Identification of Selective and Non-Selective C9ORF72 Targeting in Vivo Active Sirnas.

James W. Gilbert,Zachary Kennedy,Bruno M. D. C. Godinho,Ashley Summers,Alexandra Weiss,Dimas Echeverria,Brianna Bramato,Nicholas McHugh,David Cooper,Ken Yamada,Matthew Hassler,Helene Tran,Fen Biao Gao,Robert H. Brown Jr,Anastasia Khvorova

Molecular Therapy — Nucleic Acids（2024）

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MT: Oligonucleotides: Therapies and Applications,oligonucleotide therapeutics,siRNA,C9ORF72 amyotrophic lateral sclerosis, ALS,frontotemporal dementia, FTD,mRNA localization,dipeptide-repeat proteins

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