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Gene treatment may cure children

Thursday August 25th, 2011

A trial of gene therapy on children with severe immune disorders has proved remarkably successful, British researchers reported last night.

Some 16 children underwent the children nine years ago and all are still alive, researchers at University College London and Great Ormond Street Hospital reported.

Even better, 14 of the children are living normal lives, researchers said.

The success of the trial was reported in Science Translational Medicine. The children had two kinds of severe combined immunodeficiency disorder - SCID, one called SCID-XI and one called ADA-SCID.

Until recently the diseases were regarded as incurable and children kept shielded from the outside world to prevent infection. Then treatments were developed using stem cell transplants with some success - but not all children could be saved.

The researchers say gene therapy seems to be more effective than stem cell transplants.

Researcher Professor Bobby Gaspar said the project was among a "handful" of studies" showing that gene therapy can work.

He said: "These children are going to school, attending parties doing everything other children are able to do.

"That's why it is exciting. we have two conditions and 14 children showing that gene therapy can be both successful and long-lasting."

He said gene therapy would offer something "different" if stem cell transplants were unlikely to work or a donor could not be found.

Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency. H.B.Gaspar; S. Cooray; K.C. Gilmour; K.L. Parsley; S.J. Howe; J. Bayford; L. Brown; E. G. Davies; C. Kinnon; A.J Thrasher at University College London in London, UK; H.B. Gaspar; S. Cooray; K.C. Gilmour; K.L. Parsley; S. Adams; J. Bayford; L. Brown; E.G. Davies; A.J. Thrasher at Great Ormond Street Hospital NHS Trust in London, UK; A. Al Ghonaium at King Faisal Specialist Hospital and Research Centre in Riyadh, Saudi Arabia. Science Translational Medicine August 24 2011

Tags: Child Health | Genetics | UK News

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