Scientists at the Salk Institute for Biological Studies in La Jolla have cured a defective gene in cells taken from patients suffering from Fanconi anemia, a disease that can lead to bone marrow failure, leukemia and other cancers, it was reported Monday.
The work, published online Sunday in the journal Nature, offers the first proof that the technique used can work in human cells, according to The San Diego Union-Tribune.
“We haven’t cured a human being, but we have cured a cell,’' Salk Professor Juan-Carlos Izpisua Belmonte told the newspaper. “In theory, we could transplant it into a human and cure the disease.’'
Last month, California’s stem cell institute gave Belmonte and his fellow scientists a $6.6 million grant to continue their work. One big remaining problem is preventing the reprogrammed cells from inducing tumors, the Union-Tribune reported.