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Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+and Mg2+in the formulation Miller, Rachael; Paquette, Joseph; Barker, Alexandra; Sapp, Ellen; McHugh, Nicholas; Bramato, Brianna; Yamada, Nozomi; Alterman, Julia; Echeveria, Dimas; Yamada, Ken; Watts, Jonathan; Anaclet, Christelle; DiFiglia, Marian; Khvorova, Anastasia; Aronin, Neil 分享 收藏
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo Allen, Sarah; O'Reilly, Daniel; Miller, Rachael; Sapp, Ellen; Summers, Ashley; Paquette, Joseph; Moreno, Dimas Echeverria; Bramato, Brianna; McHugh, Nicholas; Yamada, Ken; Aronin, Neil; DiFiglia, Marian; Khvorova, Anastasia 分享 收藏
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones Yamada, Ken; Hariharan, Vignesh N.; Caiazzi, Jillian; Miller, Rachael; Ferguson, Chantal M.; Sapp, Ellen; Fakih, Hassan H.; Tang, Qi; Yamada, Nozomi; Furgal, Raymond C.; Paquette, Joseph D.; Biscans, Annabelle; Bramato, Brianna M.; Mchugh, Nicholas; Summers, Ashley; Lochmann, Clemens; Godinho, Bruno M. D. C.; Hildebrand, Samuel; Jackson, Samuel O.; Echeverria, Dimas; Hassler, Matthew R.; Alterman, Julia F.; Difiglia, Marian; Aronin, Neil; Khvorova, Anastasia 分享 收藏
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A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system 可编程双靶向siRNA支架支持中枢神经系统中有效的两基因调节 Belgrad, Jillian; Tang, Qi; Hildebrand, Sam; Summers, Ashley; Sapp, Ellen; Echeverria, Dimas; O'Reilly, Dan; Luu, Eric; Bramato, Brianna; Allen, Sarah; Cooper, David; Alterman, Julia; Yamada, Ken; Aronin, Neil; Difiglia, Marian; Khvorova, Anastasia 分享 收藏
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease (may, 10.1016/j.ymthe.2023.05.006, 2023) O'Reilly, Daniel; Belgrad, Jillian; Ferguson, Chantal; Summers, Ashley; Sapp, Ellen; McHugh, Cassandra; Mathews, Ella; Boudi, Adel; Buchwald, Julianna; Ly, Socheata; Moreno, Dimas; Furgal, Raymond; Luu, Eric; Kennedy, Zachary; Hariharan, Vignesh; Monopoli, Kathryn; Yang, X. William; Carroll, Jeffery; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia 分享 收藏
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Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease O 'Reilly, Daniel; Belgrad, Jillian; Ferguson, Chantal; Summers, Ashley; Sapp, Ellen; McHugh, Cassandra; Mathews, Ella; Boudi, Adel; Buchwald, Julianna; Ly, Socheata; Moreno, Dimas; Furgal, Raymond; Luu, Eric; Kennedy, Zachary; Hariharan, Vignesh; Monopoli, Kathryn; Yang, William; Carroll, Jeffery; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia 分享 收藏
Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity Li, Xueyi; Standley, Clive; Sapp, Ellen; Valencia, Antonio; Qin, Zheng-Hong; Kegel, Kimberly B.; Yoder, Jennifer; Comer-Tierney, Laryssa A.; Esteves, Miguel; Chase, Kathryn; Alexander, Jonathan; Masso, Nicholas; Sobin, Lindsay; Bellve, Karl; Tuft, Richard; Lifshitz, Lawrence; Fogarty, Kevin; Aronin, Neil; DiFiglia, Marian 分享 收藏
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models 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. 分享 收藏
Impaired XK recycling for importing manganese underlies striatal vulnerability in Huntington's disease Chhetri, Gaurav; Ke, Yuting; Wang, Ping; Usman, Muhammad; Li, Yan; Sapp, Ellen; Wang, Jing; Ghosh, Arabinda; Islam, Md Ariful; Wang, Xiaolong; Boudi, Adel; DiFiglia, Marian; Li, Xueyi 分享 收藏
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity 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 分享 收藏
Promise and challenges of dystonia brain banking: establishing a human tissue repository for studies of X-Linked Dystonia-Parkinsonism Fernandez-Cerado, Cara; Legarda, G. Paul; Velasco-Andrada, M. Salvie; Aguil, Abegail; Ganza-Bautista, Niecy G.; Lagarde, J. Benedict B.; Soria, Jasmin; Jamora, Roland Dominic G.; Acuna, Patrick J.; Vanderburg, Charles; Sapp, Ellen; DiFiglia, Marian; Murcar, Micaela G.; Campion, Lindsey; Ozelius, Laurie J.; Alessi, Amy K.; Singh-Bains, Malvindar K.; Waldvogel, Henry J.; Faull, Richard L. M.; Macalintal-Canlas, Regina; Munoz, Edwin L.; Penney, Ellen B.; Ang, Mark A.; Diesta, Cid Czarina E.; Bragg, D. Cristopher; Acuna-Sunshine, Geraldine 分享 收藏
Trappc9 deficiency in mice impairs learning and memory by causing imbalance of dopamine D1 and D2 neurons Ke, Yuting; Weng, Meiqian; Chhetri, Gaurav; Usman, Muhammad; Li, Yan; Yu, Qing; Ding, Yingzhuo; Wang, Zejian; Wang, Xiaolong; Sultana, Pinky; DiFiglia, Marian; Li, Xueyi 分享 收藏
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Assessing average somatic CAG repeat instability at the protein level Aviolat, Hubert; Pinto, Ricardo Mouro; Godschall, Elizabeth; Murtha, Ryan; Richey, Hannah E.; Sapp, Ellen; Vodicka, Petr; Wheeler, Vanessa C.; Kegel-Gleason, Kimberly B.; DiFiglia, Marian 分享 收藏
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system 二价siRNA化学支架，用于有效和持续地调节整个中枢神经系统的基因表达 Alterman, Julia F.; Godinho, Bruno M. D. C.; Hassler, Matthew R.; Ferguson, Chantal M.; Echeverria, Dimas; Sapp, Ellen; Haraszti, Reka A.; Coles, Andrew H.; Conroy, Faith; Miller, Rachael; Roux, Loic; Yan, Paul; Knox, Emily G.; Turanov, Anton A.; King, Robert M.; Gernoux, Gwladys; Mueller, Christian; Gray-Edwards, Heather L.; Moser, Richard P.; Bishop, Nina C.; Jaber, Samer M.; Gounis, Matthew J.; Sena-Esteves, Miguel; Pai, Athma A.; DiFiglia, Marian; Aronin, Neil; Khvorova, Anastasia 分享 收藏
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