|Department Affiliations||Departments of Neurology and Neuroscience, Director, Center for Genetic Muscle Disorders|
|SOM Address||Room 400 707 N. Broadway|
Most people on the planet will suffer from a disorder of skeletal muscle at some point in their lives. They will either be born with a genetic disorder of muscle (totaling more than 50 in number), or develop an acquired primary myopathy (toxic, metabolic, inflammatory etc.), or develop an acquired myopathy secondary to a chronic illness (infection, cancer, congestive heart failure, COPD etc.) or if they are fortunate enough to survive to be an otherwise healthy octogenarian, develop wasting of muscle with aging called sarcopenia. Our lab is devoted to developing novel therapies for muscle disorders in order to improve function and quality of life. We are interested in the factors that influence muscle growth and regeneration over muscle atrophy and fibrosis. We use a variety of techniques from molecular biology and protein biochemistry to cell culture of primary myoblasts to animal models of muscular dystrophies. With these techniques we are developing novel pharmacological, gene and cell-based therapies for muscle disorders. The lab has collaborations with a number of industry and academic partners including a Senator Paul Wellstone Muscular Dystrophy Cooperative Research Center. Several preclinical studies from the lab have gone onto human clinical trials within the Center for Genetic Muscle Disorders at Kennedy Krieger.
- Chen JCJ, King OD, Zhang Y, Clayton NP, Spencer C, Wentworth BM, Emerson CP, Wagner KR. Morpholino-Mediated Knockdown of DUX4 Towards Facioscapulohumeral Muscular Dystrophy Therapeutics. Molecular Therapy 2016.
- Choi IY, Lim H, Estrellas K, Mula J, Cohen T, Zhang Y, Donnelly C, Richard JP, Kin Y, Kim H, Kazuki Y, Oshimura M, Rothstein J, Maragakis N, Wagner KR, Lee G. Concordant but varied phenotypes among patient-specific myoblasts of Duchenne muscular dystrophy revealed by human iPSC-based model. Cell Reports 2016, 15:1-12.
- Choudhury SR, Fitzpatrick Z, Harris, AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Ray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo, C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M. In vivo selection yields AAV-B1 capsids for CNS and muscle gene therapy. Mol Ther. 2016
- Moyer AL and Wagner KR. Mammalian Mss51 is a skeletal muscle-specific gene modulating cellular metabolism. J Neuromusc Disease. 2015, 2(4): 371-385.
- Cohen TA, Kollias HD, Liu N, Ward CW, Wagner, KR. Genetic disruption of Smad7 impairs skeletal muscle growth and regeneration. J Physiol. 2015, 593 (11): 2479-97.
- Zhang Y, King OD, Rahimov, F, Jones TI, Ward CW, Kerr JP, Liu N, Emerson CP Jr, Kunkel LM, Partridge TA, Wagner KR. Human skeletal muscle xenograft as a new preclinical model for muscle disorders. Hum Mol Genet. 2014, 23 (12): 3180-3188.