Stem Cell Program

Stem Cell Research | Blood Disorders

Pluripotent stem cell research

The Stem Cell Program at Boston Children's 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 in order 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.

Recent advances:

Eleven new ES cell lines created at Boston Children's 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.

The commitment and compassion with which we care for all children and families is matched only by the pioneering spirit of discovery and innovation that drives us to think differently, to find answers, and to build a better tomorrow for children everywhere.

Kevin B. Churchwell, President and CEO

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