Search Results - "Wally, Verena"

Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa by Wally, Verena, Reisenberger, Manuela, Kitzmüller, Sophie, Laimer, Martin

“…Abstract Background Hereditary epidermolysis bullosa (EB) comprises a heterogeneous group of rare genodermatoses, which are caused by mutations in genes…”
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QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study by Bornert, Olivier, Hogervorst, Marieke, Nauroy, Pauline, Bischof, Johannes, Swildens, Jim, Athanasiou, Ioannis, Tufa, Sara F., Keene, Douglas R., Kiritsi, Dimitra, Hainzl, Stefan, Murauer, Eva M., Marinkovich, M. Peter, Platenburg, Gerard, Hausser, Ingrid, Wally, Verena, Ritsema, Tita, Koller, Ulrich, Haisma, Elisabeth M., Nyström, Alexander

“…Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as…”
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Statistical recommendations for count, binary, and ordinal data in rare disease cross-over trials by Geroldinger, Martin, Verbeeck, Johan, Hooker, Andrew C, Thiel, Konstantin E, Molenberghs, Geert, Nyberg, Joakim, Bauer, Johann, Laimer, Martin, Wally, Verena, Bathke, Arne C, Zimmermann, Georg

“…Recommendations for statistical methods in rare disease trials are scarce, especially for cross-over designs. As a result various state-of-the-art…”
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Splicing Modulation via Antisense Oligonucleotides in Recessive Dystrophic Epidermolysis Bullosa by Hainzl, Stefan, Trattner, Lisa, Liemberger, Bernadette, Bischof, Johannes, Kocher, Thomas, Ablinger, Michael, Nyström, Alexander, Obermayer, Astrid, Klausegger, Alfred, Guttmann-Gruber, Christina, Wally, Verena, Bauer, Johann W, Hofbauer, Josefina Piñón, Koller, Ulrich

“…Antisense oligonucleotides (ASOs) represent an emerging therapeutic platform for targeting genetic diseases by influencing various aspects of (pre-)mRNA…”
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COL7A1 Editing via RNA Trans -Splicing in RDEB-Derived Skin Equivalents by Liemberger, Bernadette, Bischof, Johannes, Ablinger, Michael, Hainzl, Stefan, Murauer, Eva M, Lackner, Nina, Ebner, Patricia, Kocher, Thomas, Nyström, Alexander, Wally, Verena, Mayr, Elisabeth, Guttmann-Gruber, Christina, Hofbauer, Josefina Piñón, Bauer, Johann W, Koller, Ulrich

“…Mutations in the gene lead to malfunction, reduction or complete absence of type VII collagen (C7) in the skin's basement membrane zone (BMZ), impairing skin…”
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5′RNA Trans-Splicing Repair of COL7A1 Mutant Transcripts in Epidermolysis Bullosa by Mayr, Elisabeth, Ablinger, Michael, Lettner, Thomas, Murauer, Eva M., Guttmann-Gruber, Christina, Piñón Hofbauer, Josefina, Hainzl, Stefan, Kaiser, Manfred, Klausegger, Alfred, Bauer, Johann W., Koller, Ulrich, Wally, Verena

“…Mutations within the COL7A1 gene underlie the inherited recessive subtype of the blistering skin disease dystrophic epidermolysis bullosa (RDEB). Although gene…”
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Threonine 150 Phosphorylation of Keratin 5 Is Linked to Epidermolysis Bullosa Simplex and Regulates Filament Assembly and Cell Viability by Sawant, Mugdha, Schwarz, Nicole, Windoffer, Reinhard, Magin, Thomas M., Krieger, Jan, Mücke, Norbert, Obara, Boguslaw, Jankowski, Vera, Jankowski, Joachim, Wally, Verena, Lettner, Thomas, Leube, Rudolf E.

“…A characteristic feature of the skin blistering disease epidermolysis bullosa simplex is keratin filament (KF) network collapse caused by aggregation of the…”
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A Novel Fluorescence-Based Screen of Gene Editing Molecules for Junctional Epidermolysis Bullosa by Zwicklhuber, Janine, Kocher, Thomas, Liemberger, Bernadette, Hainzl, Stefan, Bischof, Johannes, Strunk, Dirk, Raninger, Anna M, Gratz, Iris, Wally, Verena, Guttmann-Gruber, Christina, Hofbauer, Josefina Piñón, Bauer, Johann W, Koller, Ulrich

“…Junctional epidermolysis bullosa (JEB) is a severe blistering skin disease caused by mutations in genes encoding structural proteins essential for skin…”
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Transcriptome-Guided Drug Repurposing for Aggressive SCCs by Zauner, Roland, Wimmer, Monika, Dorfer, Sonja, Ablinger, Michael, Koller, Ulrich, Piñón Hofbauer, Josefina, Guttmann-Gruber, Christina, Bauer, Johann W, Wally, Verena

“…Despite a significant rise in the incidence of cutaneous squamous cell carcinoma (SCC) in recent years, most SCCs are well treatable. However, against the…”
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Topical Diacerein Decreases Skin and Splenic CD11c + Dendritic Cells in Psoriasis by Brunner, Susanne M, Ramspacher, Andrea, Rieser, Caroline, Leitner, Julia, Heil, Hannah, Ablinger, Michael, Tevini, Julia, Wimmer, Monika, Koller, Andreas, Piñón Hofbauer, Josefina, Felder, Thomas K, Bauer, Johann W, Kofler, Barbara, Lang, Roland, Wally, Verena

“…Psoriasis is an inflammatory skin disease characterized by increased neo-vascularization, keratinocyte hyperproliferation, a pro-inflammatory cytokine milieu…”
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Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa by Ablinger, Michael, Lettner, Thomas, Friedl, Nicole, Potocki, Hannah, Palmetzhofer, Theresa, Koller, Ulrich, Illmer, Julia, Liemberger, Bernadette, Hainzl, Stefan, Klausegger, Alfred, Reisenberger, Manuela, Lambert, Jo, Van Gele, Mireille, Desmet, Eline, Van Maelsaeke, Els, Wimmer, Monika, Zauner, Roland, Bauer, Johann W, Wally, Verena

“…Intermediate junctional epidermolysis bullosa caused by mutations in the gene is characterized by the frequent development of blisters and erosions on the skin…”
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Synergistic effects of dendritic cell targeting and laser-microporation on enhancing epicutaneous skin vaccination efficacy by Machado, Yoan, Duinkerken, Sanne, Hoepflinger, Veronika, Mayr, Melissa, Korotchenko, Evgeniia, Kurtaj, Almedina, Pablos, Isabel, Steiner, Markus, Stoecklinger, Angelika, Lübbers, Joyce, Schmid, Maximillian, Ritter, Uwe, Scheiblhofer, Sandra, Ablinger, Michael, Wally, Verena, Hochmann, Sarah, Raninger, Anna M., Strunk, Dirk, van Kooyk, Yvette, Thalhamer, Josef, Weiss, Richard

“…Due to its unique immunological properties, the skin is an attractive target tissue for allergen-specific immunotherapy. In our current work, we combined a…”
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Evaluating a Targeted Cancer Therapy Approach Mediated by RNA trans -Splicing In Vitro and in a Xenograft Model for Epidermolysis Bullosa-Associated Skin Cancer by Woess, Katharina, Sun, Yuchen, Morio, Hanae, Stierschneider, Anna, Kaufmann, Anna, Hainzl, Stefan, Trattner, Lisa, Kocher, Thomas, Tockner, Birgit, Leb-Reichl, Victoria, Steiner, Markus, Brachtl, Gabriele, South, Andrew P, Bauer, Johann W, Reichelt, Julia, Furihata, Tomomi, Wally, Verena, Koller, Ulrich, Piñón Hofbauer, Josefina, Guttmann-Gruber, Christina

“…Conventional anti-cancer therapies based on chemo- and/or radiotherapy represent highly effective means to kill cancer cells but lack tumor specificity and,…”
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A cancer stem cell-like phenotype is associated with miR-10b expression in aggressive squamous cell carcinomas by Wimmer, Monika, Zauner, Roland, Ablinger, Michael, Piñón-Hofbauer, Josefina, Guttmann-Gruber, Christina, Reisenberger, Manuela, Lettner, Thomas, Niklas, Norbert, Proell, Johannes, Sajinovic, Mila, De Souza, Paul, Hainzl, Stefan, Kocher, Thomas, Murauer, Eva M, Bauer, Johann W, Strunk, Dirk, Reichelt, Julia, Mellick, Albert Sleiman, Wally, Verena

“…Cutaneous squamous cell carcinomas (cSCC) are the primary cause of premature deaths in patients suffering from the rare skin-fragility disorder recessive…”
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RNA Trans-Splicing Modulation via Antisense Molecule Interference by Liemberger, Bernadette, Piñón Hofbauer, Josefina, Wally, Verena, Arzt, Claudia, Hainzl, Stefan, Kocher, Thomas, Murauer, Eva M, Bauer, Johann W, Reichelt, Julia, Koller, Ulrich

“…In recent years, RNA -splicing has emerged as a suitable RNA editing tool for the specific replacement of mutated gene regions at the pre-mRNA level. Although…”
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Extracellular Vesicles as Biomarkers for the Detection of a Tumor Marker Gene in Epidermolysis Bullosa-Associated Squamous Cell Carcinoma by Sun, Yuchen, Woess, Katharina, Kienzl, Melanie, Leb-Reichl, Victoria M., Feinle, Andrea, Wimmer, Monika, Zauner, Roland, Wally, Verena, Luetz-Meindl, Ursula, Mellerio, Jemima E., Fuentes, Ignacia, South, Andrew P., Bauer, Johann W., Reichelt, Julia, Furihata, Tomomi, Guttmann-Gruber, Christina, Piñón Hofbauer, Josefina

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Basal pharmacokinetic parameters of topically applied diacerein in pediatric patients with generalized severe epidermolysis bullosa simplex by Ablinger, Michael, Felder, Thomas K, Wimmer, Monika, Zauner, Roland, Hofbauer, Peter, Lettner, Thomas, Wolkersdorfer, Martin, Lagler, Florian B, Diem, Anja, Bauer, Johann W, Wally, Verena

“…Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in…”
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COL17A1 editing via homology-directed repair in junctional epidermolysis bullosa by Petković, Igor, Bischof, Johannes, Kocher, Thomas, March, Oliver Patrick, Liemberger, Bernadette, Hainzl, Stefan, Strunk, Dirk, Raninger, Anna Maria, Binder, Heide-Marie, Reichelt, Julia, Guttmann-Gruber, Christina, Wally, Verena, Piñón Hofbauer, Josefina, Bauer, Johann Wolfgang, Koller, Ulrich

“…Background Epidermolysis bullosa (EB), a severe genetic disorder characterized by blister formation in skin, is caused by mutations in genes encoding…”
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MMP-9 and CXCL8/IL-8 are potential therapeutic targets in epidermolysis bullosa simplex by Lettner, Thomas, Lang, Roland, Klausegger, Alfred, Hainzl, Stefan, Bauer, Johann W, Wally, Verena

“…Epidermolysis bullosa refers to a group of genodermatoses that affects the integrity of epithelial layers, phenotypically resulting in severe skin blistering…”
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MicroRNA-30d-5p—A Potential New Therapeutic Target for Prevention of Ischemic Cardiomyopathy after Myocardial Infarction by Boxhammer, Elke, Paar, Vera, Wernly, Bernhard, Kiss, Attila, Mirna, Moritz, Aigner, Achim, Acar, Eylem, Watzinger, Simon, Podesser, Bruno K, Zauner, Roland, Wally, Verena, Ablinger, Michael, Hackl, Matthias, Hoppe, Uta C, Lichtenauer, Michael

“…(1) Background and Objective: MicroRNAs (miRs) are biomarkers for assessing the extent of cardiac remodeling after myocardial infarction (MI) and important…”
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