Scientists from The Whitehead Institute used zinc finger nucleases (ZFNs) designed by Sangamo BioSciences, Inc., (Nasdaq: SGMO) to efficiently and precisely modify the genomes of human embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs). Human ESCs and iPSCs are useful tools in drug discovery and development. Scientists also hope to use these cells therapeutically in transplantation medicine and other regenerative applications. The research was described in a paper which appears in today's issue of the scientific journal Nature Biotechnology.
"The application of ZFN technology to human stem cells opens a new phase in human genetics," said Rudolf Jaenisch, M.D., a member of the Whitehead Institute and Professor of Biology at Massachusetts Institute of Technology (MIT). "In contrast to mouse stem cells which have been easy to modify, it has been very difficult and time-consuming to modify genes in human ESCs and iPSCs. This has severely limited their usefulness for the study of cell differentiation and as models for human disease. The work that our team published in Nature Biotechnology demonstrates that ZFNs enable new, rapid, efficient and specific methods to work with stem cells giving researchers the tools to gain valuable insights into how embryonic stem cells differentiate into adult cells and enabling the generation of patient-specific models of human disease."
Stem cells differ from other cell types in two fundamental ways. First, they are unspecialized cells capable of renewing themselves through cell division. Second, under certain conditions, they can be induced to become tissue- or organ-specific cells with special functions. Stem cells have the potential to develop into the body's many different cell types and in many tissues they serve a repair function, differentiating and replacing damaged cells. iPSCs are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state. An advantage of iPSCs is that they can be derived from the adult cells of a person and will possess the same genetic background as that individual.