Induced pluripotent stem cells are derived from the donated skin or blood cells of adults and, with the reactivation of four genes, are reprogrammed back to an embryonic stem cell-like state. Like embryonic stem cells, iPS cells can be differentiated toward any cell type in the body, but they do not require the use of embryos.
Since the original discovery by Nobel Prize Awardee Shinya Yamanaka, our knowledge about iPS cells has exponentially expanded.
What can we learn from iPS cells?
As an indefinitely reproducing cell type that mimics an embryonic state in the dish, iPS cells can help us understand molecular processes in development without the use of embryos. Research in developmental biology is a particular focus of CReM investigators, motivated by the philosophy that understanding how tissues form during embryonic development is the key to understanding stem cell biology.
However, the primary goal of the CReM is clinical application. Once fully understood, iPS cells have immense potential to benefit patients with inherited diseases such as cystic fibrosis, alpha-1 anti-trypsin deficiency, sickle-cell anemia, and other disease targets. Many of these inherited conditions are caused by only a single mutation in the patient’s genome. Current research in the CReM focuses on technology to correct these mutations in iPS cells from indiciduals with these illnesses and to subsequently differentiate them toward tissues of interest that could be used in the future for cell replacement therapies. Because the replacement tissues would originate with the patient in need, rejection by the patient’s immune system is virtually impossible.
An additional research focus of the CReM was born when a renowned research program in Biomedical Engineering at Boston University joined stem cell biologists on campus to focus on key issues limiting the delivery of therapeutic stem cells into the body.