- McConoughey, SJ;
- Basso, M;
- Niatsetskaya, ZV;
- Sleiman, SF;
- Smirnova, NA;
- Langley, BC;
- Mahishi, L;
- Cooper, AJL;
- Antonyak, MA;
- Cerione, RA;
- Li, B;
- Starkov, A;
- Chaturvedi, RK;
- Bea, MF;
- Coppola, G;
- Geschwind, DH;
- Ryu, H;
- Xia, L;
- Iismaa, SE;
- Pallos, J;
- Pasternack, R;
- Hils, M;
- Fan, J;
- Raymond, LA;
- Marsh, JL;
- Thompson, LM;
- Ratan, RR
Caused by a polyglutamine expansion in the huntingtin protein, Huntington's disease leads to striatal degeneration via the transcriptional dysregulation of a number of genes, including those involved in mitochondrial biogenesis. Here we show that transglutaminase 2, which is upregulated in HD, exacerbates transcriptional dysregulation by acting as a selective corepressor of nuclear genes; transglutaminase 2 interacts directly with histone H3 in the nucleus. In a cellular model of HD, transglutaminase inhibition de-repressed two established regulators of mitochondrial function, PGC-1a and cytochrome c and reversed susceptibility of human HD cells to the mitochondrial toxin, 3-nitroproprionic acid; however, protection mediated by transglutaminase inhibition was not associated with improved mitochondrial bioenergetics. A gene microarray analysis indicated that transglutaminase inhibition normalized expression of not onlymitochondrial genes but also 40% of genes that are dysregulated in HD striatal neurons, including chaperone and histone genes. Moreover, transglutaminase inhibition attenuated degeneration in a Drosophila model of HD and protectedmouse HD striatal neurons from excitotoxicity. Altogether these findings demonstrate that selective TG inhibition broadly corrects transcriptional dysregulation in HD and defines a novel HDAC-independent epigenetic strategy for treating neurodegeneration. © 2010 EMBO Molecular Medicine.