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iPS Cells “Make a Mouse”: Progress Toward Cell-Based Therapies?

By July 23, 2009December 18th, 2023Science & Research

Two groups of scientists in China have reported this week in Nature and Cell Stem Cell* that a fibroblast, a cell type that can be found in adult skin, can be genetically reprogrammed to a stem cell-like state, and can generate newborn live mice when implanted into a surrogate mom. This advances a new line of research where adult cells such as fibroblasts can be reconfigured into what are called induced pluripotent or iPS cells.

This is important news for developing treatments for conditions that might benefit from cell replacement, such as diabetes or Parkinson’s disease – as well as potentially for neurofibromatosis (see below). If iPS cells have at least some of the capabilities of embryonic stem cells they can be used for medical research instead of embryonic stem cells, which are subject to ethical concerns.

This research is in early days: only 3.5% of the Chinese experiments produced live mice and many were born with abnormalities and died after a couple of days. However a small number of mice survived, thrived, and have gone on to have ‘children’ and ‘grandchildren’, all of whom appear normal so far.

This is the first indication that iPS cells might truly have as much potential as embryonic stem cells in creating any cell type of the body, and therefore have real clinical applications to benefit a variety of medical conditions. We are excited to see this progress as the Children’s Tumor Foundation is currently funding a Drug Discovery Initiative Award to see if iPS cells from mice with NF1 plexiform tumors can be reprogrammed and transplanted into bone marrow, with a goal of generating circulating healthy mast cells that will ‘attack’ and arrest growth of the plexiform tumor.