[HTML][HTML] A guide for the diagnosis of rare and undiagnosed disease: beyond the exome

S Marwaha, JW Knowles, EA Ashley - Genome medicine, 2022 - Springer
Rare diseases affect 30 million people in the USA and more than 300–400 million
worldwide, often causing chronic illness, disability, and premature death. Traditional …

Congenital long QT syndrome

AD Krahn, Z Laksman, RW Sy, PG Postema… - Clinical …, 2022 - jacc.org
Congenital long QT syndrome (LQTS) encompasses a group of heritable conditions that are
associated with cardiac repolarization dysfunction. Since its initial description in 1957, our …

Identification of drug transporter genomic variants and inhibitors that protect against doxorubicin-induced cardiotoxicity

T Magdy, M Jouni, HH Kuo, CJ Weddle, D Lyra-Leite… - Circulation, 2022 - Am Heart Assoc
Background: Multiple pharmacogenomic studies have identified the synonymous genomic
variant rs7853758 (G> A, L461L) and the intronic variant rs885004 in SLC28A3 (solute …

[HTML][HTML] Long QT syndrome: genetics and future perspective

E Wallace, L Howard, M Liu, T O'Brien, D Ward… - Pediatric …, 2019 - Springer
Long QT syndrome (LQTS) is an inherited primary arrhythmia syndrome that may present
with malignant arrhythmia and, rarely, risk of sudden death. The clinical symptoms include …

[HTML][HTML] Human iPSC-derived cardiomyocytes cultured in 3D engineered heart tissue show physiological upstroke velocity and sodium current density

MD Lemoine, I Mannhardt, K Breckwoldt… - Scientific reports, 2017 - nature.com
Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CM) are a promising
tool for drug testing and modelling genetic disorders. Abnormally low upstroke velocity is a …

[HTML][HTML] Human engineered heart tissue models for disease modeling and drug discovery

H Tani, S Tohyama - Frontiers in Cell and Developmental Biology, 2022 - frontiersin.org
The emergence of human induced pluripotent stem cells (hiPSCs) and efficient
differentiation of hiPSC-derived cardiomyocytes (hiPSC-CMs) induced from diseased …

Human induced pluripotent stem cell–derived cardiomyocytes as models for cardiac channelopathies: a primer for non-electrophysiologists

P Garg, V Garg, R Shrestha, MC Sanguinetti… - Circulation …, 2018 - Am Heart Assoc
Life threatening ventricular arrhythmias leading to sudden cardiac death are a major cause
of morbidity and mortality. In the absence of structural heart disease, these arrhythmias …

Integrating cardiomyocytes from human pluripotent stem cells in safety pharmacology: has the time come?

L Sala, M Bellin, CL Mummery - British journal of pharmacology, 2017 - Wiley Online Library
Cardiotoxicity is a severe side effect of drugs that induce structural or electrophysiological
changes in heart muscle cells. As a result, the heart undergoes failure and potentially lethal …

Human induced pluripotent stem cell (hiPSC)-derived cells to assess drug cardiotoxicity: opportunities and problems

T Magdy, AJT Schuldt, JC Wu… - Annual review of …, 2018 - annualreviews.org
Billions of US dollars are invested every year by the pharmaceutical industry in drug
development, with the aim of introducing new drugs that are effective and have minimal side …

[HTML][HTML] Modeling cardiac disease mechanisms using induced pluripotent stem cell-derived cardiomyocytes: progress, promises and challenges

EI Parrotta, V Lucchino, L Scaramuzzino… - International Journal of …, 2020 - mdpi.com
Cardiovascular diseases (CVDs) are a class of disorders affecting the heart or blood
vessels. Despite progress in clinical research and therapy, CVDs still represent the leading …