Pluripotent Stem Cells 101

Pluripotent Stem Cell Research at Children's

The Stem Cell Program at Children's Hospital Boston is pursuing several approaches to creating pluripotent stem cells in parallel: using embryos from in vitro fertilization (IVF) donated under strict ethical criteria, using nuclear transfer and parthenogenesis to create embryonic stem cells (ES cells) and genetic reprogramming of skin cells and other body cells to create induced pluripotent stem cells (iPS cells).

Each method has its advantages and disadvantages, but by studying them all, scientists can get the greatest understanding of how stem cells work and how to maximize their treatment potential. Researchers are also examining potential safety issues with using the different kinds of pluripotent stem cells and seeking ways to address them.

Recently, the Daley Lab and collaborators won a $1.7 million National Institutes of Health grant to do a comprehensive comparison of the properties of iPS cells and ES cells derived from various sources. One goal of this project is to determine whether iPS cells are functionally equivalent to ES cells.

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Recent advances:

Eleven new ES cell lines created at Children’s Hospital Boston were among the first 13 new lines to become eligible for federal research funding in December 2009. All were created through the donation and use of poor-quality embryos that are typically discarded as part of the IVF process. The Stem Cell Program is making these lines available to scientists around the world.

The Daley Lab was one of the first three labs in the world to successfully create human iPS cells through genetic reprogramming techniques, an accomplishment cited by the journal Science in its 2008 Breakthrough of the Year issue. The lab then created the first repository of iPS cells from patients with specific diseases.

These new iPS lines, developed from the cells of patients ranging in age from one month to 57 years old, with disorders ranging from diabetes to Parkinson disease, have been deposited in a new core facility established by the Harvard Stem Cell Institute. Many are under active study at Children’s, and are already beginning to yield clues about how diseases unfold in their earliest stages. Some of these studies are beginning to suggest a path to therapy. Children’s researchers are also actively working to refine techniques for making iPS cells and to create additional lines specific to other diseases.

Giving

Spotlight

  • Disease in a dish

    Although actual treatments from pluripotent stem cells won’t happen immediately, induced pluripotent stem cells (iPS cells) are already being used to make new discoveries about disease, from premature aging to congenital heart disease to cancer, and embryonic stem cells have yielded new insights into Fanconi anemia. For more, click “Research on Diseases” at the top of this page.

  • Children’s Hospital Boston: A leader in blood research

    The Stem Cell Program at Children’s Hospital Boston’s efforts to use pluripotent
    stem cells therapeutically will likely begin in blood diseases, for which the use of stem cells is well-established: the first successful human bone marrow transplant was in 1968. Children’s has a long legacy of accomplishment in blood and cancer research with one of the oldest programs in the country. Read more.

  • The stem cell-cancer connection

    Stem cell generation and carcinogenesis are known to be closely related. Richard Gregory, PhD, in the Stem Cell Program at Children’s Hospital Boston, is studying the nature of this link, and uncovering new ways to enhance stem cell creation as well as inhibit cancer. Several other research groups at Children’s are probing the deadly ways of cancer stem cells, believing that they may lie at the heart of a cancer’s virulence.

  • Quality control

    The production ofinduced pluripotent stem cells (iPS cells) is often imprecise, yielding many incompletely reprogrammed cells. Thorsten Schlaeger, PhD, of Children’s Stem Cell Program has developed a battery of tests to ensure that he has the real thing: pure pluripotent stem cells. Schlaeger’s work creates a standard of analysis in the field. Read more and click here to see image.