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Pain & Central Nervous System Week

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Antisense Technology



Study results from University of Texas broaden understanding of antisense technology



June 15th, 2009

According to a study from the United States, "Expanded trinucleotide repeats(1) cause many neurological diseases. These include Machado-Joseph disease (MJD)(2) and Huntington's disease (HD)(3), which are caused by expanded CAG repeats within an allele of the ataxin-3 (ATXN3) and huntingtin (HTT) genes, respectively."

"Silencing expression of these genes is a promising therapeutic strategy, but indiscriminate inhibition of both the mutant and wild-type alleles may lead to toxicity, and allele-specific approaches have required polymorphisms that differ among individuals. We report that peptide nucleic acid and locked nucleic acid antisense oligomers that target CAG...


Source: Pain & Central Nervous System Week (2009-06-15)

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