'Skin cell cure' for sickle cells

'Skin cell cure' for sickle cells

Scientists say they have found a new weapon against the blood disease sickle cell anaemia - the skin.
By reprogramming skin cells in the lab to become stem cells, the US team were able to treat mice with a human type of sickle cell anaemia, Science reports.

As well as being in plentiful supply, using skin cells as a therapy also gets round the ethics surrounding the use of other stem-cell based treatments.

Experts stressed more safety work was needed before moving trials to humans.

Stem cells

Stem cells are at an early stage of development and retain the potential to turn into many different types of cell, which means they have the scope to treat a number of diseases.

Scientists believe the most useful stem cells come from the tissue of embryos.

This is because they are pluripotent - they have the ability to become virtually any type of cell within the body.

Stem cells are also found within adult organs, including skin.

While the reprogrammed skin cells in the latest study - called induced pluripotent stem cells or IPS cells - hold tremendous promise, the scientists caution they also have the potential to cause dangerous side effects.

Safety issues

To make the IPS cells, the scientists started with cells from the skin of the diseased mice, then used viruses, called retroviruses, to insert therapeutic genes into the DNA of these cells.

While this was effective, the retroviruses have the ability to make random changes to DNA elsewhere in the body, which could potentially lead to complications, such as cancer.

Lead researcher Rudolf Jaenisch, of the Whitehead Institute for Biomedical Research in Massachusetts, said: "We need a delivery system that doesn't integrate itself into the genome.

We need more research. There are still significant hurdles to overcome

Professor Azim Surani of the University of Cambridge

"Retroviruses can disrupt genes that should not be disrupted or activate genes that should not be activated."

Professor Azim Surani of the University of Cambridge said: "This is an important step forwards.

"But in addition to the safety concerns, we have to be cautious about extrapolating from mouse studies to humans.

"The mouse IPS cells are not identical to human IPS cells. We need more research. There are still significant hurdles to overcome."

More than 12,500 people in the UK have sickle cell anaemia.

It is an inherited disease which affects the ability of red blood cells to carry oxygen and can cause severe pain, and damage to the organs.