Center for Genetic Medicine Research
About the Lab
Our goal is to develop next-generation treatments for diseases in children. Our particular focus is on genetic diseases of muscle (such as Duchenne muscular dystrophy), but by targeting the underlying molecular biology of disease we believe we can help a broad array of human illnesses. Through our understanding of drug pathways, we are also developing serum-based biomarkers reflective of drug activities in patients. Ultimately, we feel this approach has the power to greatly improve the treatment of a large number of diseases featuring chronic inflammation, muscle disease or heart failure.
Multi-omics identifies circulating miRNA and protein biomarkers for facioscapulohumeral dystrophyHeier CR, Zhang A, Nguyen NY, Tully CB, Panigrahi A, Gordish-Dressman H, Nand Pandey S, Guglieri M, Ryan MM, Clemens PR, Thangarajh M, Webster R, Smith EC, Connolly AM, McDonald CM, Karachunski P, Tulinius M, Harper A, Mah JK, Fiorillo AA, Chen YW, CINRG PMCID: PMC7711540 J Pers Med 10(4):E236 November (2020)
Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathyHeier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF PMCID: PMC6371196 Life Sci Alliance 2(1). pii: e201800186 February (2019)
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamoroloneFiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR *Chosen for cover of journal PMCID: PMC6172612. Physiol Genomics 50(9):735-745 September (2018)
Identification of pathway-specific serum biomarkers of response to glucocorticoid and infliximab treatment in children with inflammatory bowel diseaseHeier CR, Fiorillo AA, Chaisson E, Gordish-Dressman H, Hathout Y, Damsker JM, Hoffman EP, and Conklin LS PMCID: PMC5288595 Clin Transl Gastroenterol 7(9):e192 September (2016)
TNF-α-induced microRNAs control dystrophin expression in Becker muscular dystrophyFiorillo 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 PMCID: PMC4757433 Cell Rep 12(10):1678-90 September (2015)