Search Results - "Alterman, Julia"

Designing novel RNA medicines for the brain by Alterman, Julia

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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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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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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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Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles by Haraszti, Reka Agnes, Miller, Rachael, Didiot, Marie-Cecile, Biscans, Annabelle, Alterman, Julia F., Hassler, Matthew R., Roux, Loic, Echeverria, Dimas, Sapp, Ellen, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

“…Extracellular vesicles are promising delivery vesicles for therapeutic RNAs. Small interfering RNA (siRNA) conjugation to cholesterol enables efficient and…”
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Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing by Mir, Aamir, Alterman, Julia F., Hassler, Matthew R., Debacker, Alexandre J., Hudgens, Edward, Echeverria, Dimas, Brodsky, Michael H., Khvorova, Anastasia, Watts, Jonathan K., Sontheimer, Erik J.

“…RNA-based drugs depend on chemical modifications to increase potency and to decrease immunogenicity in vivo. Chemical modification will likely improve the…”
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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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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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Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin by Tang, Qi, Fakih, Hassan H., Zain UI Abideen, Mohammad, Hildebrand, Samuel R., Afshari, Khashayar, Gross, Katherine Y., Sousa, Jacquelyn, Maebius, Allison S., Bartholdy, Christina, Søgaard, Pia Pernille, Jackerott, Malene, Hariharan, Vignesh, Summers, Ashley, Fan, Xueli, Okamura, Ken, Monopoli, Kathryn R., Cooper, David A., Echeverria, Dimas, Bramato, Brianna, McHugh, Nicholas, Furgal, Raymond C., Dresser, Karen, Winter, Sarah J., Biscans, Annabelle, Chuprin, Jane, Haddadi, Nazgol-Sadat, Sherman, Shany, Yıldız-Altay, Ümmügülsüm, Rashighi, Mehdi, Richmond, Jillian M., Bouix-Peter, Claire, Blanchard, Carine, Clauss, Adam, Alterman, Julia F., Khvorova, Anastasia, Harris, John E.

“…Inhibition of Janus kinase (JAK) family enzymes is a popular strategy for treating inflammatory and autoimmune skin diseases. In the clinic, small molecule JAK…”
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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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Visualization of self-delivering hydrophobically modified siRNA cellular internalization by Ly, Socheata, Navaroli, Deanna M, Didiot, Marie-Cécile, Cardia, James, Pandarinathan, Lakshmipathi, Alterman, Julia F, Fogarty, Kevin, Standley, Clive, Lifshitz, Lawrence M, Bellve, Karl D, Prot, Matthieu, Echeverria, Dimas, Corvera, Silvia, Khvorova, Anastasia

“…siRNAs are a new class of therapeutic modalities with promising clinical efficacy that requires modification or formulation for delivery to the tissue and cell…”
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An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis by Yenilmez, Batuhan, Wetoska, Nicole, Kelly, Mark, Echeverria, Dimas, Min, Kyounghee, Lifshitz, Lawrence, Alterman, Julia F., Hassler, Matthew R., Hildebrand, Samuel, DiMarzio, Chloe, McHugh, Nicholas, Vangjeli, Lorenc, Sousa, Jacquelyn, Pan, Meixia, Han, Xianlin, Brehm, Michael A., Khvorova, Anastasia, Czech, Michael P.

“…Nonalcoholic steatohepatitis (NASH) is a severe liver disorder characterized by triglyceride accumulation, severe inflammation, and fibrosis. With the recent…”
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Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1 by Davis, Sarah M., Hariharan, Vignesh N., Lo, Agnes, Turanov, Anton A., Echeverria, Dimas, Sousa, Jacquelyn, McHugh, Nicholas, Biscans, Annabelle, Alterman, Julia F., Karumanchi, S. Ananth, Moore, Melissa J., Khvorova, Anastasia

“…Preeclampsia (PE) is a rising, potentially lethal complication of pregnancy. PE is driven primarily by the overexpression of placental soluble fms-like…”
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Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors by Cheng, Shun-Yun, Caiazzi, Jillian, Biscans, Annabelle, Alterman, Julia F., Echeverria, Dimas, McHugh, Nicholas, Hassler, Matthew, Jolly, Samson, Giguere, Delaney, Cipi, Joris, Khvorova, Anastasia, Punzo, Claudio

“…Inherited retinal dystrophies caused by dominant mutations in photoreceptor (PR) cell expressed genes are a major cause of irreversible vision loss…”
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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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Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity by 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

“…Abstract Oligonucleotides is an emerging class of chemically-distinct therapeutic modalities, where extensive chemical modifications are fundamental for their…”
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Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs by Osborn, Maire F, Alterman, Julia F, Nikan, Mehran, Cao, Hong, Didiot, Marie C, Hassler, Matthew R, Coles, Andrew H, Khvorova, Anastasia

“…One of the major obstacles to the pharmaceutical success of oligonucleotide therapeutics (ONTs) is efficient delivery from the point of injection to the…”
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Enhancing siRNA efficacy in vivo with extended nucleic acid backbones by 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

“…Therapeutic small interfering RNA (siRNA) requires sugar and backbone modifications to inhibit nuclease degradation. However, metabolic stabilization by…”
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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain by Alterman, Julia F, Hall, Lauren M, Coles, Andrew H, Hassler, Matthew R, Didiot, Marie-Cecile, Chase, Kathryn, Abraham, Jasmin, Sottosanti, Emily, Johnson, Emily, Sapp, Ellen, Osborn, Maire F, Difiglia, Marian, Aronin, Neil, Khvorova, Anastasia

“…Applications of RNA interference for neuroscience research have been limited by a lack of simple and efficient methods to deliver oligonucleotides to primary…”
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Synthesis and biological properties of triazole-linked locked nucleic acid by Sharma, Vivek K, Singh, Sunil K, Krishnamurthy, Pranathi M, Alterman, Julia F, Haraszti, Reka A, Khvorova, Anastasia, Prasad, Ashok K, Watts, Jonathan K

“…We have synthesized and studied the biological and biophysical properties of triazole-linked ribo and xylo locked nucleic acid (LNA). The combination of LNA…”
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