Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

Autor: Arden B. Piepho, Jeovanna Lowe, Laurel R. Cumby, Lisa E. Dorn, Dana M. Lake, Neha Rastogi, Megan D. Gertzen, Sarah L. Sturgill, Guy L. Odom, Mark T. Ziolo, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 344-354 (2023)

Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously validated F

Externí odkaz: https://doaj.org/article/112376132c914e0588ee19091b2c6a91

BEX1 is a critical determinant of viral myocarditis.

Autor: Colton R Martens, Lisa E Dorn, Adam D Kenney, Shyam S Bansal, Jacob S Yount, Federica Accornero

Publikováno v: PLoS Pathogens, Vol 18, Iss 2, p e1010342 (2022)

Viral infection of the heart is a common but underappreciated cause of heart failure. Viruses can cause direct cardiac damage by lysing infected cardiomyocytes. Inflammatory immune responses that limit viral replication can also indirectly cause dama

Externí odkaz: https://doaj.org/article/8c6a49a89578411b9e205be4566e928a

Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model

Autor: Zachary M. Howard, Lisa E. Dorn, Jeovanna Lowe, Megan D. Gertzen, Pierce Ciccone, Neha Rastogi, Guy L. Odom, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney

Publikováno v: JCI Insight, Vol 6, Iss 7 (2021)

Gene replacement for Duchenne muscular dystrophy (DMD) with micro-dystrophins has entered clinical trials, but efficacy in preventing heart failure is unknown. Although most patients with DMD die from heart failure, cardiomyopathy is undetectable unt

Externí odkaz: https://doaj.org/article/edf8afa3bfcf4133a3ecce62112683f8

Microfibrillar-Associated Protein 4 Regulates Stress-Induced Cardiac Remodeling

Autor: Matthew S. Stratton, Federica Accornero, Bryan A. Whitson, Xianyao Xu, Peter J. Mohler, Anders Schlosser, Grith Lykke Sørensen, William R Lawrence, Jennifer M. Petrosino, Paul M.L. Janssen, Thomas J. Hund, Lisa E. Dorn

Publikováno v: Circ Res
Dorn, L E, Lawrence, W, Petrosino, J M, Xu, X, Hund, T J, Whitson, B A, Stratton, M S, Janssen, P M L, Mohler, P J, Schlosser, A, Sorensen, G L & Accornero, F 2021, ' Microfibrillar-Associated Protein 4 Regulates Stress-Induced Cardiac Remodeling ', Circulation Research, vol. 128, no. 6, pp. 723-737 . https://doi.org/10.1161/CIRCRESAHA.120.317146

Rationale: Cardiac hypertrophy, a major risk factor for heart failure, occurs when cardiomyocytes remodel in response to complex signaling induced by injury or cell stress. Although cardiomyocytes are the ultimate effectors of cardiac hypertrophy, no

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::acb8f6e1525707a6c3efaa1b731e801b
https://doi.org/10.1161/circresaha.120.317146

Remodeling of the m6A landscape in the heart reveals few conserved post-transcriptional events underlying cardiomyocyte hypertrophy

Autor: Scott A. Hinger, Paul M.L. Janssen, Chuan He, Bryan A. Whitson, Lisa E. Dorn, Jiangbo Wei, Federica Accornero

Publikováno v: Journal of Molecular and Cellular Cardiology. 151:46-55

Regulation of gene expression plays a fundamental role in cardiac stress-responses. Modification of coding transcripts by adenosine methylation (m6A) has recently emerged as a critical post-transcriptional mechanism underlying heart disease. Thousand

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::1f2e336b94f2c0cc3c954bfc139a82e8
https://doi.org/10.1016/j.yjmcc.2020.11.002