New stem cell gene therapy success

11 July 2013

Papers published in the advance version of the journal Science report the use of a form of stem cell gene therapy to treat two different serious genetic diseases.
 
The diseases treated with the new therapy were metachromatic leukodystrophy, a lysosomal storage disorder that typically leads to death in early childhood and for which no treatment is possible, and Wiskott-Aldrich Syndrome, a severe form of genetic immune deficiency that can only be cured with bone marrow transplants from tissue-matched donors.
 
The technique developed by Italian researchers used a lentivirus-based gene therapy vector to insert healthy genes into bone marrow blood-cell precursors (haematopoetic stem cells), which were then transplanted into the patients.
 
The three pre-symptomatic children with metachromatic leukodystrophy who received the therapy were reported to have shown no symptoms of the disease at up to 21 months beyond the normal age of onset. Those treated for Wiskott-Aldrich Syndrome showed improved immune functions and a reduction in symptoms.
 
Although the numbers of children treated were small, and the period since treatment relatively short so that the longer-term safety and efficacy of the gene therapy is not yet known, but experts have said the results are nevertheless very exciting.

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