Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin

por Pfister, Edith L., Chase, Kathryn O., Sun, Huaming, Kennington, Lori A., Conroy, Faith, Johnson, Emily, Miller, Rachael, Borel, Florie, Aronin, Neil, Mueller, Christian
Publicado 2017

Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells

por Miller, James R. C., Pfister, Edith L., Liu, Wanzhao, Andre, Ralph, Träger, Ulrike, Kennington, Lori A., Lo, Kimberly, Dijkstra, Sipke, Macdonald, Douglas, Ostroff, Gary, Aronin, Neil, Tabrizi, Sarah J.
Publicado 2017

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity

por Yamada, Ken, Hildebrand, Samuel, Davis, Sarah M, Miller, Rachael, Conroy, Faith, Sapp, Ellen, Caiazzi, Jillian, Alterman, Julia F, Roux, Loic, Echeverria, Dimas, Hassler, Matthew R, Pfister, Edith L, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Publicado 2021

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

por Conroy, Faith, Miller, Rachael, Alterman, Julia F., Hassler, Matthew R., Echeverria, Dimas, Godinho, Bruno M. D. C., Knox, Emily G., Sapp, Ellen, Sousa, Jaquelyn, Yamada, Ken, Mahmood, Farah, Boudi, Adel, Kegel-Gleason, Kimberly, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Pfister, Edith L.
Publicado 2022