Cardiac Cell and Gene Transfer
Principles, Protocols, and Applications
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Open AccessInducing positive inotropy in human iPSC-derived cardiac muscle by gene editing-based activation of the cardiac α-myosin heavy chain
Human induced pluripotent stem cells and their differentiation into cardiac myocytes (hiPSC-CMs) provides a unique and valuable platform for studies of cardiac muscle structure–function. This includes studies ...
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Open AccessRapid restitution of contractile dysfunction by synthetic copolymers in dystrophin-deficient single live skeletal muscle fibers
Duchenne muscular dystrophy (DMD) is caused by the lack of dystrophin, a cytoskeletal protein essential for the preservation of the structural integrity of the muscle cell membrane. DMD patients develop severe...
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Open AccessSarcomere dynamics revealed by a myofilament integrated FRET-based biosensor in live skeletal muscle fibers
The sarcomere is the functional unit of skeletal muscle, essential for proper contraction. Numerous acquired and inherited myopathies impact sarcomere function causing clinically significant disease. Mechanist...
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Open AccessCardiac myocyte intrinsic contractility and calcium handling deficits underlie heart organ dysfunction in murine cancer cachexia
Cachexia is a muscle wasting syndrome occurring in many advanced cancer patients. Cachexia significantly increases cancer morbidity and mortality. Cardiac atrophy and contractility deficits have been observed ...
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Open AccessInvestigations of an inducible intact dystrophin gene excision system in cardiac and skeletal muscle in vivo
We sought here to induce the excision of a large intragenic segment within the intact dystrophin gene locus, with the ultimate goal to elucidate dystrophin protein function and stability in striated muscles in...
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Protocol
Gene Transfer of Calcium-Binding Proteins into Adult Cardiac Myocytes
Heart failure is the leading cause of combined morbidity and mortality in the USA with 50% of cases being diastolic heart failure. Diastolic heart failure results from poor myocardial relaxation and inadequate...
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Open AccessMuscle membrane integrity in Duchenne muscular dystrophy: recent advances in copolymer-based muscle membrane stabilizers
The scientific premise, design, and structure-function analysis of chemical-based muscle membrane stabilizing block copolymers are reviewed here for applications in striated muscle membrane injury. Synthetic b...
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Article
Open AccessMolecular inotropy mediated by cardiac miR-based PDE4D/PRKAR1α/phosphoprotein signaling
Molecular inotropy refers to cardiac contractility that can be modified to affect overall heart pump performance. Here we show evidence of a new molecular pathway for positive inotropy by a cardiac-restricted ...
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Standard Operating Procedures (SOPs) for Evaluating the Heart in Preclinical Studies of Duchenne Muscular Dystrophy
A recent working group meeting focused on contemporary cardiac issues in Duchenne muscular dystrophy (DMD) was hosted by the National Heart, Lung, and Blood Institute in collaboration with the Parent Project M...
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Ca2+ Regulation of the Cardiac Thin Filament
The physiological role of calcium in a cardiac myocyte is demonstrated in the coupling of excitation and contraction. Small extracellular calcium signals are amplified in the cardiac myocyte through calcium re...
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Noncanonical EF-hand motif strategically delays Ca2+ buffering to enhance cardiac performance
The inability of the heart to efficiently relax as it beats, termed diastolic dysfunction, is a major underlying cause of heart disease. As a new strategy for treating diastolic dysfunction, Joseph Metzger and...
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Molecular Cardiology in Translation: Gene, Cell and Chemical-Based Experimental Therapeutics for the Failing Heart
Acquired and inherited diseases of the heart represent a major health care issue in this country and throughout the World. Clinical medicine has made important advancements in the past quarter century to enabl...
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Parvalbumin Isoforms for Enhancing Cardiac Diastolic Function
Diastolic heart failure (DHF), characterized by depressed myocardial relaxation performance and poor ventricular filling, is a distinct form of heart failure accounting for nearly half of the heart failure pat...
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Tuning cardiac performance in ischemic heart disease and failure by modulating myofilament function
The cardiac myofilaments are composed of highly ordered arrays of proteins that coordinate cardiac contraction and relaxation in response to the rhythmic waves of [Ca2+] during the cardiac cycle. Several cardiac ...
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Histidine button engineered into cardiac troponin I protects the ischemic and failing heart
The myofilament protein troponin I (TnI) has a key isoform-dependent role in the development of contractile failure during acidosis and ischemia. Here we show that cardiac performance in vitro and in vivo is enha...
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Article
Dystrophic heart failure blocked by membrane sealant poloxamer
Heart failure accounts for at least 15% of deaths in Duchenne muscular dystrophy, and as palliative treatments for the skeletal muscle aspects of the disease improve, this proportion is rising. Experiments on ...
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An integrative genomics approach to infer causal associations between gene expression and disease
A key goal of biomedical research is to elucidate the complex network of gene interactions underlying complex traits such as common human diseases. Here we detail a multistep procedure for identifying potentia...
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Book
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Protocol
Adenoviral Vectors
Current methodologies in first-generation adenoviral gene transfer, however novel their approach to vector delivery, are ultimately limited by the purity of the vector being delivered. Purity in this case is d...
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Protocol
Direct Gene Transfer to the Adult Rodent Myocardium In Vivo
The objective of in vivo gene transfer is the expression of a specific gene product into a target cell population with the general intention of not altering other cell populations. Because of their ability to ...
