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Christian Mueller PhD

TitleAssociate Professor
InstitutionUniversity of Massachusetts Medical School
DepartmentPediatrics
AddressUniversity of Massachusetts Medical School
368 Plantation Street, AS6-2053
Worcester MA 01605
Phone508-856-4358
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    Other Positions
    InstitutionUMMS - School of Medicine
    DepartmentGene Therapy Center

    InstitutionUMMS - School of Medicine
    DepartmentPediatrics
    DivisionPediatric Pulmonology

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentMD/PhD Program


    Collapse ORNG Applications 
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    Collapse Biography 
    Collapse education and training
    Florida Institute of Technology, Melbourne, FL, United StatesBSMolecular Biology
    University of Florida, Gainesville, FL, United StatesPHDGenetics

    Collapse Overview 
    Collapse Rotation Projects

    We seek two talented and motivated postdocs to join the laboratory of Dr Christian Mueller in the Horae Gene Therapy Center at the University of Massachusetts Medical School. Our dynamic and collaborative research environment at UMMS includes world renowned clinicians and scientists. Additionally, our location close to Boston supports many collaborations with academic and industrial research groups. Under the leadership of Dr GuangPing Gao, the UMMS GTC is one of the top US gene therapy centers, focusing its research efforts on rare diseases of the liver, CNS, and eye, with a state-of-the-art vector core that handles vector production for its researchers.


     


    Open positions



    1. CNS Lead. Responsible for advancing the lab’s preclinical programs for CNS diseases. Lead indications are SOD1, C9orf72-linked amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD). Projects will include early stage research on genome editing for ALS, an IND-enabling study in non-human primates for a gene therapy candidate for ALS, and an IND-enabling study in non-human primates for a gene therapy candidate for HD.

    2. Liver Lead. Responsible for advancing the lab’s preclinical programs for liver diseases. Lead indication is α-1 antitrypsin deficiency (AATD). Projects will include early research on genome editing for AATD, miRNA roles in liver disease, characterization of two novel animal models for AATD and an IND-enabling study in non-human primates for a gene therapy candidate for AATD.


     


    Applicants should have a PhD in biomedical sciences with a strong background in molecular biology and biochemistry, as well a track record in the field of ALS/CNS disorders (CNS Lead role) or liver diseases (Liver Lead role). Hands-on experience with relevant mouse models as well as excellent knowledge of CRISPR-based genome editing are required. Ability to work independently in a fast-paced research environment and multitask, be highly organized and adaptable are required. Strong writing, verbal and interpersonal skills are required.


     


    Required skills



    • PhD degree in biomedical sciences

    • track record in ALS/CNS disorders or liver diseases with at least two first author publications

    • hands-on experience with genetic mouse models (CNS or liver)

    • excellent knowledge of CRISPR-based gene editing

    • fluently speak and write English


     


    Preferred skills



    • hands-on experience with CRISPR-based gene editing

    • experience in next generation sequencing and bioinformatics

    • experience in tissue culture, cytokine multiplex assays and ELISA


     


    Our lab is multicultural and supports personal development and work-life balance. Attractive salary and benefits available based upon relative working experience.


    Worcester is located an hour away from Boston and offers a good quality of life with plenty or nearby outdoor activities and many restaurants.


    For more information on the Mueller Lab for Gene Therapy we invite you to visit our website: www.umassmed.edu/muellerlab


     



    Currently the lab has rotation projects open on all areas of research. Please visit or lab website (www.umassmed.edu/muellerlab) to what area interests you. 



    Collapse Post Docs

    We seek two talented and motivated postdocs to join the laboratory of Dr Christian Mueller in the Horae Gene Therapy Center at the University of Massachusetts Medical School. Our dynamic and collaborative research environment at UMMS includes world renowned clinicians and scientists. Additionally, our location close to Boston supports many collaborations with academic and industrial research groups. Under the leadership of Dr GuangPing Gao, the UMMS GTC is one of the top US gene therapy centers, focusing its research efforts on rare diseases of the liver, CNS, and eye, with a state-of-the-art vector core that handles vector production for its researchers.


     


    Open positions



    1. CNS Lead. Responsible for advancing the lab’s preclinical programs for CNS diseases. Lead indications are SOD1, C9orf72-linked amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD). Projects will include early stage research on genome editing for ALS, an IND-enabling study in non-human primates for a gene therapy candidate for ALS, and an IND-enabling study in non-human primates for a gene therapy candidate for HD.

    2. Liver Lead. Responsible for advancing the lab’s preclinical programs for liver diseases. Lead indication is α-1 antitrypsin deficiency (AATD). Projects will include early research on genome editing for AATD, miRNA roles in liver disease, characterization of two novel animal models for AATD and an IND-enabling study in non-human primates for a gene therapy candidate for AATD.


     


    Applicants should have a PhD in biomedical sciences with a strong background in molecular biology and biochemistry, as well a track record in the field of ALS/CNS disorders (CNS Lead role) or liver diseases (Liver Lead role). Hands-on experience with relevant mouse models as well as excellent knowledge of CRISPR-based genome editing are required. Ability to work independently in a fast-paced research environment and multitask, be highly organized and adaptable are required. Strong writing, verbal and interpersonal skills are required.


     


    Required skills



    • PhD degree in biomedical sciences

    • track record in ALS/CNS disorders or liver diseases with at least two first author publications

    • hands-on experience with genetic mouse models (CNS or liver)

    • excellent knowledge of CRISPR-based gene editing

    • fluently speak and write English


     


    Preferred skills



    • hands-on experience with CRISPR-based gene editing

    • experience in next generation sequencing and bioinformatics

    • experience in tissue culture, cytokine multiplex assays and ELISA


     


    Our lab is multicultural and supports personal development and work-life balance. Attractive salary and benefits available based upon relative working experience.


    Worcester is located an hour away from Boston and offers a good quality of life with plenty or nearby outdoor activities and many restaurants.


    For more information on the Mueller Lab for Gene Therapy we invite you to visit our website: www.umassmed.edu/muellerlab


     




    Collapse Bibliographic 
    Collapse selected publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
    List All   |   Timeline
    1. Esanov R, Cabrera GT, Andrade NS, Gendron TF, Brown RH, Benatar M, Wahlestedt C, Mueller C, Zeier Z. A C9ORF72 BAC mouse model recapitulates key epigenetic perturbations of ALS/FTD. Mol Neurodegener. 2017 Jun 12; 12(1):46. PMID: 28606110.
      View in: PubMed
    2. Pfister EL, Chase KO, Sun H, Kennington LA, Conroy F, Johnson E, Miller R, Borel F, Aronin N, Mueller C. Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin. Mol Ther Nucleic Acids. 2017 Jun 16; 7:324-334. PMID: 28624208.
      View in: PubMed
    3. 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 Apr 10. PMID: 28408179.
      View in: PubMed
    4. Gernoux G, Wilson JM, Mueller C. Regulatory and Exhausted T Cell Responses to AAV Capsid. Hum Gene Ther. 2017 Apr; 28(4):338-349. PMID: 28323492.
      View in: PubMed
    5. 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 Feb 14; 114(7):1655-1659. PMID: 28137880.
      View in: PubMed
    6. Guerra ES, Lee CK, Specht CA, Yadav B, Huang H, Akalin A, Huh JR, Mueller C, Levitz SM. Central Role of IL-23 and IL-17 Producing Eosinophils as Immunomodulatory Effector Cells in Acute Pulmonary Aspergillosis and Allergic Asthma. PLoS Pathog. 2017 Jan 17; 13(1):e1006175. PMID: 28095479.
      View in: PubMed
    7. Liqun Wang R, McLaughlin T, Cossette T, Tang Q, Foust K, Campbell-Thompson M, Martino A, Cruz P, Loiler S, Mueller C, Flotte TR. Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of a-1-Antitrypsin Using Invasive and Noninvasive Delivery. Mol Ther. 2009 Jan; 17(1):81-87. PMID: 28188992.
      View in: PubMed
    8. Gruenert AK, Czugala M, Mueller C, Schmeer M, Schleef M, Kruse FE, Fuchsluger TA. Self-Complementary Adeno-Associated Virus Vectors Improve Transduction Efficiency of Corneal Endothelial Cells. PLoS One. 2016; 11(3):e0152589. PMID: 27023329; PMCID: PMC4811580.
    9. Stoica L, Todeasa SH, Cabrera GT, Salameh JS, ElMallah MK, Mueller C, Brown RH, Sena-Esteves M. Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. Ann Neurol. 2016 Apr; 79(4):687-700. PMID: 26891182.
      View in: PubMed
    10. Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH, Mueller C. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. Hum Gene Ther. 2016 Jan; 27(1):19-31. PMID: 26710998.
      View in: PubMed
    11. Liu W, Pfister EL, Kennington LA, Chase KO, Mueller C, DiFiglia M, Aronin N. Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon? J Huntingtons Dis. 2016; 5(1):33-8. PMID: 27003665.
      View in: PubMed
    12. Toro Cabrera G, Mueller C. Design of shRNA and miRNA for Delivery to the CNS. Methods Mol Biol. 2016; 1382:67-80. PMID: 26611579.
      View in: PubMed
    13. Peters OM, Cabrera GT, Tran H, Gendron TF, McKeon JE, Metterville J, Weiss A, Wightman N, Salameh J, Kim J, Sun H, Boylan KB, Dickson D, Kennedy Z, Lin Z, Zhang YJ, Daughrity L, Jung C, Gao FB, Sapp PC, Horvitz HR, Bosco DA, Brown SP, de Jong P, Petrucelli L, Mueller C, Brown RH. Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice. Neuron. 2015 Dec 02; 88(5):902-9. PMID: 26637797.
      View in: PubMed
    14. Li S, Ling C, Zhong L, Li M, Su Q, He R, Tang Q, Greiner DL, Shultz LD, Brehm MA, Flotte TR, Mueller C, Srivastava A, Gao G. Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors. Mol Ther. 2015 Dec; 23(12):1867-76. PMID: 26403887.
      View in: PubMed
    15. Gruntman AM, Su L, Su Q, Gao G, Mueller C, Flotte TR. Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. Hum Gene Ther Methods. 2015 Apr; 26(2):71-6. PMID: 25819833.
      View in: PubMed
    16. Benskey MJ, Kuhn NC, Galligan JJ, Garcia J, Boye SE, Hauswirth WW, Mueller C, Boye SL, Manfredsson FP. Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. Mol Ther. 2015 Mar; 23(3):488-500. PMID: 25592336; PMCID: PMC4351472.
    17. Gombash SE, Cowley CJ, Fitzgerald JA, Hall JC, Mueller C, Christofi FL, Foust KD. Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice. Front Mol Neurosci. 2014; 7:81. PMID: 25360081; PMCID: PMC4197761.
    18. Flotte TR, Mueller C. What is suppression of anti-adeno-associated virus capsid T-cells achieving? Hum Gene Ther. 2014 Mar; 25(3):178-9. PMID: 24628440.
      View in: PubMed
    19. Borel F, Kay MA, Mueller C. Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Mol Ther. 2014 Apr; 22(4):692-701. PMID: 24352214; PMCID: PMC3982504.
    20. 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. PMID: 24231351.
      View in: PubMed
    21. Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9. PMID: 23527792.
      View in: PubMed
    22. van der Bom IM, Moser RP, Gao G, Mondo E, O'Connell D, Gounis MJ, McGowan S, Chaurette J, Bishop N, Sena-Esteves MS, Mueller C, Aronin N. Finding the striatum in sheep: use of a multi-modal guided approach for convection enhanced delivery. J Huntingtons Dis. 2013; 2(1):41-5. PMID: 25063428.
      View in: PubMed
    23. Gruntman AM, Bish LT, Mueller C, Sweeney HL, Flotte TR, Gao G. Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:Unit 14D.3. PMID: 23408131.
      View in: PubMed
    24. Bickford JS, Mueller C, Newsom KJ, Barilovits SJ, Beachy DE, Herlihy JD, Keeler B, Flotte TR, Nick HS. Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency. J Cyst Fibros. 2013 May; 12(3):258-65. PMID: 22985691.
      View in: PubMed
    25. Mueller C, Ratner D, Zhong L, Esteves-Sena M, Gao G. Production and discovery of novel recombinant adeno-associated viral vectors. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.1. PMID: 22875565.
      View in: PubMed
    26. Gruntman AM, Mueller C, Flotte TR, Gao G. Gene transfer in the lung using recombinant adeno-associated virus. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.2. PMID: 22875566.
      View in: PubMed
    27. 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. PMID: 22245800.
      View in: PubMed
    28. Ratner D, Mueller C. Immune responses in cystic fibrosis: are they intrinsically defective? Am J Respir Cell Mol Biol. 2012 Jun; 46(6):715-22. PMID: 22403802.
      View in: PubMed
    29. Keeler AM, Conlon T, Walter G, Zeng H, Shaffer SA, Dungtao F, Erger K, Cossette T, Tang Q, Mueller C, Flotte TR. Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy. Mol Ther. 2012 Jun; 20(6):1131-8. PMID: 22395529.
      View in: PubMed
    30. Bickford JS, Newsom KJ, Herlihy JD, Mueller C, Keeler B, Qiu X, Walters JN, Su N, Wallet SM, Flotte TR, Nick HS. Induction of group IVC phospholipase A2 in allergic asthma: transcriptional regulation by TNFa in bronchoepithelial cells. Biochem J. 2012 Feb 15; 442(1):127-37. PMID: 22082005.
      View in: PubMed
    31. Mueller C, Tang Q, Gruntman A, Blomenkamp K, Teckman J, Song L, Zamore PD, Flotte TR. Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. Mol Ther. 2012 Mar; 20(3):590-600. PMID: 22252449.
      View in: PubMed
    32. 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. PMID: 21609134.
      View in: PubMed
    33. Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown R, Xu Z, Gao G. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther. 2011 Aug; 19(8):1440-8. PMID: 21610699.
      View in: PubMed
    34. Nath B, Levin I, Csak T, Petrasek J, Mueller C, Kodys K, Catalano D, Mandrekar P, Szabo G. Hepatocyte-specific hypoxia-inducible factor-1a is a determinant of lipid accumulation and liver injury in alcohol-induced steatosis in mice. Hepatology. 2011 May; 53(5):1526-37. PMID: 21520168.
      View in: PubMed
    35. Flotte TR, Frederickson RM, Lowenstein PR, Mueller C. Moving forward toward a cure for Parkinson's: neuropathology of the nigrostriatal pathway determines the location of growth factor delivery. Mol Ther. 2011 May; 19(5):827-9. PMID: 21532611.
      View in: PubMed
    36. Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 15; 20(R1):R87-92. PMID: 21498872.
      View in: PubMed
    37. Flotte TR, Conlon TJ, Mueller C. Preclinical study design for rAAV. Methods Mol Biol. 2011; 807:317-37. PMID: 22034037.
      View in: PubMed
    38. Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, Gao G. MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther. 2011 Mar; 19(3):526-35. PMID: 21179009.
      View in: PubMed
    39. Mueller C, Braag SA, Keeler A, Hodges C, Drumm M, Flotte TR. Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses. Am J Respir Cell Mol Biol. 2011 Jun; 44(6):922-9. PMID: 20724552.
      View in: PubMed
    40. Martino AT, Mueller C, Braag S, Cruz PE, Campbell-Thompson M, Jin S, Flotte TR. N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance. Am J Respir Cell Mol Biol. 2011 Jun; 44(6):824-30. PMID: 20693405.
      View in: PubMed
    41. Harrison LH, Tam SK, Mueller CP. Partial anomalous pulmonary venous connection in adults: a simplified approach. Ann Thorac Surg. 2010 Jan; 89(1):283-5. PMID: 20103258.
      View in: PubMed
    42. Mueller C, Keeler A, Braag S, Menz T, Tang Q, Flotte TR. Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e. Mol Ther. 2010 Mar; 18(3):511-8. PMID: 19935781.
      View in: PubMed
    43. Mueller C, Strayer MS, Sirninger J, Braag S, Branco F, Louboutin JP, Flotte TR, Strayer DS. In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors. Gene Ther. 2010 Feb; 17(2):227-37. PMID: 19890354.
      View in: PubMed
    44. Flotte TR, Fischer AC, Goetzmann J, Mueller C, Cebotaru L, Yan Z, Wang L, Wilson JM, Guggino WB, Engelhardt JF. Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. Mol Ther. 2010 Mar; 18(3):594-600. PMID: 19826405.
      View in: PubMed
    45. 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. PMID: 19706466.
      View in: PubMed
    46. Mueller C, Flotte TR. Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol. 2008 Dec; 35(3):164-78. PMID: 18600482.
      View in: PubMed
    47. Liqun Wang R, McLaughlin T, Cossette T, Tang Q, Foust K, Campbell-Thompson M, Martino A, Cruz P, Loiler S, Mueller C, Flotte TR. Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery. Mol Ther. 2009 Jan; 17(1):81-7. PMID: 18941444.
      View in: PubMed
    48. Mueller C, Braag SA, Martino AT, Tang Q, Campbell-Thompson M, Flotte TR. The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr-/- -dependent allergy mouse model. Gene Ther. 2009 Feb; 16(2):172-83. PMID: 18818669.
      View in: PubMed
    49. Stalvey MS, Brusko TM, Mueller C, Wasserfall CH, Schatz DA, Atkinson MA, Flotte TR. CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes. Cytokine. 2008 Oct; 44(1):154-9. PMID: 18778952.
      View in: PubMed
    50. Flotte TR, Goetzmann J, Caridi J, Paolillo J, Conlon TJ, Potter M, Mueller C, Byrne BJ. Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. Hum Gene Ther. 2008 Jul; 19(7):681-9. PMID: 18588426.
      View in: PubMed
    51. Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 2008 Jun; 15(11):858-63. PMID: 18418415.
      View in: PubMed
    52. Mueller C, Torrez D, Braag S, Martino A, Clarke T, Campbell-Thompson M, Flotte TR. Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene. J Gene Med. 2008 Jan; 10(1):51-60. PMID: 18023072.
      View in: PubMed
    53. 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. PMID: 17924834.
      View in: PubMed
    54. 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. PMID: 17592477.
      View in: PubMed
    55. Müller C, Braag SA, Herlihy JD, Wasserfall CH, Chesrown SE, Nick HS, Atkinson MA, Flotte TR. Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- mice. Lab Invest. 2006 Feb; 86(2):130-40. PMID: 16424881.
      View in: PubMed
    56. Virella-Lowell I, Herlihy JD, Liu B, Lopez C, Cruz P, Muller C, Baker HV, Flotte TR. Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line. Mol Ther. 2004 Sep; 10(3):562-73. PMID: 15336656.
      View in: PubMed
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