Cardiac and skeletal striated muscles are intricately designed machines responsible for
muscle contraction. Coordination of the basic contractile unit, the sarcomere, and the …

The heart is an extraordinarily versatile pump, finely tuned to respond to a multitude of
demands. Given the heart pumps without rest for decades its efficiency is particularly …

Many genetic diseases are caused by single-nucleotide polymorphisms. Base editors can
correct these mutations at single-nucleotide resolution, but until recently, only allowed for …

Elevated blood pressure is a major risk factor for cardiovascular disease and has a
substantial genetic contribution. Genetic variation influencing blood pressure has the …

Congenital heart disease (CHD) is the most common congenital anomaly and a major cause
of infant morbidity and mortality. While morbidity and mortality are highest in infants with …

Genetic cardiomyopathies are disorders of the cardiac muscle, most often explained by
pathogenic mutations in genes encoding sarcomere, cytoskeleton, or ion channel proteins …

We sought to establish a large animal model of inherited hypertrophic cardiomyopathy
(HCM) with sufficient disease severity and early penetrance for identification of novel …

The integration of in vitro cardiac tissue models, human induced pluripotent stem cells
(hiPSCs) and genome-editing tools allows for the enhanced interrogation of physiological …

Hypertrophic cardiomyopathy (HCM) is the most prevalent genetically inherited
cardiomyopathy that follows an autosomal dominant inheritance pattern. The majority of …

Gene therapy is a promising option for severe forms of genetic diseases. We previously
provided evidence for the feasibility of trans-splicing, exon skipping, and gene replacement …