M. Carrie Miceli, Ph.D. [ Edit Your Profile ]

Work Email Address:

Lab Number: 206 6911
Office Phone Number: (310) 206-6483

Department Address:
276B Biomedical Sciences Research Building
CAMPUS - 157005

Laboratory Address:
257 Basic Sciences Research Building
Los Angeles, CA 90095

Work Address:
Department of Microbiology, Immunology, and Molecular Genetics
Los Angeles, CA 90095

Co-Director, Center for Duchenne Muscular Dystrophy
Professor, Microbiology, Immunology & Molecular Genetics
Member, Cell & Developmental Biology GPB Home Area, Immunity, Microbes & Molecular Pathogenesis GPB Home Area, Molecular Biology Institute



The Miceli lab is focused around two main research topics: T cell biology and Duchenne Muscular Dystrophy (DMD). DMD is a lethal genetic disease of childhood, caused by mutations in DMD, which encodes the dystrophin protein. Without functional dystrophin, defects in sarcolemma stabilization lead to progressive muscle damage, while inherent stem cell defects limit efficiency of regeneration. Immune cells infiltrate muscle in response to damage, coordinating muscle satellite cell activation and muscle regeneration. In the face of the chronic damage in DMD, inflammation also drives fibrosis, promoting scarring and further limiting muscle regeneration. Progressive muscle weakness leads to loss of virtually all muscle function, respiratory and cardiac failure, and death between ages 20-30. The Miceli lab works closely with the CDMD clinic, clinical trials population and industry partners to use DMD clinic and clinical trial tissue sample to model human DMD, screen for new drugs/targets, assess therapeutic efficacy and mechanisms of action and immune consequences of dystrophin replacement and immune modulating therapies.

Because DMD patient biopsy tissue is of limited availability, we are developing and applying technology for robust assessment of single cell multi-parameter immune and muscle lineage cell surface marker and gene expression, including TCR/BCR cell V region usage from cryopreserved PBMC, and frozen muscle biopsy tissue. Additionally, we developed patient mutation specific DMD skeletal muscle and cardiac culture models. We have active collaborations with industry partners involved in dystrophin replacement/rescue and immune suppression in DMD, and access to remnant trial tissue and clinic samples for our studies. By examining the consequences of therapeutics on immunity, muscle regeneration, fibrosis and tissue tolerance in human DMD, we hope to identify therapeutic mechanism of action and efficacy, cell and molecular targets for drug discovery, and potential barriers to successful treatment. We hypothesize that these studies will identify novel immune and myogenic drivers of muscle regeneration, fibrosis and immune tolerance in human DMD.


A selected list of publications:

Gibbs, EM, Barthélémy, F. Douine ED , Hardiman, N, Shieh, PB, Khanlou, N, Crosbie-Watson, RHC, Nelson, SF and Miceli, MC   Large in-frame 5’deletions in DMD associated with mild Duchenne Muscular Dystrophy: two case reports and a review of the literature. , , ; Submitted: .
Barthélémy, F, Wang, RT, Hsu, C, Douine, ED. Nelson, SF and Miceli, MC.   Targeting RyR activity boosts antisense exon 44 and 45 skipping in human DMD skeletal or cardiac muscle culture models. , , ; Submitted.
Wang Richard T, Barthelemy Florian, Martin Ann S, Douine Emilie D, Eskin Ascia, Lucas Ann, Lavigne Jenifer, Peay Holly, Khanlou Negar, Sweeney Lee, Cantor Rita M, Miceli M Carrie, Nelson Stanley F   DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype Human mutation, 2018; 39(9): 1193-1202.
Wang Derek W, Mokhonova Ekaterina I, Kendall Genevieve C, Becerra Diana, Naeini Yalda B, Cantor Rita M, Spencer Melissa J, Nelson Stanley F, Miceli M Carrie   Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse Molecular therapy. Nucleic acids, 2018; 11: 180-191.
McMorran Brian J, Miceli M Carrie, Baum Linda G   Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle Glycobiology, 2017; 27(12): 1134-1143.
Victor Ronald G, Sweeney H Lee, Finkel Richard, McDonald Craig M, Byrne Barry, Eagle Michelle, Goemans Nathalie, Vandenborne Krista, Dubrovsky Alberto L, Topaloglu Haluk, Miceli M Carrie, Furlong Pat, Landry John, Elashoff Robert, Cox David, Cox David   A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy Neurology, 2017; 89(17): 1811-1820.
Nelson Stanley F, Miceli M Carrie   FDA Approval of Eteplirsen for Muscular Dystrophy JAMA, 2017; 317(14): 1480.
Miceli M Carrie, Nelson Stanley F   The case for eteplirsen: Paving the way for precision medicine Molecular genetics and metabolism, 2016; 118(2): 70-1.
Young Courtney S, Hicks Michael R, Ermolova Natalia V, Nakano Haruko, Jan Majib, Younesi Shahab, Karumbayaram Saravanan, Kumagai-Cresse Chino, Wang Derek, Zack Jerome A, Kohn Donald B, Nakano Atsushi, Nelson Stanley F, Miceli M Carrie, Spencer Melissa J, Pyle April D   A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells Cell stem cell, 2016; 18(4): 533-40.
Capote Joana, Kramerova Irina, Martinez Leonel, Vetrone Sylvia, Barton Elisabeth R, Sweeney H Lee, Miceli M Carrie, Spencer Melissa J   Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype The Journal of cell biology, 2016; 213(2): 275-88.
Silva Oscar, Crocetti Jillian, Humphries Lisa A, Burkhardt Janis K, Miceli M Carrie   Discs Large Homolog 1 Splice Variants Regulate p38-Dependent and -Independent Effector Functions in CD8+ T Cells PloS one, 2015; 10(7): e0133353.
Crocetti Jillian, Silva Oscar, Humphries Lisa A, Tibbs Michelle D, Miceli M Carrie   Selective phosphorylation of the Dlg1AB variant is critical for TCR-induced p38 activation and induction of proinflammatory cytokines in CD8+ T cells Journal of immunology (Baltimore, Md. : 1950), 2014; 193(6): 2651-60.
Nelson Michael D, Rader Florian, Tang Xiu, Tavyev Jane, Nelson Stanley F, Miceli M Carrie, Elashoff Robert M, Sweeney H Lee, Victor Ronald G   PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy Neurology, 2014; 82(23): 2085-91.
Kendall Genevieve C, Mokhonova Ekaterina I, Moran Miriana, Sejbuk Natalia E, Wang Derek W, Silva Oscar, Wang Richard T, Martinez Leonel, Lu Qi L, Damoiseaux Robert, Spencer Melissa J, Nelson Stanley F, Miceli M Carrie   Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy Science translational medicine, 2012; 4(164): 164ra160.
Vetrone Sylvia A, Montecino-Rodriguez Encarnacion, Kudryashova Elena, Kramerova Irina, Hoffman Eric P, Liu Scot D, Miceli M Carrie, Spencer Melissa J   Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta The Journal of clinical investigation, 2009; 119(6): 1583-94.
Liu Scot D, Tomassian Tamar, Bruhn Kevin W, Miller Jeff F, Poirier Françoise, Miceli M Carrie   Galectin-1 tunes TCR binding and signal transduction to regulate CD8 burst size Journal of immunology (Baltimore, Md. : 1950), 2009; 182(9): 5283-95.
Liu Scot D, Whiting Chan C, Tomassian Tamar, Pang Mabel, Bissel Stephanie J, Baum Linda G, Mossine Valeri V, Poirier Françoise, Huflejt Margaret E, Miceli M Carrie   Endogenous galectin-1 enforces class I-restricted TCR functional fate decisions in thymocytes Blood, 2008; 112(1): 120-30.
Round June L, Humphries Lisa A, Tomassian Tamar, Mittelstadt Paul, Zhang Min, Miceli M Carrie   Scaffold protein Dlgh1 coordinates alternative p38 kinase activation, directing T cell receptor signals toward NFAT but not NF-kappaB transcription factors Nature immunology, 2007; 8(2): 154-61.
Round June L, Tomassian Tamar, Zhang Min, Patel Viresh, Schoenberger Stephen P, Miceli M Carrie   Dlgh1 coordinates actin polymerization, synaptic T cell receptor and lipid raft aggregation, and effector function in T cells The Journal of experimental medicine, 2005; 201(3): 419-30.