48. In Vivo and In Situ Delivery of Huntingtin-Specific Single-Chain Fv Intrabody Genes as Therapeutic Agents for Huntington's Disease

Huntington's disease (HD) is one of several neurodegenerative disorders caused by expansion of a CAG repeat. The resulting expanded polyglutamine tract causes the huntingtin protein to form insoluble aggregates and participate in aberrant protein-protein interactions. Therapeutic intervention t...

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Veröffentlicht in:Molecular therapy 2004-05, Vol.9 (S1), p.S20-S21
Hauptverfasser: Miller, Todd W, Wolfgang, William J, Manley, Kevin, Zhou, Yangsheng, Zhou, Chun, Webster, Jack M, Mazarakis, Nicholas D, Messer, Anne
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Sprache:eng
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