M. Carrie Miceli, Ph.D.

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

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

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

Affiliations
Affiliations
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
Biography:

Dr. Miceli is a professor in the Department of Microbiology Immunology and Molecular genetics. She received her BA from UCSD in Biochemistry and Cell Biology, her PhD from Duke University studying the role of T cells in human kidney allograft rejection (Olja Finn, mentor) and did postdoctoral work at Stanford elucidating molecular mechanisms of CD4, CD8, Lck and TCR coreceptor functions (Jane Parne’s laboratory). In her own laboratory her group has identified mechanisms and modulators of TCR signal specificity and T cell function, with broad application to signal transduction in general. In 2007, with colleagues, r. Miceli founded the Center for Duchenne Muscular Dystrophy (CDMD), which formed and catalyzed new DMD team science approaches to translational research, drug discovery, and clinical trial development on campus and nationwide. Combining her expertise in these areas she is now focused on dystrophin replacement therapies and immune drivers of regeneration, fibrosis and muscle tissue tolerance.

Publications

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.
Moran M, Miceli M C   Engagement of GPI-linked CD48 contributes to TCR signals and cytoskeletal reorganization: a role for lipid rafts in T cell activation Immunity, 1998; 9(6): 787-96.