CVI Program Unit(s):
Myocyte Biology / Heart Failure
Thrombosis / Hemostasis
CVI Research Description:
Primary research focus: pathogenesis and therapy - inherited myopathies, including cardiomyopathies. In particular we have made recent progress in vector mediated gene delivery to the heart and respiratory muscles, a critical step in the develpment of durable therapy for many forms of muscular dystrophy.
In addition, my laboratory has been involved in collaborative studies (with Drs. Katherin High and Valder Arruda) on gene therapy for hemoplia, using a vascular approach to vector delivery in skeletal muscle to achieve systemic restoration of factor IX.
Selected Publications
Song Y, Morales L, Malik AS, Mead AF, Greer CD, Mitchell MA, Petrov MT, Su LT, Choi ME, Rosenblum ST, Lu X, VanBelzen DJ, Krishnankutty RK, Balzer FJ, Loro E, French R, Propert KJ, Zhou S, Kozyak BW, Nghiem PP, Khurana TS, Kornegay JN, Stedman HH: Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models. Nature Medicine 25(10): 1505-1511, Oct. 2019 Notes: Epub Oct. 7, 2019.
Song Y, Rosenblum ST, Morales L, Petrov M, Greer C, Globerman S, Stedman HH: Suite of clinically relevant functional assays to address therapeutic efficacy and disease mechanism in the dystrophic mdx mouse. Journal of Applied Physiology 122(3): 593-602, Mar. 1 2017 Notes: Epub Dec. 8, 2016.
VanBelzen DJ, Malik AS, Henthorn PS, Kornegay JN, Stedman HH: Mechanism of deletion removing all dystrophin exons in a canine model for DMD implicates concerted evolution of X chromosome pseudogenes. Molecular Therapy Methods & Clinical Development 4: 62-71, Dec. 24 2016.
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK: Perspectives on best practices for gene therapy programs. Human Gene Therapy 26(3): 127-133, Mar. 2015 Notes: Epub Mar. 3, 2015.
Mead AF, Petrov M, Malik AS, Mitchell MA, Childers MK, Bogan JR, Seidner G, Kornegay JN, Stedman HH: Diaphragm remodeling and compensatory respiratory mechanics in a canine model of Duchenne muscular dystrophy. Journal of Applied Physiology 116(7): 807-815, Apr. 1 2014 Notes: Epub Jan. 9, 2014.
Fargnoli AS, Katz MG, Yarnall C, Isidro A, Petrov M, Steuerwald N, Ghosh S, Richardville KC, Hillesheim R, Williams RD, Kohlbrenner E, Stedman HH, Hajjar RJ, Bridges CR: Cardiac surgical delivery of the sarcoplasmic reticulum calcium ATPase rescues myocytes in ischemic heart failure. Annals of Thoracic Surgery 96(2): 586-595, Aug. 2013 Notes: Epub June 15, 2013.
Fargnoli AS, Katz MG, Yarnall C, Sumaroka MV, Stedman H, Rabinowitz JJ, Koch WJ, Bridges CR: A pharmacokinetic analysis of molecular cardiac surgery with recirculation mediated delivery of βARKct gene therapy: developing a quantitative definition of the therapeutic window. Journal of Cardiac Failure 17(8): 691-699, Aug. 2011 Notes: Epub June 14, 2011.
White JD, Thesier DM, Swain JB, Katz MG, Tomasulo C, Henderson A, Wang L, Yarnall C, Fargnoli A, Sumaroka M, Isidro A, Petrov M, Holt D, Nolen-Walston R, Koch WJ, Stedman HH, Rabinowitz J, Bridges CR: Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus vectors in vivo. Gene Therapy 18(6): 546-552, June 2011 Notes: Epub Jan. 13, 2011.
Swain JD, Katz MG, White JD, Thesier DM, Henderson A, Stedman HH, Bridges CR: A translatable, closed recirculation system for AAV6 vector-mediated myocardial gene delivery in the large animal. Methods in Molecular Biology 709: 331-354, 2011.
Petrov M, Malik A, Mead A, Bridges CR, Stedman HH: Gene transfer to muscle from the isolated regional circulation. Methods in Molecular Biology 709: 277-286, 2011.
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Last updated: 04/24/2023
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