WIKIMEDIA, GEORGE SHUKLINStatins may aid in bone growth in people with dwarfism caused by mutations in the gene FGFR3, according to a study published today (September 17) in Nature. Researchers identified the cholesterol-lowering drug’s growth-stimulating properties during a screen using induced pluripotent stem cells (iPSCs) derived from patients’ skin cells that were differentiated into cartilage-producing cells called chondrocytes. The patients had the most common form of dwarfism, achondroplasia, as well as a rarer disorder, called thanatophoric dysplasia type 1 (TD1), which is typically fatal early in life.
Obtaining chondrocytes from patients is an invasive process that is rarely done, explained coauthor Noriyuki Tsumaki of Kyoto University in Japan. This makes it difficult to assess how FGFR3 mutations function in human cells and what might be done to treat people with these abnormalities.
“The beauty of iPS cells is that any cell type can be used from the patient, including those that do not require invasive measures,” Tsumaki wrote in an e-mail to The Scientist. “These cells are then reprogrammed into chrondocytes, vastly expanding the number of cells for study.”
“They definitely show that the cellular model is really working and it’s very interesting to have that,” said Elvire Gouze, who studies achondroplasia at the French Institute of Health and Medical Research (INSERM). ...
