Stories about: Fanconi anemia

Rebooting Fanconi anemia cells: You have to fix the broken code first

David Williams wants to turn cells from Fanconi anemia (FA) patients into stem-like iPS cells. To do that, though, he needs to get the patients' cells to reboot properly. (_rockinfree/Flickr)

About a decade ago, David Williams, MD, set out to solve a problem. The chief of Dana-Farber/Children’s Hospital Cancer Center’s Hematology/Oncology division wanted to treat Fanconi anemia (FA)—a rare, inherited bone marrow failure disease—using gene therapy. In the process, he’d be able to replace patients’ faulty bone marrow cells with ones corrected for the genetic defect that causes FA.

There was one big problem though. “The main bar to attempting gene therapy in FA is that you need to be able to collect a certain number of blood stem cells from a patient in order to be able to give enough corrected cells back,” he says. “In our early clinical trials, we were unable to provide enough corrected stem cells to reverse the bone marrow failure we see in these patients.”

One way around the supply issue would be to create the necessary blood stem cells from FA patients’ own cells by first reprogramming skin cells into what are called induced pluripotent stem (iPS) cells. Using one of several methods, scientist can reboot mature skin cells into an immature, stem cell-like state—essentially turning the cells’ biological clocks back to a time when they could grow into anything the body might need.

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Stem cell experiments in genetic blood diseases

The green tips of these chromosomes are telomeres, whose length is a measure of cellular "aging" and determines how many times a cell can divide.

In a roomful of kids’ cancer specialists, like those listening to the keynote speech by George Daley, closing an international pediatric oncology meeting in Boston, the Myc gene is better known as a mutated weapon of mass destruction.

But this driver of cancer growth is also part of a four-gene cocktail that can reprogram an adult skin cell back into an embryonic-like stem cell that holds great therapeutic potential.

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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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