Here is some good news amid the general gloom: Scientists at the Salk Institute in La Jolla say they have cured a cell.
In a paper in the journal Nature, the Salk team reports it has fixed a defective gene in cells taken from patients with Fanconi anemia, a disease that can cause bone-marrow failure, leukemia and other cancers.
Their breakthrough is a big step toward showing that stem cells and gene therapy can cure genetic disease in humans.
"We haven't cured a human being, but we have cured a cell," Salk Institute professor Juan-Carlos Izpisúa Belmonte told the San Diego Union-Tribune. "In theory, we could transplant it into a human and cure the disease."
The researchers took hair and skin cells from patients with Fanconi anemia, corrected the defect, then reprogrammed the cells into stem cells in the lab. They said the new cells were indistinguishable from human embryonic stem cells or stem cells from healthy donors.
The next step is the big one: to show that the new and improved cells can be transplanted back into the patient and replicate themselves to cure the disease without causing any more problems. One big worry is that the new cells will themselves create cancerous tumors.
The researchers have received a $6.6 million grant from California's stem cell institute to continue their work, which has been under way for about two years.
Scientific journals are filled with reports of major breakthroughs that never seem to make it out of the lab and into the clinic, and there is no way of knowing if this one will be any different.
But here is hoping that Belmonte and his colleagues in San Diego will be able to take the next step. That would be great not only for people suffering from Fanconi anemia but for all manner of patients who might benefit from this kind of advanced genetic therapy.
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