Stories about: hematology

Helping disease cells handle the stress of reprogramming

Fanconi anemia cells have multiple defects in response to DNA damage, making it hard to create iPS cells from them.

I’m attending an international pediatric oncology meeting in Boston with about 2,000 other people. In a session on gene therapy, David Williams, chief of Hematology/ Oncology at Children’s, talked about trying to treat a rare inherited bone marrow failure syndrome, Fanconi anemia, by correcting the gene in the patient’s blood stem cells. It didn’t work the first time, but he has a new tactic.

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