M Carrie Miceli

M Carrie Miceli

Professor Professor of Microbiology Immunology and Molecular Genetics Co-Director Center for Duchenne Muscular Dystrophy at UCLA Director, CDMD Cell and Tissue Repository. California Center for Rare Diseases Molecular Biology Institute Graduate Programs in Bio-Science Homes: Immunity Microbes and Molecular Pathogenesis, Cell and Developmental Biology.

310-206-6483

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, Dr. 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.

Representative Publications

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

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. *denotes equal contribution, Miceli, Nelson, and Crosbie-Watson share senior authorship. Miceli Corresponding Author. Submitted

Wang, DW, Mokhonova, E, Kendall, GC, Becerra, D., Naeini, Y, Spencer, MJ, Cantor, R. Nelson, SF*, Miceli, MC* Repurposing Dantrolene for Long-term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx mouse. Molecular Therapy: Nucleic Acids. (2018) Nucleic Acids Vol. June 11: 180-191 *denotes equal contributions. Miceli corresponding author.

McMoran Brian J, Miceli, MC and Baum L. Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle.  Glycobiology, Volume 27, Issue 12, 1 December 2017, Pages 1134–1143,

Nelson, Stanley F.*, Miceli MC*# FDA Approval of Eteplirsen for Duchenne Muscular Dystrophy. *denotes equal contributions #corresponding author  JAMA 317, (14):1481-1482. doi:10.1001/jama.2017.2601

Victor, RG, Sweeney, HL, Finkel , R., McDonald, C., Byrne, B. Eagle, Goemans, N, Vandenborne, K, Alberto L, Dubrovsky, L, Topaloglu, H. Miceli, M. C., Furlong, P, Landry, J, Elashoff, R., Cox, D., For the Tadalafil DMD Study Group. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy. 2017 Oct 24; 89(17): 1811–1820. PMCID: PMC5664308 PMID:28972192

Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. (2016). 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 Apr 7;18(4):533-40 PMCID: PMC4826286.

Miceli, MC*, Nelson, SF* The case for eteplirsen: Paving the way for precision medicine. *denotes equal contributions.  Molecular Genetics and Metabolism. 118, 2, 70–71, 2016

Capote J, Kramerova I, Martinez L, Vetrone S, Barton ER, Sweeney HL, Miceli MC, Spencer, MJ. (2016). Osteopontin Ablation Ameliorates Muscular Dystrophy by shifting macrophages to a pro-regenerative phenotype. J Cell Biol. 25;213(2):275-88. 2016

Crocetti, J*, Silva, O*, Humphries, L., Tibbs, MS, Miceli, M. C. Selective phosphorylation of the Dlg1AB variant is critical for TCR-induced p38 activation and induction of pro-inflammatory cytokines in CD8+ T cells, Journal of Immunology, 193, 2651-2660. 2014 * denotes equal contributions

Wang, RT, Barthelemy, F. Martin, ED, Douine, D, Eskin, A, Lucas, K, Lavigne, JA. L Peay, H, Khanlou, N, Cantor, RM. Cantor, Miceli. MC* and Nelson SF*. DMD Genotype Correlations from the Duchenne Registry: endogenous exon skipping, is a factor in prolonged ambulation for individuals with a defined mutation sub-type.  Human Mutation. 39:1193–1202. 2018. * denotes equal contributions

Silva, O, Crocetti, J, Humphries, J, Burkhardt, J and M. C. Miceli (2015). Discs Large Homolog 1 splice variants regulate p38 dependent  and Independent Effector Functions in CD8+ T cells. PLOS One.  Online July 17, (2015).

Nelson, MD, Rader, F. Tang, X, Tavyev, J., Nelson, SF, Miceli, M. Carrie, Elashoff, R.M., Sweeney, H. L. and Victor, RG. PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy. Neurology, 82, 2085-2091, 2014

Kendall, GC, Mokhonova, E. Moran, M, Sejbuk, N, Wang, DW, Silva, O, Wang, RT, Martinez, Lu, QL, Damoiseaux, R, Spencer, MJ *, Nelson, SF* Miceli, MC*†. Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy.  Science Translational Medicine 4, 164, p. 164ra160, December 2012 * denotes equal contributions

Vetrone, S.A., Montecino-Rodriguez, E., Kudryashova, E., Kramerova, I., Hoffman, E.P., Liu, S.D., Miceli, M.C., Spencer M.J.  Osteopontin promotes fibrosis in Mdx muscle through  modulation of immune cell subsets and intramuscular TGFbeta.  Journal of Clinical Investigation, 119 (6):1583-1594, 2009. PMCID: PMC2689112

Liu, S.D., Tomassian, T., Miceli, M.C.  Galectin-1 tunes TCR binding and signal transduction to regulate CD8 burst size.  Journal of Immunology, 182 (9):5283-5295, 2009.

Liu, SD, Chung, CD, Tomassian, T, Pang, M, Baum, LG, Miceli, MC.  Endogenous galectin-1 functions to enforce class I restricted TCR functional fate decisions in thymocytes.  Blood, 112 (1):120-130, 2008.

Round, JL, Humphries, LA, Tomassian, T, Mittelstadt, P, Zhang M, Miceli, MC.  Scaffold protein Dlgh1 coordinates alternative p38 kinase activation, directing T cell receptor signals toward NFAT but not NF-kB transcription factors.  Nature Immunology, 8 (2):154-161, 2007.

Round, J.L., Tomassian, T., Zhang, M., Patel, P., Schoenberger, S.P., Miceli, M.C.  Dlgh1 coordinates actin polymerization, synaptic TCR and lipid raft aggregation and effector function in T lymphocytes.  Journal of Experimental Medicine, 201 (3):419-430, 2005.  PMCID: PMC2213022

Moran, M. and Miceli, M.C.  Engagement of GPI-anchored T cell CD48 contributes to TCR signals and cytoskeletal reorganization: a role for lipid rafts in T cell activation.  Immunity, 9: 787-796, 1998.