Sequencing-guided design of genetically encoded small RNAs targeting CAG repeats for selective inhibition of mutant huntingtin

• Main Authors: Mansi A. Parasrampuria (Author), Adam A. White (Author), Ramadevi Chilamkurthy (Author), Adrian A. Pater (Author), Fatima El-Azzouzi (Author), Katy N. Ovington (Author), Philip J. Jensik (Author), Keith T. Gagnon (Author)
• Format: Book
• Published: Elsevier, 2024-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Huntington's disease (HD) is an incurable neurodegenerative disorder caused by genetic expansion of a CAG repeat sequence in one allele of the huntingtin (HTT) gene. Reducing expression of the mutant HTT (mutHTT) protein has remained a clear therapeutic goal, but reduction of wild-type HTT (wtH...