Gene Therapy

Studies in the Area of Gene Therapy Reported from C. Spampanato and Co-Researchers

Published in Gene Therapy Weekly, June 30th, 2011

2011 JUN 30 -- According to a study from Naples, Italy, "Multiple sulfatase deficiency (MSD), a severe autosomal recessive disease is caused by mutations in the sulfatase modifying factor 1 gene (Sumf1). We have previously shown that in the Sumf1 knockout mouse model (Sumf1(-/-)) sulfatase activities are completely absent and, similarly to MSD patients, this mouse model displays growth retardation and early mortality."

"The severity of the phenotype makes MSD unsuitable to be treated by enzyme replacement or bone marrow transplantation, hence the importance of testing the efficacy of novel treatment strategies. Here we show that recombinant adeno-associated...

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Source: Gene Therapy Weekly (2011-06-30)

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