Anti-HD-polyAla Ct serum, from rabbit

Huntington disease (HD) is a progressive neurodegenerative disorder is caused by a CAG,CTG expansion in the HTT gene that results in the production of a mutant huntingtin protein (HTT) with polymeric expansions that accumulate in human brain with HD. PolyAla is a sense repeat-associated non-ATG (RAN) translation protein that accumulates in various brain regions with some toxic effects leading to microglial activation and neuronal loss. HD-RAN polyAla toxicity is shown to be lower compared to that of polyGln. The transfection of cells with polyAla construct can express high polyAla levels, but have lower toxicity. HD-polyAla RAN proteins are reported to accumulate in cells expressing CAG repeats that are equal to or greater than 52 but not at repeats less than 45. HD-RAN proteins are abundant in regions of the brain that are most affected by HD and these regions display pathologic features of HD, such as caspase-3 activity and microglial activation. HD-Ran protein accumulation and aggregation in HD brains are shown to be length dependent.