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RNA therapy for genetic heart failure moves closer to patients after lab gains
Using patient-derived cardiac tissue and stem cell-based models, the team of translational researchers demonstrated that targeting the genetic cause of disease improved cellular abnormalities and identified the biological pathways involved.
Summary
Using patient-derived cardiac tissue and stem cell-based models, the team of translational researchers demonstrated that targeting the genetic cause of disease improved cellular abnormalities and identified the biological pathways involved. The results of this study were published in Signal Transduction and Targeted Therapy . PLN R14del is a Dutch founder pathogenic variant that originated in the province of Friesland several centuries ago and is therefore particularly common in the north of the Netherlands.
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