Di-valent siRNA Mediated Silencing of MSH3 Blocks Somatic Repeat Expansion in Mouse Models of Huntington's Disease
Daniel O’Reilly, Jillian Belgrad, Chantal M. Ferguson, Ashley Summers, Ellen Sapp, Cassandra A. McHugh, Ella W. Mathews, Adel Boudi, Julianna E. Buchwald, Socheata Ly, Dimas Moreno, Raymond C. Furgal, Eric Luu, Zachary Kennedy, Vignesh Narayan Hariharan, , Anastasia Khvorova
Silencing MSH3 in the CNS of two models of Huntington's disease using di-valent siRNA blocks disease-accelerating somatic expansion of CAG repeats.
Molecular Therapy · May 11, 2023Peer-reviewedMolecular Biology