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Gene Therapy Weekly

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Antisense Technology



Investigators at University of Iowa have published new data on antisense technology



July 17th, 2008

"Huntington's disease (HID) is a fatal, dominant neurodegenerative disease caused by a polyglutamine repeat expansion in exon 1 of the HID gene, which encodes the huntingtin protein. We and others have shown that RNAi is a candidate therapy for HID because expression of inhibitory RNAs targeting mutant human HID transgenes improved neuropathology and behavioral deficits in HID mouse models," scientists in the United States report.

"Here, we developed shRNAs targeting conserved sequences in human HID and mouse HID homolog (HDh) mRNAs to initiate preclinical testing in a knockin mouse model of HID. We screened 35 shRNAs in vitro and subsequently narrowed our focus to...


Source: Gene Therapy Weekly (2008-07-17)

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