StemCell Therapy

Australian researchers have successfully turned stem cells from Friedreichs ataxia (FA) patients into heart cells to study molecular anomalies that maycontribute to this disease.

Theseheart cells in a dish provide valuable information for the design of novel treatments.

Their study, Friedreichs ataxia induced pluripotent stem cell-derived cardiomyocytes display electrophysiological abnormalities and calcium handling deficiency. appearedin the journal Aging.

FAis caused by low levels of the frataxin protein due to anomalies in the gene sequence encoding this protein repeats of DNA portions within the gene. The higher the number of repeats, the sooner the onset of FAand its associated complications.

Frataxin plays an important role in the mitochondria, the cells powerhouse, so the mutated protein accounts for several symptoms that reflect deficiencies in energy production. The heart is one of the organs affected by this lack of energy.

Cardiomyopathy is detected in two-thirds of individuals with FRDA[Friedreichs ataxia], researchers wrote. Individuals with FRDA generally present with progressive cardiomyopathy of the left ventricle, which is the leading cause of death in FRDA due to arrhythmias and/or heart failure.

Previous studies have shown that death of heart cells, or cardiomyocytes, and fibrosis may contribute to heart complications in FA, but little is known about the diseases impact on the heart.

Researchers generated stem cell cultures using cells from three FA patients with heart complications. They then stimulated the development of these stem cells into cardiomyocytes basically, heart cells in a dish.

The new cardiomyocytes had low levels of frataxin, as expected, but alsoabnormal ionic currents, which are crucial for the normal functioning of these cells. They also had morevariation in their beating rates, which was linked todeficient calcium control, ultimately affecting howthe cardiomyocytes work.

Together, these results pave the way for understanding how FA patients develop abnormal heart activity as well as theuse of induced stem cells to studycardiomyopathy within the context of this disease.

Importantly, our data clearly indicates that FRDA iPSC [stem cells]- derivedcardiomyocytes can be used for screening of compounds able to alter or reverse phenotypes, in human cells, hence providing a novel and unique tool for FRDAresearch, researchers concluded.

More here:
Researchers Create 'Heart Cells in a Dish' to Study FA Heart Disease - Friedreich's Ataxia News

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