[4S05a] 今や主流になった神経難病の核酸医薬創薬の最前線

[4S05a-02] Integrating structure and function to guide development of therapeutic siRNAs for the central nervous system and beyond.

*Chantal Ferguson Ferguson^1,2, Julia Alterman^1,2, Ken Yamada^1,2, Dimas Echeverria^1,2, Bruno Godinho^1,2,3, Matthew Hassler^1,2,3, Neil Aronin^1,2,4, Anastasia Khvorova^1,2,4 (1. University of Massachusetts Medical School, 2. RNA Therapeutics Institute, 3. Atalanta Therpeutics, 4. Department of Medicine)

キーワード：siRNA, oligonucleotide, chemically modified siRNA, neurodegeneration