Through targeted genetic knock-down, huntingtin is identified as a cell-autonomous regulator of neuropeptide trafficking, neuronal excitability and circadian output and carries direct relevance for HD therapeutic strategies, particularly those involving huntingtin-lowering approaches.
These findings highlight the PRD’s contribution to HD toxicity and point to MKL2/MRTFB and the cytoskeleton and transcriptional coactivator MKL2/MRTFB and the cytoskeleton as candidate mediators.
R. Iennaco, Camilla Maffezzini, Simone Maestri et al.· bioRxiv· 1 citation
It is shown that neurogenesis is disrupted at multiple stages of lineage progression in both rodent and human neural stem cell models of Huntington's disease, and a panel of clinically relevant epigenetic compounds hold promise for stage-spanning therapeutic strategies capable of modifying disease trajectory.
Jessica Rosati, A. Casamassa, G. Ruotolo et al.· Cell Death and Differentiati...· 0 citations
The findings indicate that polyQ has a significant role in the disease's pathogenesis, which includes altered functional activity and structural modifications leading to inclusion body formation, which can be controlled with the help of HAP40.
G. Parmar, Kharerin Hungyo· ACS Chemical Neuroscience· 0 citations
Heat shock factor 1 (HSF1) plays a pivotal role in maintaining neuronal health, and its dysfunction contributes significantly to the progression of Huntington’s disease. Rocio Gomez-Pastor tells us more. Huntington’s disease (HD) is a devastating, inherited neurodegenerative disorder marked by progressive motor dys...
Rocio Gomez-Pastor· Open Access Government· 0 citations
Findings define a biphasic course of huntingtin toxicity and highlight an early therapeutic window in which intervention is most likely to be applied, prior to irreversible deterioration of mitochondrial respiratory function.
Ekin Sonmez, Pınar Mutlu, Cinla Ozlevent et al.· bioRxiv· 0 citations
The results indicate that expression of toxic C9orf72 components in glia produces deleterious effects on lifespan, though potentially through different mechanisms than seen in TDP-43 models of ALS/FTD.
I. Hubbard, J. Dubnau· PLoS Genetics· 0 citations
We use cookies to run the site and, with your consent, for analytics and to show ads.
See our Cookie Policy.