Article
Inhibiting cellular uptake of mutant huntingtin using a monoclonal antibody: Implications for the treatment of Huntington's disease.
Neurobiology of disease - 1 Jul 2020
Bartl Stefan, Oueslati Abid, Southwell Amber L, Siddu Alberto, Parth Michela, David Linda Suzanne, Maxan Alexander, Salhat Nina, Burkert Markus, Mairhofer Andreas, Friedrich Theresa, Pankevych Halyna, Balazs Katja, Staffler Guenther, Hayden Michael R, Cicchetti Francesca, Smrzka Oskar W
Abstract excerpt
Huntington's disease (HD) is caused by a highly polymorphic CAG trinucleotide expansion in the gene encoding for the huntingtin protein (HTT). The resulting mutant huntingtin protein (mutHTT) is ubiquitously expressed but also exhibits the ability to propagate from cell-to-cell to disseminate pathology; a property which may serve as a new therapeutic focus. Accordingly, we set out to develop a monoclonal antibody...
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