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Proteomic analysis of brain organoids from creatine transporter deficiency (CTD) patients enhance the understanding of CTD offering potential therapeutic targets and a robust foundation for continued research in the field.

Genes of the MICOS complex are a major candidate pathway to be potentially linked to defects in cardiomyocyte growth and differentiation in Hypoplastic left heart syndrome (HLHS), and relevant for elucidating the polygenic nature of HLHS and other CHDs.

Chondrocytes derived from induced pluripotent stem cells with the dysplasia-causing TRPV4 mutations, V620I and T89I mutation, were resistant to BMP4-induced hypertrophy, suggesting a mechanism underlying the effects of TRPV4 dysfunction on the severity of skeletal dysplasia.

Diversification of human pacemaker subpopulations is directed by WNT and TGFβ signaling.

A 7-dehydrocholesterol-derived oxysterol in SLOS was found to profoundly impact neurogenesis during cortical development by interacting with glucocorticoid receptor, which points to new therapeutic approaches for SLOS by targeting the activities of this oxysterol.

Important mechanistic details on Duchenne Muscular Dystrophy arrhythmogenesis are revealed, providing a crucial lead for investigators interested in developing therapeutic solutions for this deadly disease.

iMyoblasts, a novel iPS-derived PAX3 muscle stem cell for gene editing, muscle engraftment, and stem cell therapeutics to treat muscle injury and muscular dystrophies.

Extracellular vesicles are proposed as novel, non-cell-autonomous mediators of neuronal atrophy in Parkinson's disease.

An integrated stem cell-based disease modeling and computational approach demonstrates how proximal airway epithelium is critical for SARS-CoV-2 infectivity and distal alveolar cells are critical for simulating the host responses.

Human liver organoids provide a patient-derived platform to interrogate host and viral mechanisms of HBV replication, perform anti-HBV and toxicity drug screens, and investigate the molecular determinants of related tumorigenesis.