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barbara
08-31-2011, 11:58 AM
Hair Cell-Derived Patient-Specific Heart Cells For Disease Modeling And Drug Screening

Article Date: 30 Aug 2011


Hair follicle keratinocytes offer a simple and accessible route to generate patient-specific induced pluripotent stem cells, iPSCs, with minimum inconvenience for the patients, shows a study presented at the ESC Congress 2011. The study presented by Dr. Katrin Streckfuss-Boemeke from Germany, won the ESC Basic Science Young Investigators Award.

"Data gathered in this study demonstrates an easy and fast possibility to generate iPSCs from hair follicles of patients with genetic cardiac diseases and their further differentiation into functional cardiomyocytes. These cells will allow us to model the heart disease of these patients, to investigate the mechanisms of the disease, to perform drug screenings and to develop patient-specific therapeutic strategies," explained Dr Streckfuss-Boemeke.

Most iPSC described in previous studies were generated from skin fibroblasts or bone marrow cells which require surgical intervention. The aim of this study was to use an alternative cell source that could be readily and noninvasively isolated from patients and which shows a high proliferation rate for efficient reprogramming.

Heart disease is one of the leading causes of death in developed countries. Although the majority of cardiac death victims are elderly, many children and young adults under the age of 35 die each year due to various cardiac pathologies. Recent progress in the fields of molecular biology and human genetics have enabled identification of the genetic causes of many heart diseases, including multiple causes of sudden cardiac death (SCD).

Genetically determined cardiac diseases can be divided into two groups : diseases with a structural change of the heart, like hypertrophic cardiomyopathy (HCM) or arrhythmogenic right ventricular dysplasia (ARVD) and a second group, without structural changes, resulting in life-threatening cardiac arrhythmia. Examples are primary electric heart diseases (the channelopathies), as the long QT syndrome (LQTS), the short QT syndrome (SQTS), the Brugada syndrome (BrS) and the Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), which causes sudden unexpected cardiac death in apparently healthy young individuals.

Several genetic cardiac diseases are not rare, for example HCM (although it is most common cause of SCD in childhood), LQTS and BrS. However, several diseases are rare, for example, CPVT and ARVD, and are very often related to high mortality in children and young adults. The cumulative mortality of the CPVT cases is 30-50% by the age of 20-30 years.

Until now, most of studies of human cardiac developmental defects or adult diseases have been done in animal models, especially in mice. However, mice differ from humans in many ways; for example, a mouse heart beats at 500 beats per minute, while the human heart beats at 70 beats per min. Therefore, the use of pluripotent stem cells is a promising approach for studying cardiac diseases.