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Genetic defects treated by 'fixing' faulty DNA

Future treatments for patients with genetic defects are a step closer to becoming reality after researchers in the US found a way to repair faulty DNA in human stem cells.

Stem cells were taken from patients with a rare inherited blood disease called Fanconi anaemia (FA) and were reprogrammed to create artificial cells.

A gene therapy technique was then used to repair the faulty DNA in the cells linked to the disorder, with tests showing that the stem cells could be coaxed into healthy bone marrow cells.

Stem cells are immature cells that can develop along a range of pathways to become different cell types and have the potential to become virtually any part of the body.

This latest breakthrough, carried out at The Salk Institute in California and published in an online version of the journal Nature, paves the way for new therapies for currently incurable genetic disorders.

Responding to the research, stem cell scientist Professor Chris Mason, from University College London, said: ‘This research demonstrates a very novel method of generating cells to potentially treat a rare inherited disorder.

‘There is no doubt that this paper will be the first of many to offer hope for conditions where today there is no real therapy, let alone a cure.’

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