Christian Mueller, PhD


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 Mar 29;11(3):e0152589. doi: 10.1371/journal.pone.0152589. eCollection 2016. PMID: 27023329

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 Huntington’s Dis. 2016 Mar 19;5(1):33-8. doi: 10.3233/JHD-150183. PMID: 27003665

Borel F, Gernoux G, Cardozo B, Metterville JP, Cabrera TG, Song L, Su Q, ElMallah MK, Brown R, Mueller C. Therapeutic rAAVrh.10-mediated SOD1 silencing in adult SOD1G93A mice and non-human primates. Hum Gene Ther. 2016 Jan;27(1):19-31. doi: 10.1089/hum.2015.122. PMID: 26710998


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* Jr. Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice. Neuron. 2015 Dec 2;88(5):902-9. doi: 10.1016/j.neuron.2015.11.018. PMID: 26637797

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 Sep 25. doi: 10.1038/mt.2015.174. PMID: 26403887

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. doi: 10.1089/hgtb.2015.040. PMID: 25819833

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. doi: 10.1038/mt.2015.7. Epub 2015 Jan 16. PMID: 25592336


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 Oct 15;7:81. doi: 10.3389/fnmol.2014.00081. PMID: 25360081

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. doi:10.1089/hum.2014.2506. PMID:24628440


Borel F, Kay MA, Mueller CRecombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference. Mol Ther. 2013 Dec 19. doi: 10.1038/mt.2013.285. PMID:24352214

Mueller C, Jeffrey D. Chulay, Bruce C. Trapnell, Margaret Humphries, Brenna Carey, Robert A. Sandhaus, Noel G. McElvaney, Louis Messina, Qiushi Tang, Farshid N. Rouhani, Martha Campbell-Thompson, Ann Dongtao Fu, Anthony Yachnis, David R. Knop, Guo-jie Ye, Mark Brantly, Roberto Calcedo, Suryanarayan Somanathan, Lee P. Richman, Robert H. Vonderheide, Maigan A. Hulme, Todd M. Brusko, James M. Wilson, Terence R. Flotte. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest. 2013 Nov 15. doi:pii: 70314. 10.1172/JCI70314. PMID:24231351

Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency.COPD. 2013 Mar;10 Suppl 1:44-9. doi: 10.3109/15412555.2013.764978. PMID:23527792

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 Feb; Chapter 14:Unit 14D.3.doi: 10.1002/9780471729259.mc14d03s28. PMID:23408131


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. 2012 Sep 14. pii: S1569-1993(12)00153-1. doi: 10.1016/j.jcf.2012.08.014. [Epub ahead of print] PMID:22985691

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

Mueller C, Ratner D, Zhong L, Esteves-Sena M, Gao GProduction and discovery of novel recombinant adeno-associated viral vectors. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.1. PMID:22875565

Ratner D, Mueller C. Immune responses in Cystic Fibrosis; are they intrinsically defective? Am J Respir Cell Mol Biol. 2012 Mar 8. PMID:22403802

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 Mar 6. doi: 10.1038/mt.2012.39. PubMed PMID:22395529

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 Jan 17. doi: 10.1038/mt.2011.292. PMID:22252449

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 alpha-1 antitrypsin interaction with caspases. Mol Med. 2012 Jan 10. PMID:22245800


Bickford JS, Newsom KJ, Herlihy JD, Muller 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 TNF-α in Bronchoepithelial Cells. Biochem J. 2011 Nov 15. PMID:22082005

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 α(1)-Antitrypsin: Interim Results. Hum Gene Ther. 2011 Aug 24. PMID:21609134

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. doi: 10.1038/mt.2011.98. Epub 2011 May 24. PMID:21610699

Flotte TR, Frederickson RM, Lowenstein PR, Mueller CMoving 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. No abstract available. PMID:21532611

Flotte TR, Mueller CGene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 27. PMID:21498872


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. 2010 Dec 21. PMID:21179009

Mueller C, Braag SA, Keeler A, Hodges C, Drumm M, Flotte TR. Lack of Cftr in CD3+ Lymphocytes Leads to Aberrant Cytokine Secretion and Hyper-inflammatory Adaptive Immune Responses. Am J Respir Cell Mol Biol. 2010 Aug 19. PMID:20724552

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. 2010 Aug 6. PMID:20693405

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. Epub 2009 Nov 24. PMID:19935781


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. Epub 2009 Nov 5. PMID:19890354

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. Epub 2009 Oct 13. PMID:19826405

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 Aug 12. PMID: 19706466 

Books and Chapters

Toro Cabrera G, Mueller C. Design of shRNA and miRNA for Delivery to the CNS. Methods Mol Biol. 2016. PMID: 26611579

Terence R. Flotte, Thomas J. Conlon, Christian MuellerPreclinical Study Design for rAAV, Chapter 14.  Methods in Molecular Biology, Adeno-Associated Virus: Methods and Protocols. R.O. Snyder and P. Moullier, eds.  Humana Press/Springer, Totowa, New Jersey. 807:317-37. 2011. PMID:22034037

Christian Mueller, and Terence R. Flotte.  Immune Responses to Pulmonary Gene Therapy; Immunology and Gene Transfer. New Jersey. John Wiley and Sons Inc. 2007.

Christian Mueller, and Terence R. Flotte. Alpha-one Antitrypsin Deficiency; Rudolph’s Pediatrics 22e. Mc Graw Hill Co. 2010.

Stalvey MS, Mueller C, Flotte TR.  “Cystic Fibrosis” in The 5-Minute Clinical Consult, Lippincott Williams & Wilkins. 2010.

Mueller C Flotte TR. “Regenerative therapy for the musculoskeletal system using recombinant adeno-associated viral vectors” Eds: Cucchiarini M. and Madry H. Research Signpost. 2010.

Christian Mueller, and Terence R. Flotte. “Lung Biology in Health and Disease” series book entitled “Cystic Fibrosis: Gene Repair: Present and Future” Eds. Julian Allen, Howard Panitch and Ronald Rubenstein. Informa Healthcare 2010.

Martino AT, Mueller C, Flotte TR. A brief guide to gene therapy treatments for pulmonary diseases. In: A Guide to Human Gene Therapy, Herzog RW and Zolotukhin S (eds), World Scientific Publishing Company, chapter 21. 2010.

Christian Mueller and Terence R. Flotte “Interfacing Stem Cells with Gene Therapy”: Human Stem Cell Technology & Biology: A Research Guide and Laboratory Manual, edited by Gary S. Stein, 2010


 Dr. Chris Mueller from the Mueller Lab (Horae Gene Therapy Center) is conducting research and developing therapeutic strategies for rare inherited diseases such as the Alpha-one antitrypsin (AAT) deficiency

Chris Mueller, PhD


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