Marfan syndrome is a connective tissue disorder affecting the cardiovascular, skeletal, and ocular systems. Here, we generated and characterized induced pluripotent stem cell (iPSC) lines derived from two Marfan syndrome patients with mutations in the FBN1 gene (c.3333C > A and c.8854_8562delinsTATCAC). Both lines exhibited typical iPSC morphology, normal karyotype, undifferentiated states, and trilineage differentiation capacity. These iPSCs serve to enable investigation into the mechanisms underlying Marfan syndrome for therapeutic discovery.
Byron W H Mui, M. Chorsi, Christopher D. Yan et al.· Stem Cell Research· 0 citations
The pathophysiological rationale for cell-based therapy in heart failure with reduced ejection fraction is summarized, and the preclinical foundations of distinct hPSC-derived product formats are examined, including cell suspensions, epicardial sheets, engineered heart muscle, and cardiac spheroids, each with distinct tradeoffs and translational considerations.
Byron W H Mui, E. Neofytou, Joseph C. Wu· Cell Reports Medicine· 0 citations
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