Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier
Godinho, Bruno M.D.C., Henninger, Nils, Bouley, James, Alterman, Julia F., Haraszti, Reka A., Gilbert, James W., Sapp, Ellen, Coles, Andrew H., Biscans, Annabelle, Nikan, Mehran, Echeverria, Dimas, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Published in Molecular therapy (07.11.2018)
Published in Molecular therapy (07.11.2018)
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Journal Article
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs
Gilbert, James W., Kennedy, Zachary, Godinho, Bruno M.D.C., Summers, Ashley, Weiss, Alexandra, Echeverria, Dimas, Bramato, Brianna, McHugh, Nicholas, Cooper, David, Yamada, Ken, Hassler, Matthew, Tran, Hélène, Gao, Fen Biao, Brown, Robert H., Khvorova, Anastasia
Published in Molecular therapy. Nucleic acids (10.09.2024)
Published in Molecular therapy. Nucleic acids (10.09.2024)
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Journal Article
PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs
Godinho, Bruno M.D.C., Knox, Emily G., Hildebrand, Samuel, Gilbert, James W., Echeverria, Dimas, Kennedy, Zachary, Haraszti, Reka A., Ferguson, Chantal M., Coles, Andrew H., Biscans, Annabelle, Caiazzi, Jillian, Alterman, Julia F., Hassler, Matthew R., Khvorova, Anastasia
Published in Molecular therapy. Nucleic acids (13.09.2022)
Published in Molecular therapy. Nucleic acids (13.09.2022)
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Journal Article
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep
Ferguson, Chantal M, Godinho, Bruno Mdc, 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
Published in JCI insight (22.12.2021)
Published in JCI insight (22.12.2021)
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Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay
Godinho, Bruno M.D.C., Gilbert, James W., Haraszti, Reka A., Coles, Andrew H., Biscans, Annabelle, Roux, Loic, Nikan, Mehran, Echeverria, Dimas, Hassler, Matthew, Khvorova, Anastasia
Published in Nucleic acid therapeutics (01.12.2017)
Published in Nucleic acid therapeutics (01.12.2017)
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Journal Article
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Weiss, Alexandra, Gilbert, James W, Flores, Iris Valeria Rivera, Belgrad, Jillian, Ferguson, Chantal, Dogan, Elif O, Wightman, Nicholas, Mocarski, Kit, Echeverria, Dimas, Summers, Ashley, Bramato, Brianna, McHugh, Nicholas, Furgal, Raymond, Yamada, Nozomi, Cooper, David, Monopoli, Kathryn, Godinho, Bruno M D C, Hassler, Matthew R, Yamada, Ken, Greer, Paul L, Henninger, Nils, Brown, Robert H, Khvorova, Anastasia
Published in bioRxiv (25.06.2024)
Published in bioRxiv (25.06.2024)
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Journal Article
OLIGONUCLEOTIDES FOR SOD1 MODULATION
BROWN, Robert, FERGUSON, Chantal, WEISS, Alexandra, KHVOROVA, Anastasia, GILBERT, James W
Year of Publication 18.09.2024
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Year of Publication 18.09.2024
Patent
OLIGONUCLEOTIDES FOR SOD1 MODULATION
BROWN, Robert, FERGUSON, Chantal, WEISS, Alexandra, KHVOROVA, Anastasia, GILBERT, James W
Year of Publication 28.09.2023
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Year of Publication 28.09.2023
Patent
Cerebellitis in infectious mononucleosis
Gilbert, J W, Culebras, A
Published in JAMA : the journal of the American Medical Association (01.05.1972)
Published in JAMA : the journal of the American Medical Association (01.05.1972)
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Journal Article
OLIGONUCLEOTIDES FOR SOD1 MODULATION
Gilbert, James W, Brown, Robert, Weiss, Alexandra, Khvorova, Anastasia, Ferguson, Chantal
Year of Publication 22.06.2023
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Year of Publication 22.06.2023
Patent
OLIGONUCLEOTIDES FOR SOD1 MODULATION
BROWN, Robert, FERGUSON, Chantal, WEISS, Alexandra, KHVOROVA, Anastasia, GILBERT, James W
Year of Publication 11.05.2023
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Year of Publication 11.05.2023
Patent