Synthesizing a human embryo from stem cells and using gene editing to insert the Huntingtin gene, researchers found the mutation affected the size of germ layers compared to the control embryos. Findings suggest Huntington's disease may be a neurodevelopmental disorder that presents as a neurodegenerative disease later
A mutated form of the huntingtin protein disrupts the normal movement of vesicles holding HT and Rab4. This leads to defects in synapses, resulting in movement abnormalities and lifespan decreases in fruit fly larvae. Findings suggest Rab4 could be a novel therapeutic target for the early intervention of Huntington's disease, before the neuronal loss and behavioral deficits associated with the neurodegenerative disorder.
Mouse models of corticospinal injuries reveal adult neurons begin a natural regeneration process by reverting back to an embryonic state. The regeneration is sustained with the help of a gene more commonly associated with Huntington's disease.
Researchers have identified a mechanism that may reduce the toxic aggregation of the huntingtin protein. The findings could lead to new treatment options for Huntington's patients.
Rockefeller University researchers reveal Huntington's neurons are much larger than healthy cells.
UCL researchers report clinical trials of IONIS-HTTRx have shown success in reducing the huntinting protein in the nervous system of those with Huntington's disease. The drug is safe and well tolerated by human patients, the researchers note.