196 results on '"Wilton, A. D."'
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2. Nonsequential Pre-mRNA Splicing: From Basic Understanding to Impacts on Splice-Manipulating Therapies
3. Limb Girdle Muscular Dystrophy Type 2B (LGMD2B): Diagnosis and Therapeutic Possibilities
4. The recurrent deep intronic pseudoexon-inducing variant COL6A1 c.930+189C>T results in a consistently severe phenotype of COL6-related dystrophy: Towards clinical trial readiness for splice-modulating therapy
5. Microalgae as a source of bioavailable heme
6. Induced alternative splicing an opportunity to study PCSK9 protein isoforms at physiologically relevant concentrations
7. Gender dimorphic species flower earlier than cosexuals
8. Supplementary Figures 1 - 5 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
9. Supplementary Methods and Figure Legend from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
10. Data from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
11. Supplementary Figures 9 - 10 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
12. Data from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
13. Supplementary Figure 11 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
14. Supplementary Figure 12 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
15. Supplementary Figures 6 - 8 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
16. Supplementary Tables 1 - 8 from MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells
17. Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment
18. Making Clean and Sober Places: The Intersections of Therapeutic Landscapes and Substance Abuse Treatment
19. Characterising splicing defects of ABCA4 variants within exons 13–50 in patient-derived fibroblasts
20. Aptamers as Therapeutic Tools in Neurological Diseases
21. Targeted Molecular Therapeutics for Parkinson's Disease: A Role for Antisense Oligonucleotides?
22. Pathogenesis and Treatment of Usher Syndrome Type IIA
23. Corrigendum: Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing
24. Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro
25. Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts
26. Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model
27. Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency
28. Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing
29. Characterising Splicing Defects of ABCA4 Variants within Exons 13-50 in Patient-Derived Fibroblasts
30. Proposal to ‘restore’ indigenous names misunderstands the complementary nature of botanical nomenclature and indigenous vernacular plant names
31. Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies
32. Splice correction therapies for familial hypercholesterolemic patients with low-density lipoprotein receptor mutations
33. Primary Nasal Epithelial Cells As a Type-II Alveolar Surrogate Cell Culture Model for ABCA-3 Deficiency
34. Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation
35. Stargardt disease and progress in therapeutic strategies
36. Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides
37. Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene
38. Generation of an induced pluripotent stem cell line from a patient with Stargardt disease caused by biallelic c.[5461–10T>C;5603A>T];[6077T>C] mutations in the ABCA4 gene
39. Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies
40. Untranslated Gene Regions and Other Non-coding Elements
41. Untranslated Gene Regions and Other Non-coding Elements
42. Optimizing Splice-Switching Oligomer Sequences Using 2′-O-Methyl Phosphorothioate Chemistry
43. Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
44. Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype
45. Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10kb C-to-T splicing mutation
46. Te reo Māori and botanical nomenclature as complementary naming systems for New Zealand’s flora
47. Generation of three induced pluripotent stem cell lines from a patient with Usher syndrome caused by biallelic c.949C > A and c.1256G > T mutations in the USH2A gene
48. Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein
49. Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1
50. A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations
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