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
Published in Nature communications (03.10.2022)
Published in Nature communications (03.10.2022)
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Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin
Pfister, Edith L., Chase, Kathryn O., Sun, Huaming, Kennington, Lori A., Conroy, Faith, Johnson, Emily, Miller, Rachael, Borel, Florie, Aronin, Neil, Mueller, Christian
Published in Molecular therapy. Nucleic acids (16.06.2017)
Published in Molecular therapy. Nucleic acids (16.06.2017)
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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
Published in Nature biotechnology (01.08.2019)
Published in Nature biotechnology (01.08.2019)
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Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease
Pfister, Edith L, DiNardo, Natalie, Mondo, Erica, Borel, Florie, Conroy, Faith, Fraser, Cara, Gernoux, Gwladys, Han, Xin, Hu, Danjing, Johnson, Emily, Kennington, Lori, Liu, PengPeng, Reid, Suzanne J, Sapp, Ellen, Vodicka, Petr, Kuchel, Tim, Morton, A Jennifer, Howland, David, Moser, Richard, Sena-Esteves, Miguel, Gao, Guangping, Mueller, Christian, DiFiglia, Marian, Aronin, Neil
Published in Human gene therapy (01.06.2018)
Published in Human gene therapy (01.06.2018)
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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
Published in Nucleic acids research (02.12.2021)
Published in Nucleic acids research (02.12.2021)
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SNP를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
PFISTER EDITH, ALTERMAN JULIA, ARONIN NEIL, CONROY FAITH, KHVOROVA ANASTASIA, YAMADA KEN
Year of Publication 19.04.2022
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Year of Publication 19.04.2022
Patent
SNP를 표적화하는 변형된 올리고뉴클레오티드
PFISTER EDITH, ALTERMAN JULIA, ARONIN NEIL, CONROY FAITH, KHVOROVA ANASTASIA, YAMADA KEN
Year of Publication 27.07.2021
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Year of Publication 27.07.2021
Patent
Modified oligonucleotides targeting SNPs
Alterman, Julia, Pfister, Edith, Aronin, Neil, Yamada, Ken, Khvorova, Anastasia, Conroy, Faith
Year of Publication 02.07.2024
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Year of Publication 02.07.2024
Patent
MODIFIED OLIGONUCLEOTIDES TARGETING SNPs
Alterman, Julia, Pfister, Edith, Aronin, Neil, Yamada, Ken, Khvorova, Anastasia, Conroy, Faith
Year of Publication 25.04.2024
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Year of Publication 25.04.2024
Patent
CHEMICALLY MODIFIED OLIGONUCLEOTIDES TARGETING SNPS
PFISTER, Edith, KHVOROVA, Anastasia, ALTERMAN, Julia, CONROY, Faith, ARONIN, Neil, YAMADA, Ken
Year of Publication 27.12.2023
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Year of Publication 27.12.2023
Patent
OLIGONUCLEOTIDES FOR HTT-1A MODULATION
Ly, Socheata, Alterman, Julia, O'Reilly, Daniel, Khvorova, Anastasia, Conroy, Faith
Year of Publication 24.03.2022
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Year of Publication 24.03.2022
Patent
Modified oligonucleotides targeting SNPs
Alterman, Julia, Pfister, Edith, Aronin, Neil, Yamada, Ken, Khvorova, Anastasia, Conroy, Faith
Year of Publication 28.11.2023
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Year of Publication 28.11.2023
Patent
MODIFIED OLIGONUCLEOTIDES TARGETING SNPS
PFISTER, Edith, KHVOROVA, Anastasia, ALTERMAN, Julia, CONROY, Faith, ARONIN, Neil, YAMADA, Ken
Year of Publication 19.07.2023
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Year of Publication 19.07.2023
Patent
OLIGONUCLEOTIDES FOR HTT-1A MODULATION
KHVOROVA, Anastasia, LY, Socheata, ALTERMAN, Julia, CONROY, Faith, O'REILLY, Daniel, ALLEN, Sarah
Year of Publication 20.04.2023
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Year of Publication 20.04.2023
Patent
OLIGONUCLEOTIDES FOR HTT-1A MODULATION
KHVOROVA, Anastasia, LY, Socheata, ALTERMAN, Julia, CONROY, Faith, O'REILLY, Daniel, ALLEN, Sarah
Year of Publication 09.02.2023
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Year of Publication 09.02.2023
Patent
CHEMICALLY MODIFIED OLIGONUCLEOTIDES TARGETING SNPS
PFISTER, Edith, KHVOROVA, Anastasia, ALTERMAN, Julia, CONROY, Faith, ARONIN, Neil, YAMADA, Ken
Year of Publication 15.06.2022
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Year of Publication 15.06.2022
Patent
OLIGONUCLEOTIDES FOR HTT-1A MODULATION
KHVOROVA, Anastasia, LY, Socheata, ALTERMAN, Julia, CONROY, Faith, O'REILLY, Daniel
Year of Publication 31.03.2022
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Year of Publication 31.03.2022
Patent
Chemically modified oligonucleotides targeting SNPs
PFISTER, Edith, KHVOROVA, Anastasia, ALTERMAN, Julia, CONROY, Faith, ARONIN, Neil, YAMADA, Ken
Year of Publication 10.03.2022
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Year of Publication 10.03.2022
Patent
OLIGONUCLEOTIDES FOR HTT-1A MODULATION
KHVOROVA, Anastasia, LY, Socheata, ALTERMAN, Julia, CONROY, Faith, O'REILLY, Daniel
Year of Publication 10.02.2022
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Year of Publication 10.02.2022
Patent
MODIFIED OLIGONUCLEOTIDES TARGETING SNPS
PFISTER, Edith, KHVOROVA, Anastasia, ALTERMAN, Julia, CONROY, Faith, ARONIN, Neil, YAMADA, Ken
Year of Publication 16.06.2021
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Year of Publication 16.06.2021
Patent