Gene therapy 'success' revealed


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Gene therapy has been used for the first time to treat an inherited blood disorder in what doctors say is a major step forward.

A man given pioneering treatment to correct a faulty gene has made "remarkable" progress, a US and French team has revealed.

Gene therapy is an experimental technique that manipulates genes in order to treat disease.

Beta thalassaemia is an inherited blood disorder that affects the body's ability to create red blood cells.

The first gene therapy trial was in an 18-year-old man with a severe form of the condition, who had been receiving regular blood transfusions since the age of three.

Stem cells from his bone marrow were treated with a gene to correct for the faulty one.

They were then transfused back into his body, where they gradually gave rise to healthy red blood cells.

Three years after the treatment, which took place in 2007, the man remains mildly anaemic, but no longer needs blood transfusions, doctors said.

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I think this qualifies more as an in vitro gene correction. In this case, they could extract the progenitor cells, modify them, and pop them back into the bone marrow. I don't think it uses techniques beyond those known by anyone with a BS/BA in biology.

I have a feeling that gene modification/corrections will not be easily accepted by society. We really can get to a point where we give someone an injection full of virus reprogrammed to overwrite, add, or delete any gene in order to correct a genetic disorder, but there are ethical and philosophical objections to such treatments. I think those, beyond any technical hurdles, represent the ultimate barrier towards these sorts of cures.

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How can there be ethical objections to curing a disorder or disease? The only ones I can think of would be religious in nature, and they need to be ignored completely as they have no sensible grounding.

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