Cardiovascular Disease in Duchenne Muscular Dystrophy: Overview and Insight Into Novel Therapeutic Targets.
JACC Basic Transl Sci
; 7(6): 608-625, 2022 Jun.
Article
em En
| MEDLINE
| ID: mdl-35818510
ABSTRACT
Duchenne muscular dystrophy (DMD) is a devastating disease affecting approximately 1 in every 3,500 male births worldwide. Multiple mutations in the dystrophin gene have been implicated as underlying causes of DMD. However, there remains no cure for patients with DMD, and cardiomyopathy has become the most common cause of death in the affected population. Extensive research is under way investigating molecular mechanisms that highlight potential therapeutic targets for the development of pharmacotherapy for DMD cardiomyopathy. In this paper, the authors perform a literature review reporting on recent ongoing efforts to identify novel therapeutic strategies to reduce, prevent, or reverse progression of cardiac dysfunction in DMD.
ACE, angiotensin-converting enzyme; ARB, angiotensin receptor blocker; ApN, adiponectin; BB, beta-blocker; BDNF, brain-derived neurotrophic factor; CMR, cardiac magnetic resonance imaging; Cx, connexin; DMD, Duchenne muscular dystrophy; DPC, dystrophin-associated protein complex; Duchenne muscular dystrophy; FFA, free fatty acid; HF, heart failure; LNP, lipid nanoparticle; LV, left ventricular; LVEF, left ventricular ejection fraction; NIV, noninvasive ventilation; Nrf2, nuclear factor erythroid 2-related factor 2; PKA, protein kinase A; PTX3, pentraxin 3; Px, pannexin; RNP, ribonucleoprotein complexes; RT-qPCR, reverse transcription-quantitative polymerase chain reaction; RyR2, ryanodine receptor isoform 2; SR, sarcoplasmic reticulum; TRPV2, transient receptor potential cation channel, subfamily V, member 2; TrkB, tyrosine kinase B; arrhythmias; cardiomyopathy; inflammatory modulators; miR, microRNA; myocardial fibrosis; sgRNA, single guide RNA
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Ano de publicação:
2022
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Article