Cabrera, L Song, ... Human gene therapy 27 (1), 19-31, 2016 | 122 | 2016 |
| Immune responses in cystic fibrosis: are they intrinsically defective? D Ratner, C Mueller American journal of respiratory cell and molecular biology 46 (6), 715-722, 2012 | 121 | 2012 |
| Precise therapeutic gene correction by a simple nuclease-induced double-stranded break S Iyer, S Suresh, D Guo, K Daman, JCJ Chen, P Liu, M Zieger, K Luk, ... Nature 568 (7753), 561-565, 2019 | 113 | 2019 |
| Adeno‐associated virus–delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model L Stoica, SH Todeasa, GT Cabrera, JS Salameh, MK ElMallah, C Mueller, ... Annals of neurology 79 (4), 687-700, 2016 | 110 | 2016 |
| Efficient and targeted transduction of nonhuman primate liver with systemically delivered optimized AAV3B vectors S Li, C Ling, L Zhong, M Li, Q Su, R He, Q Tang, DL Greiner, LD Shultz, ... Molecular Therapy 23 (12), 1867-1876, 2015 | 110 | 2015 |
| Artificial miRNAs reduce human mutant Huntingtin throughout the striatum in a transgenic sheep model of Huntington's disease EL Pfister, N DiNardo, E Mondo, F Borel, F Conroy, C Fraser, G Gernoux, ... Human gene therapy 29 (6), 663-673, 2018 | 100 | 2018 |
Terence R FlotteProfessor, Dean, Provost, University of Massachusetts Medical School在 umassmed.edu 的电子邮件经过验证
