Cardiomyopathies are among the major triggers of heart failure, but their clinical and genetic
complexity have hampered our understanding of these disorders and delayed the …

Human pluripotent stem cells (hPSCs) are uniquely suited to study human development and
disease and promise to revolutionize regenerative medicine. These applications rely on …

Long QT syndrome (LQTS) is a cardiovascular disease characterized by QT interval
prolongation that can lead to sudden cardiac death. Many mutations with heterogeneous …

Cortical excitability is commonly measured by applying magnetic stimulation in combination
with measuring behavioral response. This measure has, however, some shortcomings …

Prime editing technologies enable precise genome editing without the caveats of CRISPR
nuclease-based methods. Nonetheless, current approaches to identify and isolate prime …

Advancements in genome editing make possible to exploit the functions of enzymes for
efficient DNA modifications with tremendous potential to treat human genetic diseases …

Stem cells such as induced pluripotent stem cells, embryonic stem cells, and hematopoietic
stem and progenitor cells are growing in importance in disease modeling and regenerative …

Base editing has emerged as a revolutionary technology for single nucleotide modifications.
The cytosine and adenine base editors (CBEs and ABEs) have demonstrated great potential …

LMNA-related Congenital Muscular Dystrophy (L-CMD) is a rare disease caused by
mutations in the LMNA gene, encoding nuclear lamina proteins. The most frequent mutation …

Cardiovascular disease (CVD) remains the leading cause of mortality, resulting in 1 out of 4
deaths in the United States at the alarming rate of 1 death every 36 seconds, despite great …