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1Academic Journal
المؤلفون: 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
المصدر: Molecular Therapy: Nucleic Acids. 35(4)
مصطلحات موضوعية: CNS therapeutics, Huntington’s disease, MT: Oligonucleotides: Therapies and Applications, RNAi, brain delivery, genetic diseases, neurological disorders, oligonucleotide-based therapies, oligonucleotides
وصف الملف: application/pdf
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2Academic Journal
المؤلفون: Kloske, Courtney, Belloy, Michael, Blue, Elizabeth, Bowman, Gregory, Carrillo, Maria, Chen, Xiaoying, Chiba-Falek, Ornit, Davis, Albert, Paolo, Gilbert, Garretti, Francesca, Gate, David, Golden, Lesley, Heinecke, Jay, Herz, Joachim, Huang, Yadong, Iadecola, Costantino, Johnson, Lance, Kanekiyo, Takahisa, Karch, Celeste, Khvorova, Anastasia, Koppes-den Hertog, Sascha, Lamb, Bruce, Lawler, Paige, Guen, Yann, Litvinchuk, Alexandra, Liu, Chia-Chen, Mahinrad, Simin, Marcora, Edoardo, Marino, Claudia, Michaelson, Danny, Miller, Justin, Morganti, Josh, Narayan, Priyanka, Naslavsky, Michel, Oosthoek, Marlies, Ramachandran, Kapil, Ramakrishnan, Abhirami, Raulin, Ana-Caroline, Robert, Aiko, Saleh, Rasha, Sexton, Claire, Shah, Nilomi, Shue, Francis, Sible, Isabel, Soranno, Andrea, Strickland, Michael, Tcw, Julia, Thierry, Manon, Tsai, Li-Huei, Tuckey, Ryan, Ulrich, Jason, van der Kant, Rik, Wang, Na, Wellington, Cheryl, Weninger, Stacie, Yassine, Hussein, Zhao, Na, Bu, Guojun, Goate, Alison, Holtzman, David
المصدر: Alzheimers & Dementia: The Journal of the Alzheimers Association. 20(9)
مصطلحات موضوعية: APOE, Alzheimers disease, apolipoprotein E, conference proceedings, dementia, lipids, microglia, neuroinflammation, risk factor, therapeutics, vasculature, Humans, Apolipoproteins E, Alzheimer Disease, Congresses as Topic, Animals, Amyloid beta-Peptides, Dementia, Biomedical Research
وصف الملف: application/pdf
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3Academic Journal
المؤلفون: Lobov, Arseniy, Kuchur, Polina, Boyarskaya, Nadezhda, Perepletchikova, Daria, Taraskin, Ivan, Ivashkin, Andrei, Kostina, Daria, Khvorova, Irina, Uspensky, Vladimir, Repkin, Egor, Denisov, Evgeny, Gerashchenko, Tatiana, Tikhilov, Rashid, Bozhkova, Svetlana, Karelkin, Vitaly, Wang, Chunli, Xu, Kang, Malashicheva, Anna
المساهمون: Russian Science Foundation
المصدر: GigaScience ; volume 14 ; ISSN 2047-217X
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4Academic Journal
المؤلفون: Rachael Miller, Joseph Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna Bramato, Nozomi Yamada, Julia Alterman, Dimas Echeveria, Ken Yamada, Jonathan Watts, Christelle Anaclet, Marian DiFiglia, Anastasia Khvorova, Neil Aronin
المصدر: Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102359- (2024)
مصطلحات موضوعية: MT: Oligonucleotides: Therapies and Applications, oligonucleotides, oligonucleotide-based therapies, RNAi, neurological disorders, genetic diseases, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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5Academic Journal
المؤلفون: James W. Gilbert, Zachary Kennedy, Bruno M.D.C. Godinho, Ashley Summers, Alexandra Weiss, Dimas Echeverria, Brianna Bramato, Nicholas McHugh, David Cooper, Ken Yamada, Matthew Hassler, Hélène Tran, Fen Biao Gao, Robert H. Brown, Jr., Anastasia Khvorova
المصدر: Molecular Therapy: Nucleic Acids, Vol 35, Iss 3, Pp 102291- (2024)
مصطلحات موضوعية: MT: Oligonucleotides: Therapies and Applications, oligonucleotide therapeutics, siRNA, C9ORF72 amyotrophic lateral sclerosis, ALS, frontotemporal dementia, FTD, mRNA localization, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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6Academic Journal
المؤلفون: Vignesh N. Hariharan, Takahiro Nakamura, Minwook Shin, Qi Tang, Vyankat Sontakke, Jillian Caiazzi, Samuel Hildebrand, Anastasia Khvorova, Ken Yamada
المصدر: Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102230- (2024)
مصطلحات موضوعية: MT: Oligonucleotides: Therapies and Applications, RNA interference, lipid-conjugated siRNA, extrahepatic delivery, phosphocholine head group, chemical structure, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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7Academic Journal
المؤلفون: Qi Tang, Hassan H. Fakih, Mohammad Zain UI Abideen, Samuel R. Hildebrand, Khashayar Afshari, Katherine Y. Gross, Jacquelyn Sousa, Allison S. Maebius, Christina Bartholdy, Pia Pernille Søgaard, Malene Jackerott, Vignesh Hariharan, Ashley Summers, Xueli Fan, Ken Okamura, Kathryn R. Monopoli, David A. Cooper, Dimas Echeverria, Brianna Bramato, Nicholas McHugh, Raymond C. Furgal, Karen Dresser, Sarah J. Winter, Annabelle Biscans, Jane Chuprin, Nazgol-Sadat Haddadi, Shany Sherman, Ümmügülsüm Yıldız-Altay, Mehdi Rashighi, Jillian M. Richmond, Claire Bouix-Peter, Carine Blanchard, Adam Clauss, Julia F. Alterman, Anastasia Khvorova, John E. Harris
المصدر: Nature Communications, Vol 14, Iss 1, Pp 1-15 (2023)
مصطلحات موضوعية: Science
وصف الملف: electronic resource
Relation: https://doaj.org/toc/2041-1723
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8Academic Journal
المؤلفون: Kathryn R. Monopoli, Dmitry Korkin, Anastasia Khvorova
المصدر: Molecular Therapy: Nucleic Acids, Vol 33, Iss , Pp 93-109 (2023)
مصطلحات موضوعية: MT: Oligonucleotides: Therapies and Applications, siRNA, RNA interference, machine learning, artificial intelligence, oligonucleotides, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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9Academic Journal
المؤلفون: Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
المصدر: eLife, Vol 12 (2024)
مصطلحات موضوعية: NASH, NAFLD, stellate cells, liver fibrosis, RNAi therapeutics, AAV, Medicine, Science, Biology (General), QH301-705.5
وصف الملف: electronic resource
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10Academic Journal
المؤلفون: Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F. Alterman, Dimas Echeverria, Nicholas McHugh, Matthew Hassler, Samson Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo
المصدر: Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102088- (2024)
مصطلحات موضوعية: MT: Oligonucleotides: Therapies and Applications, siRNA, photoreceptors, gene silencing, IRD, inherited retinal dystrophy, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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11Academic Journal
المؤلفون: Qi Tang, Katherine Y. Gross, Hassan H. Fakih, Samuel O. Jackson, Mohammad Zain U.I. Abideen, Kathryn R. Monopoli, Carine Blanchard, Claire Bouix-Peter, Thibaud Portal, John E. Harris, Anastasia Khvorova, Julia F. Alterman
المصدر: Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102117- (2024)
مصطلحات موضوعية: MT: RNA/DNA Editing, immunomodulation, RNAi therapeutics, JAK1 sirna, multispecies targeting, inflammatory skin diseases, Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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12Academic Journal
المؤلفون: Saha, Krishanu, Sontheimer, Erik J, Brooks, PJ, Dwinell, Melinda R, Gersbach, Charles A, Liu, David R, Murray, Stephen A, Tsai, Shengdar Q, Wilson, Ross C, Anderson, Daniel G, Asokan, Aravind, Banfield, Jillian F, Bankiewicz, Krystof S, Bao, Gang, Bulte, Jeff WM, Bursac, Nenad, Campbell, Jarryd M, Carlson, Daniel F, Chaikof, Elliot L, Chen, Zheng-Yi, Cheng, R Holland, Clark, Karl J, Curiel, David T, Dahlman, James E, Deverman, Benjamin E, Dickinson, Mary E, Doudna, Jennifer A, Ekker, Stephen C, Emborg, Marina E, Feng, Guoping, Freedman, Benjamin S, Gamm, David M, Gao, Guangping, Ghiran, Ionita C, Glazer, Peter M, Gong, Shaoqin, Heaney, Jason D, Hennebold, Jon D, Hinson, John T, Khvorova, Anastasia, Kiani, Samira, Lagor, William R, Lam, Kit S, Leong, Kam W, Levine, Jon E, Lewis, Jennifer A, Lutz, Cathleen M, Ly, Danith H, Maragh, Samantha, McCray, Paul B, McDevitt, Todd C, Mirochnitchenko, Oleg, Morizane, Ryuji, Murthy, Niren, Prather, Randall S, Ronald, John A, Roy, Subhojit, Roy, Sushmita, Sabbisetti, Venkata, Saltzman, W Mark, Santangelo, Philip J, Segal, David J, Shimoyama, Mary, Skala, Melissa C, Tarantal, Alice F, Tilton, John C, Truskey, George A, Vandsburger, Moriel, Watts, Jonathan K, Wells, Kevin D, Wolfe, Scot A, Xu, Qiaobing, Xue, Wen, Yi, Guohua, Zhou, Jiangbing
المصدر: Nature. 592(7853)
مصطلحات موضوعية: Biological Sciences, Biomedical and Clinical Sciences, Genetics, Biotechnology, Human Genome, Gene Therapy, 5.2 Cellular and gene therapies, Generic health relevance, Animals, Cells, Gene Editing, Genetic Therapy, Genome, Human, Goals, Humans, National Institutes of Health (U.S.), United States, SCGE Consortium, General Science & Technology
وصف الملف: application/pdf
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13Academic Journal
المؤلفون: A. Kononov, S. Strekalova, E. Kobeleva, G. Savelyev, A. Zlygostev, M. Khvorova, V. Morozov, O. Babaeva, Y. Budnikova
المصدر: Current Research in Green and Sustainable Chemistry, Vol 8, Iss , Pp 100406- (2024)
مصطلحات موضوعية: Electro-oxidation, C–H functionalization, C–C coupling, C–N bond formation, Metal-free, Biaryl, Chemistry, QD1-999
وصف الملف: electronic resource
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14Academic Journal
المؤلفون: Kloske, Courtney M., Belloy, Michael E., Blue, Elizabeth E., Bowman, Gregory R., Carrillo, Maria C., Chen, Xiaoying, Chiba-Falek, Ornit, Davis, Albert A., Di Paolo, Gilbert, Garretti, Francesca, Gate, David, Golden, Lesley R., Heinecke, Jay W., Herz, Joachim, Huang, Yadong, Iadecola, Costantino, Johnson, Lance A., Kanekiyo, Takahisa, Karch, Celeste M., Khvorova, Anastasia, Koppes-den Hertog, Sascha J., Lamb, Bruce T., Lawler, Paige E., Le Guen, Yann, Litvinchuk, Alexandra, Liu, Chia-Chen, Mahinrad, Simin, Marcora, Edoardo, Marino, Claudia, Michaelson, Danny M., Miller, Justin J., Morganti, Josh M., Narayan, Priyanka S., Naslavsky, Michel S., Oosthoek, Marlies, Ramachandran, Kapil V., Ramakrishnan, Abhirami, Raulin, Ana-Caroline, Robert, Aiko, Saleh, Rasha N. M., Sexton, Claire, Shah, Nilomi, Shue, Francis, Sible, Isabel J., Soranno, Andrea, Strickland, Michael R., Tcw, Julia, Thierry, Manon, Tsai, Li-Huei, Tuckey, Ryan A., Ulrich, Jason D., van der Kant, Rik, Wang, Na, Wellington, Cheryl L., Weninger, Stacie C., Yassine, Hussein N., Zhao, Na, Bu, Guojun, Goate, Alison M., Holtzman, David M.
المساهمون: Neurology, School of Medicine
المصدر: PMC
مصطلحات موضوعية: APOE, Alzheimer's disease, Apolipoprotein E, Conference proceedings, Dementia, Lipids, Microglia, Neuroinflammation, Risk factor, Therapeutics, Vasculature
وصف الملف: application/pdf
Relation: Alzheimer's & Dementia; https://hdl.handle.net/1805/44538
الاتاحة: https://hdl.handle.net/1805/44538
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15Academic Journal
المؤلفون: 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
المساهمون: Morningside Graduate School of Biomedical Sciences, Psychiatry, RNA Therapeutics Institute, Samuel Hildebrand, Julianna Buchwald
المصدر: Alzheimer's & dementia : the journal of the Alzheimer's Association ; 20 ; 4 ; 2632 ; 2652 ; United States
مصطلحات موضوعية: Alzheimer's Disease, Apoe, RNAi, neurodegeneration, oligonucleotide therapeutics, siRNA
Relation: This article is based on a previously available preprint in bioRxiv, https://doi.org/10.1101/2022.06.28.498012.; Alzheimer’s & Dementia; https://doi.org/10.1002/alz.13703; Ferguson CM, Hildebrand S, Godinho BMDC, Buchwald J, Echeverria D, Coles A, Grigorenko A, Vangjeli L, Sousa J, McHugh N, Hassler M, Santarelli F, Heneka MT, Rogaev E, Khvorova A. Silencing Apoe with divalent-siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease. Alzheimers Dement. 2024 Apr;20(4):2632-2652. doi:10.1002/alz.13703. Epub 2024 Feb 20. PMID: 38375983; PMCID: PMC11032532.; http://hdl.handle.net/20.500.14038/53522; Alzheimer's & dementia : the journal of the Alzheimer's Association
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16Academic Journal
المؤلفون: Fakih, Hassan H, Tang, Qi, Summers, Ashley, Shin, Minwook, Buchwald, Julianna E, Gagnon, Rosemary, Hariharan, Vignesh N, Echeverria, Dimas, Cooper, David A, Watts, Jonathan K, Khvorova, Anastasia, Sleiman, Hanadi F
المساهمون: Morningside Graduate School of Biomedical Sciences, RNA Therapeutics Institute, Julianna E Buchwald, David A Cooper
المصدر: Molecular therapy. Nucleic acids ; 34 ; 102080 ; United States
مصطلحات موضوعية: MT: Delivery Strategies, albumin, drug delivery, oligonucleotide therapeutics, protein binding, siRNAs
Relation: Molecular Therapy Nucleic Acids; https://doi.org/10.1016/j.omtn.2023.102080; Fakih HH, Tang Q, Summers A, Shin M, Buchwald JE, Gagnon R, Hariharan VN, Echeverria D, Cooper DA, Watts JK, Khvorova A, Sleiman HF. Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity. Mol Ther Nucleic Acids. 2023 Nov 17;34:102080. doi:10.1016/j.omtn.2023.102080. PMID: 38089931; PMCID: PMC10711485.; http://hdl.handle.net/20.500.14038/53096; Molecular therapy. Nucleic acids
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17Academic Journal
المؤلفون: Zhang, Han, Kelly, Karen, Lee, Jonathan, Echeverria, Dimas, Cooper, David, Panwala, Rebecca, Amrani, Nadia, Chen, Zexiang, Gaston, Nicholas, Wagh, Atish, Newby, Gregory A, Xie, Jun, Liu, David R, Gao, Guangping, Wolfe, Scot A, Khvorova, Anastasia, Watts, Jonathan K, Sontheimer, Erik J
المساهمون: Biochemistry and Molecular Biotechnology, Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, Microbiology and Physiological Systems, Molecular, Cell and Cancer Biology, Morningside Graduate School of Biomedical Sciences, NeuroNexus Institute, Program in Molecular Medicine, RNA Therapeutics Institute, Han Zhang, David A Cooper, Rebecca Panwala, Nicholas Gaston
المصدر: Nucleic acids research ; 52 ; 2 ; 977 ; 997 ; United States ; England
Relation: Nucleic Acids Research; https://doi.org/10.1093/nar/gkad1125; Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Amrani N, Chen Z, Gaston N, Wagh A, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ. Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Nucleic Acids Res. 2024 Jan 25;52(2):977-997. doi:10.1093/nar/gkad1125. PMID: 38033325; PMCID: PMC10810193.; http://hdl.handle.net/20.500.14038/53101
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18Academic Journal
المساهمون: Morningside Graduate School of Biomedical Sciences, RNA Therapeutics Institute, David A Cooper
المصدر: Journal of controlled release : official journal of the Controlled Release Society ; 349 ; 831 ; 843 ; United States ; Netherlands
مصطلحات موضوعية: Endosomal escape, Ionizable lipid, Lipid conjugation, RNA interference, Therapeutic oligonucleotides, siRNA extrahepatic delivery
Relation: Journal of Controlled Release; https://doi.org/10.1016/j.jconrel.2022.07.041; Biscans A, Ly S, McHugh N, Cooper DA, Khvorova A. Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo. J Control Release. 2022 Sep;349:831-843. doi:10.1016/j.jconrel.2022.07.041. Epub 2022 Aug 3. PMID: 35917865; PMCID: PMC10281028.; http://hdl.handle.net/20.500.14038/52940; Journal of controlled release : official journal of the Controlled Release Society
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19Academic Journal
المؤلفون: Ferguson, Chantal M, Godinho, Bruno M D C, Echeverria, Dimas, Hassler, Matthew R, Vangjeli, Lorenc, Sousa, Jacquelyn, McHugh, Nicholas, Alterman, Julia F, Hariharan, Vignesh, Krishnamurthy, Pranathi Meda, Watts, Jonathan K, Rogaev, Evgeny I, Khvorova, Anastasia
المساهمون: Medicine, Psychiatry, RNA Therapeutics Institute
المصدر: Nucleic acids research ; 52 ; 9 ; 5273 ; 5284 ; United States ; England
Relation: Nucleic Acids Research; https://doi.org/10.1093/nar/gkae100; Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. A combinatorial approach for achieving CNS-selective RNAi. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi:10.1093/nar/gkae100. PMID: 38348876; PMCID: PMC11109952.; http://hdl.handle.net/20.500.14038/53542
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20Academic Journal
المؤلفون: Sarli, Samantha L, Fakih, Hassan H, Kelly, Karen, Devi, Gitali, Rembetsy-Brown, Julia M, McEachern, Holly R, Ferguson, Chantal M, Echeverria, Dimas, Lee, Jonathan, Sousa, Jacquelyn, Sleiman, Hanadi F, Khvorova, Anastasia, Watts, Jonathan K
المساهمون: Biochemistry and Molecular Biotechnology, Program in Molecular Medicine, RNA Therapeutics Institute
المصدر: Nucleic acids research ; 52 ; 9 ; 4799 ; 4817 ; United States ; England
Relation: Nucleic Acids Research; https://doi.org/10.1093/nar/gkae260; Sarli SL, Fakih HH, Kelly K, Devi G, Rembetsy-Brown JM, McEachern HR, Ferguson CM, Echeverria D, Lee J, Sousa J, Sleiman HF, Khvorova A, Watts JK. Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Res. 2024 May 22;52(9):4799-4817. doi:10.1093/nar/gkae260. PMID: 38613388; PMCID: PMC11109979.; http://hdl.handle.net/20.500.14038/53541