the exact mechanism by which mutant huntingtin causes neuronal dysfunction is not known. Recent studies demonstrating altered messenger RNA expression point to transcriptional dysregulation as a ...

Symptoms of Huntington's disease appear late in life. Research reveals how DNA repeats grow toxic over time, explaining the delayed onset and disease variability.

The paper contributes to an ongoing debate about the role of inclusion bodies—intracellular clumps of mutant huntingtin (Htt) protein—in the pathology of diseases like Huntington's and spinocerebellar ...

For instance, a new small molecule called branaplam has shown promise in reducing mutant huntingtin levels by promoting ... (ASOs): Short strands of DNA or RNA that can bind to specific RNA ...

Current attempts to develop treatments for Huntington’s are based on the premise that all mutant huntingtin is toxic, and that its suppression with drugs will, therefore, prevent or ameliorate ...

For years the lab has been conducting basic research into the Spt5 regulatory protein, a large protein with many functional segments that helps in the production of messenger RNA molecules and is ...

Atalanta Therapeutics, a Boston biotechnology company making medicines for the brain from RNA molecules ... muffles the gene making the “huntingtin” protein, which, when misformed in people ...

HD is caused by an expanded glutamine repeat in the N-terminal region of the huntingtin (HTT) protein ... High expression of the mutant HTT is significantly correlated with the downregulation of the ...