Daniel O'Reilly, Jillian Belgrad, Chantal Ferguson, Ashley Summers, Ellen Sapp, Cassandra McHugh, Ella Mathews, Adel Boudi, Julianna Buchwald, Socheata Ly, Dimas Moreno, Raymond Furgal, Eric Luu, Zachary Kennedy, Vignesh Hariharan, Kathryn Monopoli, X William Yang, Jeffery Carroll, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Huntington's Disease (HD) is a severe neurodegenerative disorder caused by expansion of the CAG trinucleotide repeat tract in the huntingtin gene. Inheritance of expanded CAG repeats is needed for HD manifestation, but further somatic expansion of the repeat tract in non-dividing cells, particularly striatal neurons, hastens disease onset. Called somatic repeat expansion, this process is mediated by the mismatch repair (MMR) pathway. Among MMR components identified as modifiers of HD onset, MutS Homolog 3 (MSH3) has emerged as a potentially safe and effective target for therapeutic intervention...
May 11, 2023: Molecular Therapy