Fiorillo Laboratory
About the Lab
The Fiorillo Laboratory has a strong focus on Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disease caused by the absence of dystrophin protein. To restore dystrophin, a personalized medicine approach called exon skipping has been given conditional approval for treatment of DMD patients. However, the amount of dystrophin restoration that occurs through exon skipping is inconsistent and patchy. In our laboratory, we employ a variety of techniques to understand and improve dystrophin rescue in DMD. This includes investigating microRNAs that target and downregulate dystrophin, understanding the crosstalk between muscle and the immune system, determining how a secondary dystrophin deficiency might impact other muscle disorders and characterizing novel models of microRNA deletion that may affect the pathophysiology of muscle disorders.
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Lab Focus Areas
Therapeutic Targeting of Dystrophin-Targeting microRNA The Role of Inflammatory microRNAs in Duchenne Muscular Dystrophy & Other Inflammatory Muscle Disorders Genetic Models of Becker Muscular Dystrophy
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Contact
Alyson A. Fiorillo, Ph.D. Principal Investigator, Center for Genetic Medicine Research afiorillo@childrensnational.org 202-476-2472
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Featured Publications
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Estrogen receptor, inflammatory, and FOXO transcription factors regulate expression of myasthenia gravis-associated circulating microRNAs
Fiorillo AA, Heier CR, Huang YF, Tully CB, Punga T, Punga AR PubMed PMID: 32153563; PubMed Central PMCID: PMC7046803. Front Immunol 11:151. doi: 10.3389/fimmu.2020.00151. eCollection (2020) -
Muscle weakness in myositis: MicroRNA-mediated dystrophin reduction in a myositis mouse model and human muscle biopsies
Kinder TB, Heier CR, Tully CB, Van der Muelen JH, Hoffman EP, Nagaraju K, Fiorillo AA PubMed PMID: 32009304; PubMed Central PMCID: PMC7384101. Arthritis Rheumatol 72(7):1170-1183. doi: 10.1002/art.41215 May (2020) -
Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy
Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF PubMed PMID: 30745312; PubMed Central PMCID: PMC6371196. Life Sci Alliance 2(1). doi: 10.26508/lsa.201800186. Print February (2019) -
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone
Fiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR PubMed PMID: 29883261; PubMed Central PMCID: PMC6172612. Physiol Genomics 50(9):735-745. doi: 10.1152/physiolgenomics.00134.2017. Epub September (2018) -
TNF-α-induced microRNAs control dystrophin expression in Becker muscular dystrophy
Fiorillo AA, Heier CR, Novak JS, Tully CB, Brown KJ, Uaesoontrachoon K, Vila MC, Ngheim PP, Bello L, Kornegay JN, Angelini C, Partridge TA, Nagaraju K, Hoffman EP PubMed PMID: 26321630; PubMed Central PMCID: PMC4757433. Cell Rep 12(10):1678-90. doi: 10.1016/j.celrep.2015.07.066. Epub August (2015)