Search Results - "Freier, Sue"

Intracerebral Infusion of Antisense Oligonucleotides Into Prion-infected Mice by Nazor Friberg, Karah, Hung, Gene, Wancewicz, Ed, Giles, Kurt, Black, Chris, Freier, Sue, Bennett, Frank, DeArmond, Stephen J, Freyman, Yevgeniy, Lessard, Pierre, Ghaemmaghami, Sina, Prusiner, Stanley B

“…Mice deficient for the cellular prion protein (PrPC) do not develop prion disease; accordingly, gene-based strategies to diminish PrPC expression are of…”
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Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice by Guo, Shuling, Casu, Carla, Gardenghi, Sara, Booten, Sheri, Aghajan, Mariam, Peralta, Raechel, Watt, Andy, Freier, Sue, Monia, Brett P, Rivella, Stefano

“…β-Thalassemia and HFE-related hemochromatosis are 2 of the most frequently inherited disorders worldwide. Both disorders are characterized by low levels of…”
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Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration by Murray, Susan F, Jazayeri, Ali, Matthes, Michael T, Yasumura, Douglas, Yang, Haidong, Peralta, Raechel, Watt, Andy, Freier, Sue, Hung, Gene, Adamson, Peter S, Guo, Shuling, Monia, Brett P, LaVail, Matthew M, McCaleb, Michael L

“…To preserve photoreceptor cell structure and function in a rodent model of retinitis pigmentosa with P23H rhodopsin by selective inhibition of the mutant…”
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Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides by Ackermann, Elizabeth J., Guo, Shuling, Benson, Merrill D., Booten, Sheri, Freier, Sue, Hughes, Steven G., Kim, Tae-Won, Jesse Kwoh, T., Matson, John, Norris, Dan, Yu, Rosie, Watt, Andy, Monia, Brett P.

“…Transthyretin amyloidosis (ATTR amyloidosis) is a rare disease that results from the deposition of misfolded transthyretin (TTR) protein from the plasma into…”
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Trends in Financial Toxicity for Patients of a Community Cancer Clinic by Santhosh-Kumar, Cherupolil R., Struve, Stephanie, Freier, Sue

“…Background: Rising health care costs - especially those of new medications for cancer treatment - have caused a severe financial strain on patients, leading to…”
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Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs by Lagier-Tourenne, Clotilde, Polymenidou, Magdalini, Hutt, Kasey R, Vu, Anthony Q, Baughn, Michael, Huelga, Stephanie C, Clutario, Kevin M, Ling, Shuo-Chien, Liang, Tiffany Y, Mazur, Curt, Wancewicz, Edward, Kim, Aneeza S, Watt, Andy, Freier, Sue, Hicks, Geoffrey G, Donohue, John Paul, Shiue, Lily, Bennett, C Frank, Ravits, John, Cleveland, Don W, Yeo, Gene W

“…In this study, the authors compare the RNA binding patterns of FUS/TLS and TDP-43. Although these proteins regulate the processing of mostly distinct gene…”
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Targeting KLF1 for the Treatment of Sickle Cell Disease Using Antisense Oligonucleotides by Peralta, Raechel, Low, Audrey, Booten, Sheri, Zhou, Dewang, Kim, Aneeza, Freier, Sue, Guo, Shuling, Murray, Sue, Townes, Tim M., Hung, Gene

“…Sickle cell anemia (SCD) is a hereditary blood disorder in which red blood cells (RBC) become sickle-shaped and block blood vessels, leading to painful…”
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Targeting BCL11A and KLF1 For The Treatment Of Sickle Cell Disease and β-Thalassemia In Vitro using Antisense Oligonucleotides by Peralta, Raechel, Low, Audrey, Kim, Aneeza, Murray, Sue, Guo, Shuling, Freier, Sue, Townes, Tim M., Hung, Gene

“…Sickle cell anemia (SCD) is a hereditary blood disorder in which red blood cells (RBC) become sickle-shaped and block blood vessels, leading to painful…”
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Pharmacokinetic properties of 2'-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats by Geary, R S, Watanabe, T A, Truong, L, Freier, S, Lesnik, E A, Sioufi, N B, Sasmor, H, Manoharan, M, Levin, A A

“…Plasma pharmacokinetics, biodistribution, excretion, and metabolism of four modified 20-mer antisense oligonucleotides targeted to human intercellular adhesion…”
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Reducing TMPRSS6 ameliorates hemochromatosis and [Beta]-thalassemia in mice by Guo, Shuling, Casu, Carla, Gardenghi, Sara, Booten, Sheri, Aghajan, Mariam, Peralta, Raechel, Watt, Andy, Freier, Sue, Monia, Brett P, RiveIla, Stefano

“…β-Thalassemia and HFE-related hemochromatosis are 2 of the most frequently inherited disorders worldwide. Both disorders are characterized by low levels of…”
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A Sense Oligonucleotide Specifically Reverses the Prothrombin Depletion and Anticoagulation Caused by ISIS 401025, an Antisense Oligonucleotide Targeting Prothrombin RNA in Mice by Zhao, Chenguang, Monia, Brett P., Siwkowski, Andy, Freier, Sue, Witchell, Donna, Zhang, Hong

“…The ability to safely and effectively neutralize anticoagulant activity is of particular importance in the case of long acting drugs. While antisense…”
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Antisense Strategies to Specifically Deplete Levels of Factors II, VII and IX in Mice To Assess Their Potential as Targets for Anticoagulation by Zhang, Hong, Zhao, Chenguang, Crosby, Jeff, Siwkowski, Andy, Freier, Sue, Witchell, Donna, Monia, Brett P.

“…Numerous potential intervention points exist within coagulation pathways for development of novel anticoagulant agents. To help determine the most suitable…”
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Artificial neural network prediction of antisense oligodeoxynucleotide activity by Giddings, Michael C., Shah, Atul A., Freier, Sue, Atkins, John F., Gesteland, Raymond F., Matveeva, Olga V.

“…An mRNA transcript contains many potential antisense oligodeoxynucleotide target sites. Identifi cation of the most efficacious targets remains an important…”
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