June 2, 2009

Study advances regenerative medicine

U.S. and Spanish scientists say they have proven, in principle, that a human genetic disease can be cured using a combination of gene and stem cell therapy.

The researchers, led by the Salk Institute for Biological Studies, said their achievement has catapulted the field of regenerative medicine significantly forward.

It's been 10 years since human stem cells were first cultured in a Petri dish, Professor Juan-Carlos Izpisua Belmonte of the Center of Regenerative Medicine in Barcelona, Spain, said. The hope in the field has always been that we'll be able to correct a disease genetically and then make (induced pluripotent stem cells) that differentiate into the type of tissue where the disease is manifested and bring it to clinic.

Although several studies have demonstrated the efficacy of the approach in mice, its feasibility in humans had not been established. The researchers said the Salk study offers the first proof that the technology can work in human cells.

We haven't cured a human being, but we have cured a cell, Belmonte said. In theory we could transplant it into a human and cure the disease.

The research is reported in the early online edition of the journal Nature.