A group led by the Sanger Institute and the University of Cambridge, working with the Sangamo Biosciences, has shown that it’s possible to fix mutations in reprogrammed cells. This work, which was published in Nature, takes two previous advances and combines them into one proof-of-concept.

Since 2007 stem cell scientists have been able to reprogram adult cells such as skin back into an embryonic-like state. These so-called iPS cells can then mature into any cell type in the body, much like embryonic stem cells.

Other groups have shown that it’s possible to take adult stem cells such as those from the bone marrow, correct mutations, and create mutation-free cells that, at least in animal models, can fix diseases. That’s the idea behind CIRM’s two HIV/AIDS disease team awards (described here and here) and a sickle cell disease team. In fact, one of the HIV/AIDS disease teams is also working with technology developed by Sangamo to fix the mutations.

In the work reported in Nature, the team created iPS cells from a person with a genetic liver disease, fixed the mutation, then matured the iPS cells into functional liver cells. A Reuters story quotes Allan Bradley, director of the Sanger Institute:

“These are early steps, but if this technology can be taken into treatment, it will offer great possible benefits for patients,” he added.

Reuters quoted David Lomas, who was part of the team from Cambridge, saying that the liver cells survived when transplanted into mice.

The researchers said it could be another five to 10 years before full clinical trials of the technique could be run using patients with liver disease. But if they succeed, liver transplants — costly and complicated procedures where patients need a lifetime of drugs to ensure the new organ is not rejected — could become a thing of the past.

“If we can use a patient’s own skins cells to produce liver cells that we can put back into the patient, we may prevent the future need for transplantation,” said Lomas.

A.A.