VIDEO: Scientists closer to cure for childhood sudden death

A stem-cell research breakthrough has brought Irish scientists a step closer to developing treatments for heart disease and sudden death in young children.

VIDEO: Scientists closer to cure for childhood sudden death

Biomedical researchers at NUI Galway, in collaboration with clinicians from Our Lady’s Children Hospital, in Dublin, have developed synchronised beating-heart cells from a skin biopsy.

The stem-cell study was initiated by Prof Timothy O’Brien, director of the Regenerative Medicine Institute (REMEDI) at NUI Galway. “We are excited about the potential to develop new therapies for children at risk of sudden death, using this technology,” said Prof O’Brien.

The researchers in NUI Galway have developed a highly skilled and technically specialised expertise in ‘re-programming’ skin cells into stem cells that become heart cells.

Their goal is to repair a genetic defect in affected heart cells by using advanced technology.

The researchers, who are funded by REMEDI and the National Children’s Research Centre, now want to test therapies that may reduce the risk of sudden death in survivors and relatives.

The scientists can generate patient-specific heart tissue on a laboratory dish to test new treatments, without placing the patient at risk.

NUI Galway wants families affected by an inherited rhythm disease, known as Long QT Syndrome, to provide a skin biopsy, in consultation with their doctor.

New medicines can be tested on the cells for their effectiveness in preventing arrhythmias.

Also, the genetic defect in the heart cell can be repaired through genome editing, and can then be compared with the diseased cell in the laboratory.

The research was carried out by Sanbing Shen, professor of fundamental stem-cell biology, and post-doctoral researcher, Dr Min Liu, in the Biomedical Sciences Building at NUI Galway.

The project is being driven by Dr Terry Prendiville, from the Department of Paediatric Cardiology at Our Lady’s Children’s Hospital.

“Research such as this allows us to continue to push the envelope on developing medical therapies that safeguard against risk of sudden death,” said Dr Prendiville.

Families being selected at this early stage of research have clearly abnormal electrocardiograms that are associated with a risk of sudden death, and a known, causative gene mutation.

Those with a diagnosis of Long QT syndrome, with a known gene mutation, can contact Dr Prendiville, at Our Lady’s Children’s Hospital, for more information and eligibility criteria.

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