Christian Mueller
Christian Mueller
Associate Professor of Pediatrics, University of Massachusetts Medical School
Verified email at umassmed.edu - Homepage
Title
Cited by
Cited by
Year
Clinical gene therapy using recombinant adeno-associated virus vectors
C Mueller, TR Flotte
Gene therapy 15 (11), 858-863, 2008
3692008
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
3072009
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
2792011
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
2242011
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
1862015
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
1562011
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
1342011
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
114*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
1122013
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
992012
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
892014
Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector
J Sirninger, C Muller, S Braag, Q Tang, H Yue, C Detrisac, T Ferkol, ...
Human gene therapy 15 (9), 832-841, 2004
792004
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
762012
In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA
PE Cruz, C Mueller, TL Cossette, A Golant, Q Tang, SG Beattie, M Brantly, ...
Laboratory investigation 87 (9), 893-902, 2007
742007
Gene therapy for cystic fibrosis
C Mueller, TR Flotte
Clinical reviews in allergy & immunology 35 (3), 164-178, 2008
602008
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
592015
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
572016
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
572016
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway
TR Flotte, AC Fischer, J Goetzmann, C Mueller, L Cebotaru, Z Yan, ...
Molecular Therapy 18 (3), 594-600, 2010
572010
Gene therapy for alpha-1 antitrypsin deficiency
TR Flotte, C Mueller
Human molecular genetics 20 (R1), R87-R92, 2011
512011
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