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ESC derived human cortical neurons harboring the NACC1 c.892C > T p.R298W missense mutation exhibit molecular differences from controls that influence neuronal maturation Deehan, Mark; Sapp, Ellen; Iwanowicz, Andrew; Kothuis, Josine; Weisman, Elizabeth; Liu, Sophia; Jones, Erin; Iuliano, Maria; Robertson, Riannon; Seeley, Connor; Li, Zhaozhi; Noori, Ayush; Li, Xueyi; Das, Sudeshna; Brodsky, Michael; Aronin, Neil; DiFiglia, Marian; Kegel-Gleason, Kimberly B. Share Save
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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 Share Save
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 Share Save
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 Share Save
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system 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 Share Save
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 Share Save
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 Share Save
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 Share Save
Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains Ly, Socheata; Didiot, Marie-Cecile; Ferguson, Chantal M.; Coles, Andrew H.; Miller, Rachael; Chase, Kathryn; Echeverria, Dimas; Wang, Feng; Sadri-Vakili, Ghazaleh; Aronin, Neil; Khvorova, Anastasia Share Save
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. Share Save
A RET::GRB2 fusion in pheochromocytoma defies the classic paradigm of RET oncogenic fusions Estrada-Zuniga, Cynthia M.; Cheng, Zi-Ming; Ethiraj, Purushoth; Guo, Qianjin; Gonzalez-Cantu, Hector; Adderley, Elaina; Lopez, Hector; Landry, Bethany N.; Zainal, Abir; Aronin, Neil; Ding, Yanli; Wang, Xiaojing; Aguiar, Ricardo C. T.; Dahia, Patricia L. M. Share Save
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset Ferguson, Chantal M.; Godinho, Bruno M. D. C.; Alterman, Julia F.; Coles, Andrew H.; Hassler, Matthew; Echeverria, Dimas; Gilbert, James W.; Knox, Emily G.; Caiazzi, Jillian; Haraszti, Reka A.; King, Robert M.; Taghian, Toloo; Puri, Ajit; Moser, Richard P.; Gounis, Matthew J.; Aronin, Neil; Gray-Edwards, Heather; Khvorova, Anastasia Share Save
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 Share Save
Genotype-Phenotype Features of Germline Variants of the TMEM127 Pheochromocytoma Susceptibility Gene: A 10-Year Update Armaiz-Pena, Gustavo; Flores, Shahida K.; Cheng, Zi-Ming; Zhang, Xhingyu; Esquivel, Emmanuel; Poullard, Natalie; Vaidyanathan, Anusha; Liu, Qianqian; Michalek, Joel; Santillan-Gomez, Alfredo A.; Liss, Michael; Ahmadi, Sara; Katselnik, Daniel; Maldonado, Enrique; Salgado, Sarimar Agosto; Jimenez, Camilo; Fishbein, Lauren; Hamidi, Oksana; Else, Tobias; Lechan, Ron; Tischler, Art S.; Benn, Diana E.; Dwight, Trisha; Clifton-Bligh, Rory; Sanso, Gabriela; Barontini, Marta; Vincent, Deepa; Aronin, Neil; Biondi, Bernadette; Koops, Maureen; Bowhay-Carnes, Elizabeth; Gimenez-Roqueplo, Anne-Paule; Alvarez-Eslava, Andrea; Bruder, Jan M.; Kitano, Mio; Burnichon, Nelly; Ding, Yanli; Dahia, Patricia L. M. Share Save
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A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna Taghian, Toloo; Marosfoi, Miklos G.; Puri, Ajit S.; Cataltepe, Oguz. I.; King, Robert M.; Diffie, Elise B.; Maguire, Anne S.; Martin, Douglas R.; Fernau, Deborah; Batista, Ana Rita; Kuchel, Tim; Christou, Chris; Perumal, Raj; Chandra, Sundeep; Gamlin, Paul D.; Bertrand, Stephanie G.; Flotte, Terence R.; McKenna-Yasek, Diane; Tai, Phillip W. L.; Aronin, Neil; Gounis, Matthew J.; Sena-Esteves, Miguel; Gray-Edwards, Heather L. Share Save
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system 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 Share Save
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