Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in vivo in CNS
Faith Conroy, Rachael Miller, Julia F. Alterman, Matthew R. Hassler, Dimas Echeverria, Bruno M.D.C. Godinho, Emily G. Knox, Ellen Sapp, Jaquelyn Sousa, Ken Yamada, Farah Mahmood, Adel Boudi, Kimberly B. Kegel-Gleason, Marian DiFiglia, Neil Aronin M. D, , Edith L. Pfister
This paper demonstrates allele-specific siRNA silencing of mutant huntingtin in CNS using SNP-based targeting in vitro and in vivo.
bioRxiv · Jun 29, 2022 · PreprintNeuroscience