A lipid-lowering drug is expected to treat achondroplasia

A lipid-lowering drug is expected to treat achondroplasia

September 19, 2014 Source: Xinhuanet

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Cartilage hypoplasia is the most common type of congenital dwarfism. Kyoto University in Japan has announced that its research team will use the skin cells of the patient to produce induced pluripotent stem cells (iPS cells), and then use the lipid-lowering drug statin to promote its differentiation into chondrocytes. This finding is expected to develop new methods for treating achondroplasia.

iPS cells are stem cells transformed by somatic cells treated with an inducing factor. They function similarly to embryonic stem cells and can develop into various tissues and organs.

Cartilage hypoplasia is a refractory disease, and genetic abnormalities cause cartilage to fail to develop, with one in every 20,000 to 40,000 newborns. Because it is difficult to collect cartilage from the patient, detailed investigations are not possible and there is no underlying treatment.

The research team led by Prof. Wu Mufan, a professor at the iPS Institute of Kyoto University, used iPS cells from skin cells of a newborn and two adult patients with achondroplasia and found that even if a protein is added to differentiate it into chondrocytes, Only abnormal cells can be obtained, which reproduces the characteristics of achondroplasia.

Since then, the research team has tried to add more than 10 drugs that have been reported to promote cartilage growth during the process of growing iPS cells into chondrocytes. It has been found that the lipid-lowering drug statin can most effectively promote the differentiation of iPS cells into normal. Chondrocytes.

After 3 to 2 weeks of birth in rats with achondroplasia, the team injected statin directly into the abdomen. The results showed that the cartilage of this experimental mouse also grew normally.

The team believes that achondroplasia is caused by an excessive increase in fibroblast growth factor receptor 3, a protein that breaks down cartilage, and statin can contain this protein.

The research team is preparing to conduct clinical trials 1 to 2 years later to determine the amount of medication and the method of administration. However, the research team warned that taking the statin currently on the market is not only ineffective, but also the risk of side effects, so don't take it privately. Related papers have been published in the latest issue of the British magazine Nature.

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