Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs

• Main Authors: James W. Gilbert (Author), Zachary Kennedy (Author), Bruno M.D.C. Godinho (Author), Ashley Summers (Author), Alexandra Weiss (Author), Dimas Echeverria (Author), Brianna Bramato (Author), Nicholas McHugh (Author), David Cooper (Author), Ken Yamada (Author), Matthew Hassler (Author), Hélène Tran (Author), Fen Biao Gao (Author), Robert H. Brown, Jr (Author), Anastasia Khvorova (Author)
• Format: Book
• Published: Elsevier, 2024-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

A hexanucleotide (G4C2) repeat expansion (HRE) within intron one of C9ORF72 is the leading genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). C9ORF72 haploinsufficiency, formation of RNA foci, and production of dipeptide repeat (DPR) proteins have been proposed a...