216 results on '"Wally, Verena"'
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2. Challenges and progress related to gene editing in rare skin diseases
3. Statistical recommendations for count, binary, and ordinal data in rare disease cross-over trials
4. Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa
5. QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study
6. Therapy Development for Epidermolysis Bullosa
7. Towards a roadmap for COSEB: the next steps in harmonization of outcomes for epidermolysis bullosa
8. Splicing Modulation via Antisense Oligonucleotides in Recessive Dystrophic Epidermolysis Bullosa
9. Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial
10. Threonine 150 Phosphorylation of Keratin 5 Is Linked to Epidermolysis Bullosa Simplex and Regulates Filament Assembly and Cell Viability
11. Towards a roadmap for COSEB: the next steps in harmonization of outcomes for epidermolysis bullosa.
12. Harmonization of outcomes in epidermolysis bullosa: report of the Core Outcome Sets for Epidermolysis Bullosa (COSEB) kick-off meeting
13. MicroRNA-30d-5p—A Potential New Therapeutic Target for Prevention of Ischemic Cardiomyopathy after Myocardial Infarction
14. miR-200b-mediated reversion of a spectrum of epithelial to mesenchymal transition states in recessive dystrophic epidermolysis bullosa squamous cell carcinomas
15. MicroRNA-30d—A Potential New Therapeutic Target for Prevention of Ischemic Cardiomyopathy after Myocardial Infarction
16. Synergistic effects of dendritic cell targeting and laser-microporation on enhancing epicutaneous skin vaccination efficacy
17. Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases
18. Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa
19. A cancer stem cell-like phenotype is associated with miR-10b expression in aggressive squamous cell carcinomas
20. Biomarker Discovery in Rare Malignancies: Development of a miRNA Signature for RDEB-cSCC
21. Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a scoping review as a first step towards outcome harmonization
22. A Novel Fluorescence-Based Screen of Gene Editing Molecules for Junctional Epidermolysis Bullosa
23. COL7A1 Editing via RNA Trans-Splicing in RDEB-Derived Skin Equivalents
24. Chondroitin Sulfate Proteoglycan 4 as a Marker for Aggressive Squamous Cell Carcinoma
25. Harmonization of outcomes in epidermolysis bullosa: report of the Core Outcome Sets for Epidermolysis Bullosa (COSEB) kick-off meeting.
26. Basal pharmacokinetic parameters of topically applied diacerein in pediatric patients with generalized severe epidermolysis bullosa simplex
27. ESDR287 - Development of a non-invasive, non-viral RNA therapy approach for dystrophic epidermolysis bullosa
28. ESDR476 - Differential regulation of miR-200b in squamous cell carcinoma of patients with recessive dystrophic epidermolysis bullosa
29. COL17A1 editing via homology-directed repair in junctional epidermolysis bullosa
30. Evaluating a Targeted Cancer Therapy Approach Mediated by RNA
31. 5′RNA Trans-Splicing Repair of COL7A1 Mutant Transcripts in Epidermolysis Bullosa
32. Topical Diacerein Decreases Skin and Splenic CD11c + Dendritic Cells in Psoriasis.
33. Transcriptome-Guided Drug Repurposing for Aggressive SCCs
34. Evaluating a Targeted Cancer Therapy Approach Mediated by RNA trans-Splicing In Vitro and in a Xenograft Model for Epidermolysis Bullosa-Associated Skin Cancer
35. 6. Wissenschaftstage der Österreichischen Gesellschaft für Dermatologie und Venerologie – ÖGDV Forschungstage
36. 5′ Trans-Splicing Repair of the PLEC1 Gene
37. Cladosporium herbarum translationally controlled tumor protein (TCTP) is an IgE-binding antigen and is associated with disease severity
38. Haploinsufficiency due to deletion within the 3′-UTR of C1-INH-gene associated with hereditary angioedema
39. Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa
40. Epigenetic and metabolic regulation of epidermal homeostasis
41. RNA Trans-Splicing for Genodermatoses
42. High-Throughput Screening for Highly Functional RNA-Trans-Splicing Molecules: Correction of Plectin in Epidermolysis Bullosa Simplex
43. A novel screening system improves genetic correction by internal exon replacement
44. MiRNA-10b marks aggressive squamous cell carcinomas, and confers a cancer stem cell-like phenotype
45. K14 mRNA reprogramming for dominant epidermolysis bullosa simplex
46. Additional file 2 of Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa
47. Additional file 1 of A cancer stem cell-like phenotype is associated with miR-10b expression in aggressive squamous cell carcinomas
48. Additional file 3 of Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa
49. Additional file 1 of Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa
50. Extracellular Vesicles as Biomarkers for the Detection of a Tumor Marker Gene in Epidermolysis Bullosa-Associated Squamous Cell Carcinoma
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