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.
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