GeneticsNucleic Acids ResearchPeer-reviewed
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…·Ricardo Mouro Pinto
This study shows that targeting the MLH3 endonuclease domain reduces somatic CAG expansion in Huntington's disease models and patient cells.