Healthy blood cells from patient's skin cells

Friday June 5th, 2009

Scientists have developed a new method of producing healthy blood cells from skin cells of patients with a rare blood disorder.

Fanconi anaemia is a genetic disease which causes restricted growth and skeletal abnormalities, as well as an increased risk of solid tumors and leukemias.

Dr Jordi Surralles of the Autonomous University of Barcelona, Spain, and colleagues were able to correct the genetic defect in skin cells by converting them into cells similar to embryonic stem cells, which can later form healthy blood cells.

The process is based on gene therapy and cell reprogramming techniques, explains the team in the journal Nature.

They add that these results are the "proof of concept" that this strategy can work.

It is particularly important in the treatment of diseases such as Fanconi anaemia, in which the patient may not produce sufficient blood cells in their bone marrow, they say. It may also benefit a range of other patients.

The authors write: "Patient-specific induced pluripotent stem (iPS) cells are thought to hold great therapeutic potential, although direct evidence for this is still lacking. Here we show that, on correction of the genetic defect, somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency to generate patient-specific iPS cells.

"These cell lines appear indistinguishable from human embryonic stem cells and iPS cells from healthy individuals.

"Most importantly," they add, "these corrected cells can give rise to haematopoietic stem cells that are disease-free.

"These data offer proof-of-concept that iPS cell technology can be used for the generation of disease-corrected, patient-specific cells with potential value for cell therapy applications," they conclude.

Raya, A. et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature, published online May 31, 2009.

Tags: Europe | General Health

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