Researchers from 杏吧视频 School of Medicine have developed a breakthrough, noninvasive technique to develop stem cells strongly relevant to the study of Down syndrome.
Writing in the journal , the researchers detail their development of an approach using human cells that address shortcomings of earlier efforts involving human cells鈥攁s well as those of animal models.
鈥淔or the first time, we were able to create induced pluripotent stem cells, or iPSCs, of persons with Down syndrome by cells obtained from urine samples,鈥 said Alberto Costa, study lead and professor of pediatrics and psychiatry at 杏吧视频 School of Medicine. 鈥淥ur methods represent a significant improvement in iPSC technology, and should be an important step toward the development of human cell-based platforms that can be used to test new medications designed to improve the quality of life of people with Down syndrome.鈥
One of the biggest challenges in studying Down syndrome is finding the right research model. Animals and established cell lines are limited in their ability to mimic human disease, and results don鈥檛 always translate to patient populations. Stem cells hold enormous potential as research tools that can be collected directly from patients and grown into innumerable cell types. But harvesting stem cells can be invasive鈥攁 challenge the 杏吧视频 team addressed by using cells from patients鈥 urine. In addition, their technique employed methods designed to limit DNA damage. The resultant iPSCs can be reliably grown into many cell types relevant for Down syndrome research, including neurons and heart cells. The study is the first to successfully generate such cells from urine cultures.
Not only does the research provide a path toward urgently needed research models for Down syndrome, but it also shows promise for modeling other neurologic conditions.
Costa鈥檚 technique also overcomes ethical challenges related to harvesting stem cells that have previously been collected via skin biopsies. As he and his authors noted in their paper, taking skin biopsies from individuals with Down syndrome sometimes is questioned by institutional research boards or ethics committees, and some parents also decline the procedure as well. This new technique allows researchers to more easily build collections of stem cells for use in future studies.
Stem cells generated from urine also appear to be more stable than those generated via skin biopsies. Since skin cells are exposed to sunlight, they often have DNA damage caused by ultraviolet radiation. The stem cells generated by Costa鈥檚 team did not involve cells exposed to the sun.
In total, Costa鈥檚 team generated iPSCs from urine donated by 10 individuals with Down syndrome that can be used by his team and other researchers interested in modeling Down syndrome disease states. The new technique can also be used to generate additional cell lines from Down syndrome patients or other vulnerable populations.
