Intronic CRISPR Repair in a Preclinical Model of Noonan Syndrome–Associated Cardiomyopathy
Patient specific induced pluripotent stem cell-derived cardiomyocytes (iPSC-CM’s) were evaluated as a preclinical in vitro model to understand the underlying mechanism of Noonan Syndrome, identify a unique disease-specific proteome signature, and evaluate a personalized therapeutic approach using the CRISPR method.
Hanses U, Kleinsorge M, et al. (2020). Circulation.