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New stem cell pathway discovered in research on rare genetic disease.

SAN FRANCISCO, Calif., October 24, 2016 -- Researchers at the Gladstone Institutes have found a way to increase the efficiency of stem cell reprogramming through research on a rare genetic disease.

Induced pluripotent stem (iPS) cells created from skin cells have contributed to breakthroughs in regenerative medicine and drug discovery, surpassing what researchers once thought possible.

But like any new technology, there is room for improvement. For example, using existing techniques, fewer than one percent of adult skin cells are reprogrammed into iPSCs.

"Inefficiency in creating iPSCs is a major roadblock toward applying this technology to biomedicine," said Shinya Yamanaka, who won the Nobel Prize as the first to create iPSCs.

But his new study identified a surprising way to increase the number of iPSCs that can be generated.

The scientists started with a very different goal: to create a cellular model to study fibrodysplasia ossificans progressiva (FOP).

This extremely rare genetic disease causes muscle, tendons, and ligaments to turn into bone, earning it the nickname "stone man syndrome."

It is caused by a mutation in the ACVR1 gene, which over-activates a cellular signaling process important for embryo development and involves a protein called BMP.

The scientists discovered that they could create more iPSCs from cells taken from FOP patients than those taken from healthy individuals.

They believe this is because BMP signaling enhances a cell's ability to replicate and keeps it in a pluripotent state, able to turn into any type of cell. These are key characteristics of stem cells.

To confirm their hunch, the researchers prevented BMP signaling, which resulted in fewer iPSCs being generated from FOP patients' cells. Activating the signaling pathway yielded more iPSCs.

Originally, the researchers wanted to establish a disease model for FOP that might help us understand how specific gene mutations affect bone formation.

They learned, however, that cells from patients with FOP reprogrammed much more efficiently than cells from healthy patients. This may be because the same pathway that causes bone cells to proliferate also helps stem cells to regenerate.

This is the first reported case showing that a naturally occurring genetic mutation improves the efficiency of iPSC generation. Creating iPSCs from patient cells carrying genetic mutations is useful for disease modeling, and can offer new insights into the reprogramming process, according to the researchers.

Citation: Cody Kime et al., "Autotaxin-mediated lipid signaling intersects with LIF and BMP signaling to promote the naive pluripotency transcription factor program," PNAS 2016 113 (44) 12478-12483; published ahead of print October 13, 2016, DOI:10.1073/pnas.1608564113

Abstract/Article: http://bit.ly/2fgvEWH

Contact: Kiichiro Tomoda, kiichiro.tomoda@gladstone.ucsf.edu

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Title Annotation:Basic Research
Publication:Stem Cell Research News
Date:Nov 7, 2016
Words:435
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