chapter  10
12 Pages

Chapter Recombinant Intrabodies as Molecular Tools and Potential Therapeutics for Huntington’s Disease

The therapeutic potential of intracellularly expressed, recombinant, or single-chain fragment variable (scFv) antibodies (intrabodies) is being explored for several diseases, including cancer, HIV, and neurodegenerative disorders. Intrabodies can bind and inactivate toxic intracellular proteins, prevent misfolding, promote degradation, and block aberrant protein-protein interactions with extreme molecular speci city. Neurodegenerative disorders are particularly attractive candidates for these reagents because many of these diseases involve protein misfolding, oligomerization, and aggregation [1]. In particular, intrabodies have shown ef cacy in blocking the toxicity of the amyloidogenic protein fragment Aβ in cell culture and mouse models of Alzheimer’s disease (AD), paving the way for clinical trials of these reagents in brain

Introduction ........................................................................................................... 255 Strategies for Intrabody Construction ...................................................................256