Sickle cell anemia treated with stem cells

MIT researchers have successfully treated mice with sickle-cell anemia in a process that begins by directly reprogramming the mice’s own cells to an embryonic-stem-cell-like state, without the use of eggs.

This is the first proof-of-principle of therapeutic application in mice of directly reprogrammed induced pluripotent stem (IPS) cells, which recently have been derived in mice as well as humans.

“This demonstrates that IPS cells have the same potential for therapy as embryonic stem cells, without the ethical and practical issues raised in creating embryonic stem cells,” said MIT biology professor Rudolf Jaenisch, a member of the Whitehead Institute for Biomedical Research.

The research, reported Dec. 6 in Science online, was carried out in Jaenisch’s laboratory. The IPS cells were derived using modifications of the approach originally discovered in 2006 by the Shinya Yamanaka laboratory at Kyoto University.

A disease of the blood marrow caused by a defect in a single gene, sickle-cell anemia is the most common inherited blood disorder in the United States, affecting more than 70,000 Americans and one in 500 African-Americans, according to the National Institutes of Health.

The scientists in Jaenisch’s lab studied a therapeutic application of IPS cells with the sickle-cell anemia model mouse developed by the laboratory of Tim Townes of the University of Alabama at Birmingham. The mouse model had been designed to include relevant human genes involved in blood production, including the defective version of that gene.

This work was funded in part the National Institutes of Health.

Source: Massachusetts Institute of Technology, USA

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