566 results on '"Wilton, A. D."'
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52. Why do you care what other people think? A qualitative investigation of social influence and telecommuting
53. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
54. Corrigendum: Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing
55. Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro
56. Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts
57. Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice
58. Correcting the NLRP3 inflammasome deficiency in macrophages from autoimmune NZB mice with exon skipping antisense oligonucleotides
59. Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing
60. Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping
61. Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations
62. Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials
63. Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model
64. Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency
65. Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing
66. Toward an Understanding of the Spatiality of Social Movements: Labor Organizing at a Private University in Los Angeles
67. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
68. Proposal to ‘restore’ indigenous names misunderstands the complementary nature of botanical nomenclature and indigenous vernacular plant names
69. Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies
70. Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements
71. Understanding, embracing, rejecting: Women's negotiations of disability constructions and categorizations after becoming chronically ill
72. Normal and aberrant splicing of LMNA
73. Primary Nasal Epithelial Cells As a Type-II Alveolar Surrogate Cell Culture Model for ABCA-3 Deficiency
74. Stargardt disease and progress in therapeutic strategies
75. Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene
76. 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
77. Hybridisation within Brassica and allied genera: evaluation of potential for transgene escape
78. Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse
79. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
80. Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript
81. The Influence of Antisense Oligonucleotide Length on Dystrophin Exon Skipping
82. Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies
83. Untranslated Gene Regions and Other Non-coding Elements
84. Poverty and Mental Health: A Qualitative Study of Residential Care Facility Tenants
85. Optimizing Splice-Switching Oligomer Sequences Using 2′-O-Methyl Phosphorothioate Chemistry
86. Primary over-expression of AβPP in muscle does not lead to the development of inclusion body myositis in a new lineage of the MCK-AβPP transgenic mouse
87. O24: PMO-MEDIATED DYSTROPHIN EXON 23 SKIPPING RESTORES MITOCHONDRIAL FUNCTION IN THE MDX HEART
88. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
89. Proposal to 'restore' indigenous names misunderstands the complementary nature of botanical nomenclature and indigenous vernacular plant names.
90. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
91. Personalised Genetic Intervention for Duchenne Muscular Dystrophy: Antisense Oligomers and Exon Skipping
92. Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
93. Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype
94. Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10kb C-to-T splicing mutation
95. Te reo Māori and botanical nomenclature as complementary naming systems for New Zealand’s flora
96. 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
97. Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish
98. Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1
99. A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations
100. Exploring microperimetry and autofluorescence endpoints for monitoring disease progression in PRPF31-associated retinopathy
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