Somatic CAG expansion in Huntington's disease is dependent on the MLH3 endonuclease domain, which can be excluded via MLH3 splice redirection to suppress expansion
Jennie C. L. Roy, Antonia G. Vitalo, Marissa A Andrew, Eduarda Mota‐Silva, Marina V. Kovalenko, Zoe N. Burch, Anh M. Nhu, Paula E. Cohen, Ed Grabczyk, Vanessa Chantal Wheeler, Ricardo Mouro Pinto
This study shows that targeting the MLH3 endonuclease domain reduces somatic CAG expansion in Huntington's disease models and patient cells.
Nucleic Acids Research · Feb 23, 2021Peer-reviewedGenetics