Sangamo’s Zinc Finger Nuclease Technology Used To Efficiently Modify Human Stem Cells

Zinc finger nucleases (ZFNs), designed by Sangamo, is used by scientists to efficiently and precisely modify the genomes of human embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs).

The company said, 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 the scientific journal Nature Biotechnology.

Rudolf Jaenisch, member of the Whitehead Institute and professor of biology at Massachusetts Institute of Technology, said: “The application of ZFN technology to human stem cells opens a new phase in human genetics. 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.”

Philip Gregory, chief scientific officer and vice president of research at Sangamo, said: “These data are another powerful demonstration of the specificity and broad applicability of Sangamo’s ZFP technology across medically and commercially relevant cell types. The ability to efficiently modify stem cells enables the generation of valuable new tools for drug screening and the study of human disease as well as therapeutic applications in regenerative medicine.”