Research Topics
| Christian MuellerSummaryAffiliation: University of Massachusetts Medical School Country: USA Publications
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Detail Information
Publications
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profilesChristian Mueller
Department of Pediatrics and Gene Therapy Center, UMass Medical School, Worcester, Massachusetts 01605, USA
Mol Ther 20:590-600. 2012..This safe dual-therapy approach can be applied to other disorders such as amyotrophic lateral sclerosis, Huntington disease, cerebral ataxia, and optic atrophies...- Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responsesChristian Mueller
University of Massachusetts Medical School Department of Pediatrics and Gene Therapy Center, Worcester, Massachusetts 01605, USA
Am J Respir Cell Mol Biol 44:922-9. 2011..In summary, our data identified that CFTR dysfunction in T cells can lead directly to aberrant immune responses. These findings implicate the lymphocyte population as a potentially important target for CF therapeutics... - Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17eChristian Mueller
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA
Mol Ther 18:511-8. 2010..These studies demonstrate the feasibility of targeting T helper 2 (Th2) cytokines with rAAV-delivered fusion proteins as a means to treat aberrant immune responses... - In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectorsC Mueller
Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01605, USA
Gene Ther 17:227-37. 2010..The reduction in inflammatory cytokine levels led to an evident decrease in neutrophil influx to the airways. These results indicate that further study of the application of rSV40-CFTR to CF gene therapy is warranted... - Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous systemHongwei Zhang
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Mol Ther 19:1440-8. 2011.... - Gene transfer in the lung using recombinant adeno-associated virusAlisha M Gruntman
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Curr Protoc Microbiol . 2012..Detailed procedures for lung delivery (intranasal, orotracheal, and surgical tracheal injection), sample collection, and post-mortem tissue processing will be described... - Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airwayTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
Mol Ther 18:594-600. 2010..T-cell responses to AAV5 capsid were stronger than AAV1 capsid. This dual vector indexing approach may be useful in selecting lead vector serotypes for clinical gene therapy and suggests rAAV1 is preferred for cystic fibrosis... - MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expressionJun Xie
Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
Mol Ther 19:526-35. 2011..Our findings promise to facilitate the development of miRNA-regulated rAAV for CNS-targeted gene delivery and other applications... - Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzeesTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA
Hum Gene Ther 19:681-9. 2008..These data indicate an increased susceptibility to subclinical liver toxicity from portal vein injection of rAAV2 in the presence of HCV infection... - Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapyAllison M Keeler
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Mol Ther 20:1131-8. 2012..These promising results suggest rAAV9 gene therapy as a potential treatment for VLCAD deficiency in humans... 
Preclinical study design for rAAVTerence R Flotte
Department of Pediatrics, University of Massachusetts Medical School Gene Therapy Center, Worcester, MA, USA
Methods Mol Biol 807:317-37. 2011....- Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim resultsTerence R Flotte
University of Massachusetts Medical School, 55 Lake Avenue North, Worcester, MA 01655, USA
Hum Gene Ther 22:1239-47. 2011..However, further improvements in the design or delivery of rAAV-AAT vectors will be required to achieve therapeutic target serum AAT concentrations... - CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetesMichael S Stalvey
Department of Pediatrics, University of Florida, College of Medicine, P O Box 100296, Gainesville, FL 32610, USA
Cytokine 44:154-9. 2008..These results suggest that, hyperglycemia may exacerbate the clinical course in CF by impacting immune reactivity. There is clear need to maximize metabolic management in CFRD... - Gene therapy for cystic fibrosisChristian Mueller
Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA, USA
Clin Rev Allergy Immunol 35:164-78. 2008..This learning curve has led to the optimization of vector technology and an appreciation of immune and mechanical barriers that have to be overcome for successful delivery... 
In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNAPedro E Cruz
Department of Pediatrics, University of Florida, Gainesville, FL, USA
Lab Invest 87:893-902. 2007..The rAAV8-3X-siRNA vector may hold promise as a potential therapy for patients with AAT liver disease...- N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearanceAshley T Martino
Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida, USA
Am J Respir Cell Mol Biol 44:824-30. 2011..Augmenting N-glycosylation to attenuate colonization of P. aeruginosa in CF airways reveals a new therapeutic avenue for a hallmark disease condition in CF... - Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virusAlisha M Gruntman
Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts, USA
Curr Protoc Microbiol . 2013.... - Production and discovery of novel recombinant adeno-associated viral vectorsChristian Mueller
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
Curr Protoc Microbiol . 2012.... - Immune responses in cystic fibrosis: are they intrinsically defective?Dmitry Ratner
Pediatrics and Gene Therapy Center, University of Massachusetts Medical School, 381 Plantation Street, Worcester MA 01605, USA
Am J Respir Cell Mol Biol 46:715-22. 2012..This review focuses on the emerging role of Cftr in gene expression and other functions in cells of the innate and adaptive immune system... - Gene therapy for alpha-1 antitrypsin deficiencyTerence R Flotte
Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, S1 340, 55 Lake Avenue North, Worcester, MA 01655, USA
Hum Mol Genet 20:R87-92. 2011..Over the years, numerous strategies have been employed for the gene therapy of both AAT-deficient lung disease and liver disease. These will be reviewed with an emphasis on modalities that have reached clinical trials recently... - Gene-based therapy for alpha-1 antitrypsin deficiencyChristian Mueller
1 Gene Therapy Center and the Departments of Pediatrics and Microbiology and Physiologic Systems, University of Massachusetts Medical School, Worcester, Massachusetts, USA
COPD 10:44-9. 2013..This indicates the need to further increase the dose of the vector and/or to increase the levels to within the therapeutic range... - Hepatocyte-specific hypoxia-inducible factor-1α is a determinant of lipid accumulation and liver injury in alcohol-induced steatosis in miceBharath Nath
Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA
Hepatology 53:1526-37. 2011..The alcohol-induced chemokine MCP-1 triggers lipid accumulation in hepatocytes via HIF-1α activation, suggesting a mechanistic link between inflammation and hepatic steatosis in alcoholic liver disease...