Children’s Researchers Receive NIH Grant to Develop Blood Disease Gene Therapy

Children’s Hospital Boston’s Stem Cell Program’s Dr. George Daley and colleagues at Children’s and Dana-Farber have received an NIH grant to develop a stem cell-based gene therapy to treat Fanconi anemia, a fatal blood disease.

The project is a collaboration among David Williams, MD, chief of Hematology/Oncology and director of Translational Research at Children’s, George Daley, MD, PhD, director of Stem Cell Transplantation, and Alan D’Andrea, MD, chief of research in Radiation Oncology at Children’s. All three are members of Dana-Farber/Children’s Hospital Cancer Center.

Fanconi anemia is a rare genetic disease in which a defect in DNA repair causes the loss of blood stem cells, resulting in bone marrow failure, increased likelihood of developing cancer, and early death.

For more information about this research, please read our full press release and our blog post on Vector, Children’s Hospital Boston’s science and clinical innovation blog.



  • Blood diseases: The First Frontier

    Hematologist George Q. Daley, MD, PhD, Director of Stem Cell Transplantation
    Program, has seen children with blood diseases die, often because they aren’t candidates for bone marrow transplants, currently the best tool for treating many of these diseases. Daley hopes to employ pluripotent stem cells to create safer, genetically matched bone-marrow transplants for patients. Read more.

  • Blood stem cells grow with the flow

    Children’s Hospital Boston researchers, with collaborators, have discovered that a beating heart and blood flow are needed for the blood system to develop, and that mechanical stresses cue its formation. If scientists could figure out how to harness this process, the findings could provide new ways of making more blood stem cells for patients. Read more.

  • 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.

  • Combining stem cell therapy with gene therapy

    The possibility of curing a disease by making pluripotent stem cells and repairing the defective gene was first demonstrated in mice in 2002. Read more.