Search Results - "NICHOLS, Timothy C"

Targeted disruption of LDLR causes hypercholesterolemia and atherosclerosis in Yucatan miniature pigs by Davis, Bryan T, Wang, Xiao-Jun, Rohret, Judy A, Struzynski, Jason T, Merricks, Elizabeth P, Bellinger, Dwight A, Rohret, Frank A, Nichols, Timothy C, Rogers, Christopher S

“…Recent progress in engineering the genomes of large animals has spurred increased interest in developing better animal models for diseases where current…”
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Preclinical evaluation of a next-generation, subcutaneously administered, coagulation factor IX variant, dalcinonacog alfa by Nichols, Timothy C, Levy, Howard, Merricks, Elizabeth P, Raymer, Robin A, Lee, Martin L

“…The rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B…”
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Intrinsic and acquired drug resistance to LSD1 inhibitors in small cell lung cancer occurs through a TEAD4‐driven transcriptional state by Yan, Wen, Chung, Chi‐Yeh, Xie, Tao, Ozeck, Mark, Nichols, Timothy C., Frey, Jessica, Udyavar, Akshata R., Sharma, Shikhar, Paul, Thomas A.

“…Small‐cell lung cancer (SCLC) is a heterogeneous disease, consisting of intratumoral and intertumoral neuroendocrine (ASCL1 and/or NEUROD1), mesenchymal‐like,…”
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Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs by Dumont, Jennifer A., Liu, Tongyao, Low, Susan C., Zhang, Xin, Kamphaus, George, Sakorafas, Paul, Fraley, Cara, Drager, Douglas, Reidy, Thomas, McCue, Justin, Franck, Helen W.G., Merricks, Elizabeth P., Nichols, Timothy C., Bitonti, Alan J., Pierce, Glenn F., Jiang, Haiyan

“…Despite proven benefits, prophylactic treatment for hemophilia A is hampered by the short half-life of factor VIII. A recombinant factor VIII-Fc fusion protein…”
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Prolonged activity of factor IX as a monomeric Fc fusion protein by Peters, Robert T., Low, Susan C., Kamphaus, George D., Dumont, Jennifer A., Amari, John V., Lu, Qi, Zarbis-Papastoitsis, Greg, Reidy, Thomas J., Merricks, Elizabeth P., Nichols, Timothy C., Bitonti, Alan J.

“…Treatment of hemophilia B requires frequent infusions of factor IX (FIX) to prophylax against bleeding episodes. Hemophilia B management would benefit from a…”
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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy by Finn, Jonathan D., Ozelo, Margareth C., Sabatino, Denise E., Franck, Helen W.G., Merricks, Elizabeth P., Crudele, Julie M., Zhou, Shangzhen, Kazazian, Haig H., Lillicrap, David, Nichols, Timothy C., Arruda, Valder R.

“…Inhibitory antibodies to factor VIII (FVIII) are a major complication in the treatment of hemophilia A, affecting approximately 20% to 30% of patients. Current…”
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Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide by Østergaard, Henrik, Bjelke, Jais R., Hansen, Lene, Petersen, Lars Christian, Pedersen, Anette A., Elm, Torben, Møller, Flemming, Hermit, Mette B., Holm, Pernille K., Krogh, Thomas N., Petersen, Jørn M., Ezban, Mirella, Sørensen, Brit B., Andersen, Mette D., Agersø, Henrik, Ahmadian, Haleh, Balling, Kristoffer W., Christiansen, Marie Louise S., Knobe, Karin, Nichols, Timothy C., Bjørn, Søren E., Tranholm, Mikael

“…Current management of hemophilia B entails multiple weekly infusions of factor IX (FIX) to prevent bleeding episodes. In an attempt to make a longer acting…”
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Mechanisms of Foreign Body Response Mitigation by Nitric Oxide Release by Taylor, James B, Malone-Povolny, Maggie J, Merricks, Elizabeth P, Wimsey, Lauren E, Soliman, Daniel, Nichols, Timothy C, Wallet, Shannon M, Maile, Robert, Schoenfisch, Mark H

“…Implantable glucose biosensors provide real-time information about blood glucose fluctuations, but their utility and accuracy are time-limited due to the…”
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Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors by Sabatino, Denise E, Lange, Amy M, Altynova, Ekaterina S, Sarkar, Rita, Zhou, Shangzhen, Merricks, Elizabeth P, Franck, Helen G, Nichols, Timothy C, Arruda, Valder R, Kazazian Jr, Haig H

“…Developing adeno-associated viral (AAV)–mediated gene therapy for hemophilia A (HA) has been challenging due to the large size of the factor VIII (FVIII)…”
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Chimeric Mice Engrafted With Canine Hepatocytes Exhibits Similar AAV Transduction Efficiency to Hemophilia B Dog by Shao, Wenwei, Sun, Junjiang, Chen, Xiaojing, Dobbins, Amanda, Merricks, Elizabeth P, Samulski, R Jude, Nichols, Timothy C, Li, Chengwen

“…Adeno-associated virus (AAV) mediated gene therapy has been successfully applied in clinical trials, including hemophilia. Novel AAV vectors have been…”
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Adeno-associated virus-mediated expression of activated factor V (FVa) for hemophilia phenotypic correction by Sun, Junjiang, Chen, Xiaojing, Chai, Zheng, Niu, Hongqian, Dobbins, Amanda L, Nichols, Timothy C, Li, Chengwen

“…Adeno-associated virus (AAV) gene therapy has been successfully applied in hemophilia patients excluding patients with inhibitors. During the coagulation…”
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Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B by Arruda, Valder R., Stedman, Hansell H., Haurigot, Virginia, Buchlis, George, Baila, Stefano, Favaro, Patricia, Chen, Yifeng, Franck, Helen G., Zhou, Shangzhen, Wright, J. Fraser, Couto, Linda B., Jiang, Haiyan, Pierce, Glenn F., Bellinger, Dwight A., Mingozzi, Federico, Nichols, Timothy C., High, Katherine A.

“…Muscle represents an important tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX) gene in hemophilia B (HB)…”
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AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life by Doshi, Bhavya S., Samelson-Jones, Benjamin J., Nichols, Timothy C., Merricks, Elizabeth P., Siner, Joshua I., French, Robert A., Lee, Ben J., Arruda, Valder R., Callan, Mary Beth

“…The hemophilias are the most common severe inherited bleeding disorders and are caused by deficiency of clotting factor (F) VIII (hemophilia A) or FIX…”
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Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models by Markusic, David M, Nichols, Timothy C, Merricks, Elizabeth P, Palaschak, Brett, Zolotukhin, Irene, Marsic, Damien, Zolotukhin, Sergei, Srivastava, Arun, Herzog, Roland W

“…Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as…”
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Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog by Van Gorder, Lucas, Doshi, Bhavya S., Willis, Elinor, Nichols, Timothy C., Cook, Emma, Everett, John K., Merricks, Elizabeth P., Arruda, Valder R., Bushman, Frederic D., Callan, Mary Beth, Samelson-Jones, Benjamin J.

“…Adeno-associated viral (AAV) vectors have traditionally been viewed as predominantly nonintegrating, with limited concerns for oncogenesis. However,…”
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Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity by Pei, Xiaolei, Shao, Wenwei, Xing, Allene, Askew, Charles, Chen, Xiaojing, Cui, Caibin, Abajas, Yasmina L., Gerber, David A., Merricks, Elizabeth P., Nichols, Timothy C., Li, Wuping, Samulski, R. Jude, Li, Chengwen

“…Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major…”
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Global measurement of coagulation in plasma from normal and haemophilia dogs using a novel modified thrombin generation test - Demonstrated in vitro and ex vivo by Madsen, Daniel Elenius, Nichols, Timothy C, Merricks, Elizabeth P, Waters, Emily K, Wiinberg, Bo

“…Canine models of severe haemophilia resemble their human equivalents both regarding clinical bleeding phenotype and response to treatment. Therefore…”
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Successful treatment of canine hemophilia by continuous expression of canine FVIIa by Margaritis, Paris, Roy, Elise, Aljamali, Majed N., Downey, Harre D., Giger, Urs, Zhou, Shangzhen, Merricks, Elizabeth, Dillow, Aaron, Ezban, Mirella, Nichols, Timothy C., High, Katherine A.

“…Continuous expression of activated factor VII (FVIIa) via gene transfer is a potential therapeutic approach for hemophilia patients with or without inhibitory…”
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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs by Sun, Junjiang, Shao, Wenwei, Chen, Xiaojing, Merricks, Elizabeth P., Wimsey, Lauren, Abajas, Yasmina L., Niemeyer, Glenn P., Lothrop, Clinton D., Monahan, Paul E., Samulski, R. Jude, Nichols, Timothy C., Li, Chengwen

“…Adeno-associated virus (AAV) vectors have been successfully applied in hemophilia clinical trials. However, this approach is limited to patients without…”
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Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy by Wang, Lili, Calcedo, Roberto, Nichols, Timothy C., Bellinger, Dwight A., Dillow, Aaron, Verma, Inder M., Wilson, James M.

“…Adeno-associated virus 8 (AAV8), a new member of the AAV family isolated from nonhuman primates, is an attractive candidate for hepatic gene transfer…”
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