Search Results - "Tanner, Matthew K."

Actinomycin D Specifically Reduces Expanded CUG Repeat RNA in Myotonic Dystrophy Models by Siboni, Ruth B., Nakamori, Masayuki, Wagner, Stacey D., Struck, Adam J., Coonrod, Leslie A., Harriott, Shanee A., Cass, Daniel M., Tanner, Matthew K., Berglund, J. Andrew

“…Myotonic dystrophy type 1 (DM1) is an inherited disease characterized by the inability to relax contracted muscles. Affected individuals carry large CTG…”
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Targeted splice sequencing reveals RNA toxicity and therapeutic response in myotonic dystrophy by Tanner, Matthew K, Tang, Zhenzhi, Thornton, Charles A

“…Abstract Biomarker-driven trials hold promise for therapeutic development in chronic diseases, such as muscular dystrophy. Myotonic dystrophy type 1 (DM1)…”
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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial by Thornton, Charles A, Moxley, Richard Thomas, Eichinger, Katy, Heatwole, Chad, Mignon, Laurence, Arnold, W David, Ashizawa, Tetsuo, Day, John W, Dent, Gersham, Tanner, Matthew K, Duong, Tina, Greene, Ericka P, Herbelin, Laura, Johnson, Nicholas E, King, Wendy, Kissel, John T, Leung, Doris G, Lott, Donovan J, Norris, Daniel A, Pucillo, Evan M, Schell, Wendy, Statland, Jeffrey M, Stinson, Nikia, Subramony, Sub H, Xia, Shuting, Bishop, Kathie M, Bennett, C Frank

“…Myotonic dystrophy type 1 results from an RNA gain-of-function mutation, in which DM1 protein kinase (DMPK) transcripts carrying expanded trinucleotide repeats…”
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Cumulative donor-specific antibody threshold predicts platelet transfusion response in HLA-alloimmunized patients by Boothby, Aaron B., Tanner, Matthew K., Alswied, Abdullah, Youngs, Danny, Bribiesca Rodriguez, Josefine, Bikkani, Thejaswi, Cha, Nuri, Gernsheimer, Terry, Gimferrer, Idoia, Hess, John R., Sokol-Hessner, Lauge, Marivada, Snigdha, Nash, Michael G., Flegel, Willy A., Vassallo, Ralph R., Stroncek, David F., Tsang, Hamilton C., Panch, Sandhya R.

“…•In severe HLA-alloimmune platelet transfusion refractoriness, DSA-MFI sums >10 000 and major-ABO mismatch actionably predict poor 2-hour CCI.•Computer-aided…”
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RNA Splicing Is a Marker of Molecular Pathology and Disease Severity in Myotonic Muscular Dystrophy by Tanner, Matthew K

“…Myotonic muscular dystrophy is a progressive, dominantly inherited disease characterized by skeletal muscle myotonia, weakness, and atrophy in addition to…”
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