We have implicated a new gene in the progression of Huntington’s disease in a brain organoid model. The gene may contribute to brain abnormalities much earlier than previously thought. Moreover, our genome editing strategies, in particular the removal of the CAG repeat region in the Huntington gene, showed great promise in reversing some of observed developmental defects. This suggests a potential gene therapy approach.
More information can be found:
https://www.nature.com/articles/s41467-024-51216-w
https://www.sciencedaily.com/releases/2024/08/240822125933.htm
https://e3.eurekalert.org/news-releases/1055398
https://e3.eurekalert.org/multimedia/1038832
https://huntingtonsdiseasenews.com/news/proteins-loss-brain-may-cause-huntingtons-long-before-onset/