Christian Mueller

Summary

Affiliation: University of Massachusetts Medical School
Country: USA

Publications

  1. pmc Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles
    Christian Mueller
    Department of Pediatrics and Gene Therapy Center, UMass Medical School, Worcester, Massachusetts 01605, USA
    Mol Ther 20:590-600. 2012
  2. pmc Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses
    Christian 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
  3. pmc Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e
    Christian Mueller
    Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA
    Mol Ther 18:511-8. 2010
  4. pmc In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors
    C Mueller
    Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01605, USA
    Gene Ther 17:227-37. 2010
  5. pmc Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    Hongwei Zhang
    Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA
    Mol Ther 19:1440-8. 2011
  6. pmc Gene transfer in the lung using recombinant adeno-associated virus
    Alisha M Gruntman
    Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
    Curr Protoc Microbiol . 2012
  7. pmc Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway
    Terence R Flotte
    Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
    Mol Ther 18:594-600. 2010
  8. pmc Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus
    Alisha M Gruntman
    Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts, USA
    Curr Protoc Microbiol . 2013
  9. pmc MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression
    Jun Xie
    Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA
    Mol Ther 19:526-35. 2011
  10. pmc Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees
    Terence R Flotte
    Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA
    Hum Gene Ther 19:681-9. 2008

Collaborators

Detail Information

Publications22

  1. pmc Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles
    Christian 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...
  2. pmc Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses
    Christian 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...
  3. pmc Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e
    Christian 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...
  4. pmc In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors
    C 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...
  5. pmc Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    Hongwei Zhang
    Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA
    Mol Ther 19:1440-8. 2011
    ....
  6. pmc Gene transfer in the lung using recombinant adeno-associated virus
    Alisha 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...
  7. pmc Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway
    Terence 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...
  8. pmc Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus
    Alisha M Gruntman
    Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts, USA
    Curr Protoc Microbiol . 2013
    ....
  9. pmc MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression
    Jun 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...
  10. pmc Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees
    Terence 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...
  11. pmc Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy
    Allison 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...
  12. doi request reprint Preclinical study design for rAAV
    Terence R Flotte
    Department of Pediatrics, University of Massachusetts Medical School Gene Therapy Center, Worcester, MA, USA
    Methods Mol Biol 807:317-37. 2011
    ....
  13. pmc Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results
    Terence 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...
  14. doi request reprint CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes
    Michael 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...
  15. ncbi request reprint Gene therapy for cystic fibrosis
    Christian 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...
  16. ncbi request reprint In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA
    Pedro 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...
  17. pmc N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance
    Ashley 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...
  18. doi request reprint Gene-based therapy for alpha-1 antitrypsin deficiency
    Christian Mueller
    Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, 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...
  19. doi request reprint Production and discovery of novel recombinant adeno-associated viral vectors
    Christian Mueller
    Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA
    Curr Protoc Microbiol . 2012
    ....
  20. doi request reprint 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...
  21. pmc Gene therapy for alpha-1 antitrypsin deficiency
    Terence 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...
  22. pmc Hepatocyte-specific hypoxia-inducible factor-1α is a determinant of lipid accumulation and liver injury in alcohol-induced steatosis in mice
    Bharath Nath
    Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA
    Hepatology 53:1526-37. 2011
    ..Small interfering RNA inhibition of HIF-1α prevented MCP-1-induced lipid accumulation, suggesting a mechanistic role for HIF-1α in hepatocyte lipid accumulation...