Thu, Jun 27, 2013 - Page 3 News List

Team generates first-ever iPSC for Pompe disease

GROUNDBREAKING:Researchers have generated pluripotent stem cells from patients’ fibroblasts by applying a temporary genetic rescue method

By Lee I-chia  /  Staff reporter

A research team from National Taiwan University (NTU) and Academia Sinica yesterday said it has produced the world’s first induced pluripotent stem cells (iPSC) for Pompe disease.

Pompe disease is a rare genetic disease and metabolic disorder caused by acid maltase deficiency, and the incidence rate in Taiwan is slightly higher than in many other countries, the National Science Council said.

Current treatment through gene recombination has its limits and patients still have to undergo painful treatment, it added.

The council-funded research team said it has successfully generated Pompe disease-iPSC from the fibroblasts — a type of cell — of patients, by applying a temporary genetic rescue method, becoming the first Pompe disease-iPSC recognized by the international medical society.

The team said the Pompe disease-iPSC can authentically reproduce the cell phenotype and pathological features of Pompe disease, and it is using the Pompe disease-iPSC to screen for compounds that can reduce apoptosis, or programmed cell death, that can be used to produce medicine.

The council said results in the field of stem cell research has good potential for further development in biotechnology and medicine.

Citing Shinya Yamanaka, the winner of the Nobel Prize in Physiology or Medicine last year, as an example, the council said the award was a recognition of the Japanese physician and researcher’s discovery that mature cells can be reprogrammed and shows the significance of stem cell technology in improving human health.

The team from the university has also successfully produced iPSC for premature ovarian failure in the past, and is trying to use the skills it developed to help produce a cure or treatment for other diseases.

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