Science: CRS4 contributes to gene therapy success treating metachromatic leukodystrophy and Wiskott-Aldrich syndrome

On July 11, 2013, Science’s ahead-of-print electronic publication service published two studies conducted by researchers at the Telethon Institute for Gene Therapy (TIGET) in Milan, Italy, directed by Luigi Naldini. These works show that gene therapy vectors derived from the HIV virus are effective in treating two serious genetic diseases: metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS): six children, after three years of treatment, are well and show significant benefits.
CRS4 contributed to this research effort through its expertise with bioinformatics and big data technologies: scalable computational tools developed at CRS4 allowed researchers at TIGET to efficiently analyze the huge flow of data produced by the molecular analysis of patient cells.


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