Gene Therapy

Data on gene therapy detailed by researchers at Genethon

Published in Gene Therapy Weekly, July 16th, 2009

"The woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) is widely used in retroviral gene transfer vectors. However, this element contains an open-reading frame (ORF) encoding a truncated peptide of the woodchuck hepatitis virus X protein (WHX)," researchers in Evry, France report.

"Because we are developing a lentiviral vector for the gene therapy of Wiskott-Aldrich syndrome (WAS), we evaluated whether the WPRE was needed in the gene transfer cassette and tested the possibility of replacing it with a mutated derivative. The transcriptional activity of the WPRE was undetectable in the context of the lentiviral vector but the element was capable of...

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Source: Gene Therapy Weekly (2009-07-16)

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