Search Results - "Janson, Anneke"

Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy by Beekman, Chantal, Janson, Anneke A, Baghat, Aabed, van Deutekom, Judith C, Datson, Nicole A

“…Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by progressive weakness of the skeletal and cardiac muscles. This X-linked disorder…”
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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy by Goemans, Nathalie M, Tulinius, Mar, van den Akker, Johanna T, Burm, Brigitte E, Ekhart, Peter F, Heuvelmans, Niki, Holling, Tjadine, Janson, Anneke A, Platenburg, Gerard J, Sipkens, Jessica A, Sitsen, J.M. Ad, Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B, Buyse, Gunnar, Darin, Niklas, Verschuuren, Jan J, Campion, Giles V, de Kimpe, Sjef J, van Deutekom, Judith C

“…Local intramuscular administration of PRO051 in patients with Duchenne's muscular dystrophy was previously reported to induce exon-51 skipping during…”
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Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms by Aartsma-Rus, Annemieke, van Vliet, Laura, Hirschi, Marscha, Janson, Anneke AM, Heemskerk, Hans, de Winter, Christa L, de Kimpe, Sjef, van Deutekom, Judith CT, 't Hoen, Peter AC, van Ommen, Gert-Jan B

“…Antisense oligonucleotides (AONs) can interfere with mRNA processing through RNase H–mediated degradation, translational arrest, or modulation of splicing. The…”
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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051 by van Deutekom, Judith C, Janson, Anneke A, Ginjaar, Ieke B, Frankhuizen, Wendy S, Aartsma-Rus, Annemieke, Bremmer-Bout, Mattie, den Dunnen, Johan T, Koop, Klaas, van der Kooi, Anneke J, Goemans, Nathalie M, de Kimpe, Sjef J, Ekhart, Peter F, Venneker, Edna H, Platenburg, Gerard J, Verschuuren, Jan J, van Ommen, Gert-Jan B

“…This study explored the safety, tolerability, and dystrophin-restoring effect of a single, intramuscular dose of an antisense oligonucleotide, PRO051, to…”
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Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients by Aartsma-Rus, Annemieke, Janson, Anneke A.M., Kaman, Wendy E., Bremmer-Bout, Mattie, den Dunnen, Johan T., Baas, Frank, van Ommen, Gert-Jan B., van Deutekom, Judith C.T.

“…The dystrophin deficiency leading to the severely progressing muscle degeneration in Duchenne muscular dystrophy (DMD) patients is caused by frame-shifting…”
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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy by Aartsma-Rus, Annemieke, Janson, Anneke A M, van Ommen, Gert-Jan B, van Deutekom, Judith C T

“…Antisense-mediated exon skipping is currently one of the most promising therapeutic approaches for Duchenne muscular dystrophy (DMD). Using antisense…”
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DMD transcript imbalance determines dystrophin levels by Spitali, Pietro, Bergen, Janneke C., Verhaart, Ingrid E. C., Wokke, Beatrijs, Janson, Anneke A. M., Eijnde, Rani, Dunnen, Johan T., Laros, Jeroen F. J., Verschuuren, Jan J. G. M., Hoen, Peter A. C. 't, Aartsma‐Rus, Annemieke

“…Duchenne and Becker muscular dystrophies are caused by out‐of‐frame and in‐frame mutations, respectively, in the dystrophin encoding DMD gene. Molecular…”
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Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice by Jirka, Silvana M G, Heemskerk, Hans, Tanganyika-de Winter, Christa L, Muilwijk, Daan, Pang, Kar Him, de Visser, Peter C, Janson, Anneke, Karnaoukh, Tatyana G, Vermue, Rick, 't Hoen, Peter A C, van Deutekom, Judith C T, Aguilera, Begoña, Aartsma-Rus, Annemieke

“…Antisense oligonucleotide (AON)-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy that is currently being tested in…”
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Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense by Aartsma-Rus, Annemieke, Janson, Anneke A.M., Kaman, Wendy E., Bremmer-Bout, Mattie, van Ommen, Gert-Jan B., den Dunnen, Johan T., van Deutekom, Judith C.T.

“…Dystrophin deficiency, which leads to severe and progressive muscle degeneration in patients with Duchenne muscular dystrophy (DMD), is caused by frameshifting…”
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Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides by Bremmer-Bout, Mattie, Aartsma-Rus, Annemieke, de Meijer, Emile J, Kaman, Wendy E, Janson, Anneke A M, Vossen, Rolf H A M, van Ommen, Gert-Jan B, den Dunnen, Johan T, van Deutekom, Judith C T

“…The therapeutic potential of frame-restoring exon skipping by antisense oligonucleotides (AONs) has recently been demonstrated in cultured muscle cells from a…”
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Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells by VAN DEUTEKOM, Judith C. T, BREMMER-BOUT, Mattie, JANSON, Anneke A. M, GINJAAR, Leke B, BAAS, Frank, DEN DUNNEN, Johan T, VAN OMMEN, Gert-Jan B

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Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy by Aartsma-Rus, Annemieke, Bremmer-Bout, Mattie, Janson, Anneke A.M, den Dunnen, Johan T, van Ommen, Gert-Jan B, van Deutekom, Judith C.T

“…Duchenne muscular dystrophy is primarily caused by frame-disrupting mutations in the Duchenne muscular dystrophy gene which abort dystrophin synthesis. We have…”
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Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells by van Deutekom, J C, Bremmer-Bout, M, Janson, A A, Ginjaar, I B, Baas, F, den Dunnen, J T, van Ommen, G J

“…Due to frame-shifting mutations in the DMD gene that cause dystrophin deficiency, Duchenne muscular dystrophy (DMD) patients suffer from lethal muscle…”
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Use of capillary Western immunoassay by Beekman, Chantal, Janson, Anneke A, Baghat, Aabed, van Deutekom, Judith C, Datson, Nicole A

“…Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by progressive weakness of the skeletal and cardiac muscles. This X-linked disorder…”
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Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites by Aartsma-Rus, Annemieke, De Winter, Christa L, Janson, Anneke A M, Kaman, Wendy E, Van Ommen, Gert-Jan B, Den Dunnen, Johan T, Van Deutekom, Judith C T

“…As small molecule drugs for Duchenne muscular dystrophy (DMD), antisense oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD…”
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HLA-Class II-Associated Control of Antigen Recognition by T Cells in Leprosy: A Prominent Role for the 30/31-kDa Antigens by Thole, Jelle E. R, Janson, Anneke A. M, Cornelisse, Yolanthe, Schreuder, Geziena M. T, Wieles, Brigitte, Naafs, Ben, de Vries, Rene R. P, Ottenhoff, Tom H. M

“…The recognition of 16 mycobacterial Ags by a panel of T cell lines from leprosy patients and healthy exposed individuals from an endemic population was…”
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A Mycobacterium leprae--Specific Human T Cell Epitope Cross-Reactive with an HLA-DR2 Peptide by Anderson, D. C., Wim C. A. Van Schooten, Barry, Michael E., Anneke A. M. Janson, Buchanan, Tom M., René R. P. De Vries

“…Mycobacterium leprae induces T cell reactivity and protective immunity in the majority of exposed individuals, but the minority that develop leprosy exhibit…”
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Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice by Datson, Nicole A, Bijl, Suzanne, Janson, Anneke, Testerink, Janwillem, van den Eijnde, Rani, Weij, Rudie, Puoliväli, Jukka, Lehtimäki, Kimmo, Bragge, Timo, Ahtoniemi, Toni, van Deutekom, Judith C

“…Duchenne muscular dystrophy (DMD) is a severe childhood muscle disease primarily caused by the lack of functional dystrophin at the muscle fiber membranes…”
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Mycobacteria contain two groEL genes: the second Mycobacterium leprae groEL gene is arranged in an operon with groES by Rinke de Wit, T F, Bekelie, S, Osland, A, Miko, T L, Hermans, P W, van Soolingen, D, Drijfhout, J W, Schöningh, R, Janson, A A, Thole, J E

“…In contrast to other bacterial species, mycobacteria were thus far considered to contain groEL and groES genes that are present on separate loci on their…”
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A Mycobacterium leprae-specific gene encoding an immunologically recognized 45 kDa protein by Rinke de Wit, T F, Clark-Curtiss, J E, Abebe, F, Kolk, A H, Janson, A A, van Agterveld, M, Thole, J E

“…By screening a Mycobacterium leprae lambda gt11 expression library with a serum from an Ethiopian lepromatous leprosy (LL) patient a clone was isolated (LL4)…”
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