Search Results - "Godinho, Bruno M.D.C."

Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O‑Phosphocholine‑N‑docosahexaenoyl‑l‑serine siRNA Conjugate in Mouse Brain by Nikan, Mehran, Osborn, Maire F, Coles, Andrew H, Biscans, Annabelle, Godinho, Bruno M.D.C, Haraszti, Reka A, Sapp, Ellen, Echeverria, Dimas, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

“…Ligand-conjugated siRNAs have the potential to achieve targeted delivery and efficient silencing in neurons following local administration in the central…”
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Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier by 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

“…Effective transvascular delivery of therapeutic oligonucleotides to the brain presents a major hurdle to the development of gene silencing technologies for…”
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Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition by Haraszti, Reka Agnes, Miller, Rachael, Dubuke, Michelle L, Rockwell, Hannah E, Coles, Andrew H, Sapp, Ellen, Didiot, Marie-Cecile, Echeverria, Dimas, Stoppato, Matteo, Sere, Yves Y, Leszyk, John, Alterman, Julia F, Godinho, Bruno M D C, Hassler, Matthew R, McDaniel, Justice, Narain, Niven R, Wollacott, Rachel, Wang, Yang, Shaffer, Scott A, Kiebish, Michael A, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

“…Exosomes can serve as delivery vehicles for advanced therapeutics. The components necessary and sufficient to support exosomal delivery have not been…”
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Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs by 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

“…A hexanucleotide (G4C2) repeat expansion (HRE) within intron one of C9ORF72 is the leading genetic cause of amyotrophic lateral sclerosis (ALS) and…”
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Differential nanotoxicological and neuroinflammatory liabilities of non-viral vectors for RNA interference in the central nervous system by Godinho, Bruno M.D.C, McCarthy, David J, Torres-Fuentes, Cristina, Beltrán, Caroll J, McCarthy, Joanna, Quinlan, Aoife, Ogier, Julien R, Darcy, Raphael, O'Driscoll, Caitriona M, Cryan, John F

“…Abstract Progression of RNA interference-based gene silencing technologies for the treatment of disorders of the central nervous system (CNS) depends on the…”
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PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs by 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

“…Effective systemic delivery of small interfering RNAs (siRNAs) to tissues other than liver remains a challenge. siRNAs are small (∼15 kDa) and therefore…”
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5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo by Haraszti, Reka A., Roux, Loic, Coles, Andrew H., Turanov, Anton A., Alterman, Julia F., Echeverria, Dimas, Godinho, Bruno M.D.C., Aronin, Neil, Khvorova, Anastasia

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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep by 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

“…siRNAs comprise a class of drugs that can be programmed to silence any target gene. Chemical engineering efforts resulted in development of divalent siRNAs…”
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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing by Didiot, Marie-Cécile, Hall, Lauren M, Coles, Andrew H, Haraszti, Reka A, Godinho, Bruno MDC, Chase, Kathryn, Sapp, Ellen, Ly, Socheata, Alterman, Julia F, Hassler, Matthew R, Echeverria, Dimas, Raj, Lakshmi, Morrissey, David V, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

“…Delivery represents a significant barrier to the clinical advancement of oligonucleotide therapeutics for the treatment of neurological disorders, such as…”
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Intrathecal Delivery of Therapeutic Oligonucleotides for Potent Modulation of Gene Expression in the Central Nervous System by Kennedy, Zachary, Gilbert, James W, Godinho, Bruno M D C

“…Therapeutic oligonucleotides hold tremendous potential for treating central nervous system (CNS) disorders. The route of administration of oligonucleotides…”
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RNAi modulation of placental sFLT1 for the treatment of preeclampsia by Turanov, Anton A, Lo, Agnes, Hassler, Matthew R, Makris, Angela, Ashar-Patel, Ami, Alterman, Julia F, Coles, Andrew H, Haraszti, Reka A, Roux, Loic, Godinho, Bruno M D C, Echeverria, Dimas, Pears, Suzanne, Iliopoulos, Jim, Shanmugalingam, Renuka, Ogle, Robert, Zsengeller, Zsuzsanna K, Hennessy, Annemarie, Karumanchi, S Ananth, Moore, Melissa J, Khvorova, Anastasia

“…Preeclampsia, a hypertension condition with no approved therapies, is treated in monkeys by gene silencing in the placenta. Preeclampsia is a placentally…”
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5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo by Haraszti, Reka A, Roux, Loic, Coles, Andrew H, Turanov, Anton A, Alterman, Julia F, Echeverria, Dimas, Godinho, Bruno M D C, Aronin, Neil, Khvorova, Anastasia

“…5΄-Vinylphosphonate modification of siRNAs protects them from phosphatases, and improves silencing activity. Here, we show that 5΄-vinylphosphonate confers…”
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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system by 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

“…Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA…”
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Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways by Osborn, Maire F, Coles, Andrew H, Biscans, Annabelle, Haraszti, Reka A, Roux, Loic, Davis, Sarah, Ly, Socheata, Echeverria, Dimas, Hassler, Matthew R, Godinho, Bruno M D C, Nikan, Mehran, Khvorova, Anastasia

“…Abstract Efficient delivery of therapeutic RNA beyond the liver is the fundamental obstacle preventing its clinical utility. Lipid conjugation increases plasma…”
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Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo by Hassler, Matthew R, Turanov, Anton A, Alterman, Julia F, Haraszti, Reka A, Coles, Andrew H, Osborn, Maire F, Echeverria, Dimas, Nikan, Mehran, Salomon, William E, Roux, Loïc, Godinho, Bruno M D C, Davis, Sarah M, Morrissey, David V, Zamore, Phillip D, Karumanchi, S Ananth, Moore, Melissa J, Aronin, Neil, Khvorova, Anastasia

“…Abstract Small interfering RNA (siRNA)-based drugs require chemical modifications or formulation to promote stability, minimize innate immunity, and enable…”
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A combinatorial approach for achieving CNS-selective RNAi by Ferguson, Chantal M, Godinho, Bruno M D C, Echeverria, Dimas, Hassler, Matthew, Vangjeli, Lorenc, Sousa, Jacquelyn, McHugh, Nicholas, Alterman, Julia, Hariharan, Vignesh, Krishnamurthy, Pranathi Meda, Watts, Jonathan, Rogaev, Eveny, Khvorova, Anastasia

“…RNA interference (RNAi) is an endogenous process that can be harnessed using chemically modified small interfering RNAs (siRNAs) to potently modulate gene…”
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Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection by Hariharan, Vignesh N, Shin, Minwook, Chang, Ching-Wen, O'Reilly, Daniel, Biscans, Annabelle, Yamada, Ken, Guo, Zhiru, Somasundaran, Mohan, Tang, Qi, Monopoli, Kathryn, Krishnamurthy, Pranathi Meda, Devi, Gitali, McHugh, Nicholas, Cooper, David A, Echeverria, Dimas, Cruz, John, Chan, Io Long, Liu, Ping, Lim, Sun-Young, McConnell, Jill, Singh, Satya Prakash, Hildebrand, Samuel, Sousa, Jacquelyn, Davis, Sarah M, Kennedy, Zachary, Ferguson, Chantal, Godinho, Bruno M D C, Thillier, Yann, Caiazzi, Jillian, Ly, Socheata, Muhuri, Manish, Kelly, Karen, Humphries, Fiachra, Cousineau, Alyssa, Parsi, Krishna Mohan, Li, Qi, Wang, Yang, Maehr, René, Gao, Guangping, Korkin, Dmitry, McDougall, William M, Finberg, Robert W, Fitzgerald, Katherine A, Wang, Jennifer P, Watts, Jonathan K, Khvorova, Anastasia

“…The continuous evolution of SARS-CoV-2 variants complicates efforts to combat the ongoing pandemic, underscoring the need for a dynamic platform for the rapid…”
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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models by 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.

“…Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently…”
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Silencing Apoe with divalent‐siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease by Ferguson, Chantal M., Hildebrand, Samuel, Godinho, Bruno M. D. C., Buchwald, Julianna, Echeverria, Dimas, Coles, Andrew, Grigorenko, Anastasia, Vangjeli, Lorenc, Sousa, Jacquelyn, McHugh, Nicholas, Hassler, Matthew, Santarelli, Francesco, Heneka, Michael T., Rogaev, Evgeny, Khvorova, Anastasia

“…INTRODUCTION The most significant genetic risk factor for late‐onset Alzheimer's disease (AD) is APOE4, with evidence for gain‐ and loss‐of‐function…”
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Self-assembling Modified β‑Cyclodextrin Nanoparticles as Neuronal siRNA Delivery Vectors: Focus on Huntington’s Disease by Godinho, Bruno M. D. C, Ogier, Julien R, Darcy, Raphael, O’Driscoll, Caitriona M, Cryan, John F

“…Huntington’s disease (HD) is a rare autosomal dominant neurodegenerative disease caused by the expression of a toxic Huntingtin (HTT) protein. The use of short…”
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