Gene therapy and stem cells unite

Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.

It means patients with a genetic disease could, one day, be treated with their own cells.

A study in Nature corrected a mutation in stem cells made from a patient with a liver disease.

Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.

At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would also contain the corrupted genetic code.

Scientists, at the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease.

It is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.

As a result, a protein which protects the body from damage, antitrypsin, cannot escape from the liver where it is made.

The research group took a skin cell from a patient and converted it to a stem cell.

A molecular scalpel was used to cut out the single mutation and insert the right letter - correcting the genetic fault.

The stem cells were then turned into liver cells. One of the lead researchers, Prof David Lomas, said: "They functioned beautifully with normal secretion and function".

Further animal studies and human clinical trials would be needed before any treatment as "the key thing is safety".

This is another big step in the "personalized medicine."

From related article: Human 'cloning' makes embryonic stem cells - BBC, by James Gallagher, 2011 October 05

A form of cloning has been used to create personalised embryonic stem cells in humans, say researchers.

Genetic material was taken from an adult skin cell and transferred into a human egg. This was grown to produce an early embryo.

Stem cells have huge potential in medicine as they can transform into any other cell type in the body.

However, the stem cells formed contained chromosomes from both the adult and the egg cells.

..... < snip >

The lead researcher at the New York Stem Cell Foundation Laboratory, Dr Dieter Egli, said ... other "groups had tried before, but failed".

Writing in the journal Nature, he said his group had also failed using traditional techniques.

When they removed the genetic material from the egg and replaced it with the chromosomes from a skin cell, the egg divided but failed to go past the 6-12 cell stage.

However, when they left the egg's own genetic material in place and added the skin chromosomes, the egg developed. It reached the blastocyst stage, which can contain up to 100 cells and is the usual source of embryonic stem cells.

..... < snip >

Dr Egli told the BBC: "The cells we have made are not yet for therapeutic use. There is clearly more work to be done, this is early days. ... "While this approach does not in itself provide a solution, it takes us a step closer to understanding where the problems lie."

No embryos required

Recently a different route to stem cells has been used. Instead of using an egg, a chemical bath "reprogrammes" an adult cell into a stem cell. ..... < snip >

From related article: Human 'cloning' makes embryonic stem cells

Thanks for reminding me about that story. I saw it last week at ScienceNOW, Science's free website. Alpha 1-antitrypsin deficiency is the name of the disease for this gene therapy/induced pluripotent stem cell stoty. I just posted it. It has the abstract from Nature.

Spell-Checked Stem Cells Show Promise Against Liver Disease

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