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Combining Stem Cell Therapy with Gene Therapy

When pluripotent stem cells are made from a patient’s own cells, it may be also be possible to replace the faulty gene that caused their disease with a normal, healthy copy. The repaired stem cells could then be directed to form the tissue type needed, introduced into the body, allowed to divide, and used to reconstitute the diseased tissue. It's a treatment that should last a lifetime.

Boston Children’s Hospital researcher George Q. Daley, MD, PhD, then at the Whitehead Institute, was the first to demonstrate, in 2002, that pluripotent stem cells could successfully treat a disease. Working with mice that possess a genetic defect caused by an immune deficiency, the research team created genetically-matched embryonic stem cells through nuclear transfer, introduced corrective genes, then derived healthy blood stem cells and infused them into the mice, partially restoring their immune function. Daley, Director of Stem Cell Transplantation at Children’s, would like to do the same for his patients with blood diseases.



  • Stem cell therapy for blood diseases

    In this video clip, Leonard Zon, MD, discusses why stem cell therapy for blood diseases will likely make it to the bedside first. He then goes on to discuss an exciting new treatment—now in clinical trial—that promises to make this therapy more effective and efficient.

  • iPS cells: Rewinding cellular time

    Rejection-proof transplants made from our own cells? Learn more about the treatment and research potential of iinduced pluripotent stem cells
    (iPS cells) in this feature article and 4-part video series.