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Translating the lessons from gene therapy to the development of regenerative medicine.

M. Porteus

Published 2011 in Molecular Therapy

ABSTRACT

One of the more exciting advances in the past decade has been the surprising discovery by Takahashi and Yamanaka that primary somatic cells can be converted into pluripotent cells (induced pluripotent stem (iPS) cells) by the forced but transient expression of a small number of defined transcription factors, a discovery that may well prove worthy of a Nobel Prize.1 While there is significant excitement about the study and the therapeutic potential of human embryonic stem (ES) cells, the ethical issues surrounding the destruction of embryos that is necessary to generate such cells has slowed scientific investigation into their use. Because iPS cells are generated without the need to destroy an embryo, their discovery has further energized the field of regenerative medicine and stem cell biology. Indeed, there are clear similarities between the excitement generated by iPS cells and regenerative medicine today and that generated by the advent of gene therapy several decades ago. One hopes that the lessons learned from the growing pains experienced by the field of gene therapy will be applied by the leaders of this new field so as to hasten and facilitate the clinical translation of safe iPS cell technology. In this Commentary, I focus on several areas where these lessons can be applied to the iPS cell field.

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