RICHMOND, Calif., Dec. 13, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new data demonstrating the ability to permanently correct a disease gene in an adult mouse model of hemophilia B using systemic delivery of zinc finger nucleases (ZFNs) at the 53nd Annual Meeting of the American Society of Hematology (ASH).
"We have demonstrated functional correction of a human gene for the clotting factor, Factor IX, with a single, systemic administration of ZFNs in an animal model of disease," said Geoff Nichol, M.B, Ch.B., Sangamo's executive vice president of research and development. "Our approach enables permanent correction of the genetic defect responsible for hemophilia B. This circumvents the problems of traditional gene-addition approaches that uncouple the gene from its normal regulatory mechanism and which may result in gene silencing and random gene insertion and potentially lead to malignancy or other unintended consequences."
Abst. No.668 - Robust Factor IX Expression Following ZFN-mediated Genome Editing in an Adult Mouse Model of Hemophilia B (Oral Session: 801)
In this study, scientists demonstrated efficient ZFN-mediated gene correction in an adult mouse model of hemophilia B with a single systemic administration of ZFNs and a donor DNA sequence encoding the corrected human Factor IX gene. Stable levels of protein made from the corrected human gene could be measured in the plasma of the treated animals and resulted in the restoration of normal rates of blood clotting for the period of the study. This work expands upon earlier studies, published in Nature*, that demonstrated similar results in neonatal mice. Data described at the ASH meeting demonstrate that growth of liver cells, as is observed in neonates, is not required for efficient
|SOURCE Sangamo BioSciences, Inc.|
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