Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidence.
Terence Gall-Duncan, Sangyoon Y. Ko, Isabelle K. Quick, Mahreen Khan, Kristie Feng, Chase P. Kelley, Annabelle Coleman, Alexiane Touzé, Shuqian Tang, Mustafa Hussain Mehkary, Katsuyuki Yokoi, Casey R. Herrington, Justin You, Scott C. Lambie, Tanya K. Prasolava, , Christopher E. Pearson
This study shows that reducing somatic CAG expansions in HD mice improves motor function and disease markers, suggesting therapeutic potential for HD and similar repeat expansion diseases.
bioRxiv · Apr 25, 2025 · PreprintGenetics