Novel RNA based therapies for treatment-resistant forms of severe cardiomyopathy caused by LMNA mutations


 Yigal Pinto
University of Amsterdam, Amsterdam, The Netherlands

Gisèle Bonne, INSERM, Paris, France
Lior Gepstein Technion, Institute of Technology, Haifa, Israel
Thomas Eschenhagen and Lucie Carrier University Medical Center Hamburg, Hamburg, Germany

Research focus:
Some forms of heart failure are often very difficult to treat. Patients who have a mutation in Lamin A/C can develop a form of heart disease that often does not respond well enough to existing therapies. VARIATION aims to develop a new technology to inhibit mutated Lamin A to halt the disease. The project will test this new RNA based technology in human cell that are derived from stem cells obtained from patients that actually carry the disease. VARIATION is expected to provide the first steps towards a treatment that is applicable to most patients that carry such a mutation and can halt progression of the associated heart failure.