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Huntingtin Lowering Strategies

Marxreiter, Franz ; Stemick, Judith ; Kohl, Zacharias



Abstract

Trials using antisense oligonucleotide technology to lower Huntingtin levels in Huntington's disease (HD) are currently ongoing. This progress, taking place only 27 years after the identification of the Huntingtin gene (HTT) in 1993 reflects the enormous development in genetic engineering in the last decades. It is also the result of passionate basic scientific work and large worldwide registry ...

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