45 results on '"Wilton, A. D."'
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2. Pathogenesis and Treatment of Usher Syndrome Type IIA
3. Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation
4. Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein
5. Antisense Oligonucleotides Targeting Angiogenic Factors as Potential Cancer Therapeutics
6. Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping
7. Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript
8. Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro
9. Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes
10. Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
11. Complement-mediated muscle cell lysis: A possible mechanism of myonecrosis in anti-SRP associated necrotizing myopathy (ASANM)
12. A Cell-Based High-Throughput Screening Assay for Posttranscriptional Utrophin Upregulation
13. Multiple exon skipping strategies to by-pass dystrophin mutations
14. Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
15. Gene therapy: therapeutic applications and relevance to pathology
16. Why do you care what other people think? A qualitative investigation of social influence and telecommuting
17. 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
18. Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing
19. Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping
20. Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations
21. Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials
22. 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
23. Understanding, embracing, rejecting: Women's negotiations of disability constructions and categorizations after becoming chronically ill
24. Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse
25. Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript
26. The Influence of Antisense Oligonucleotide Length on Dystrophin Exon Skipping
27. Multiple QTLs influencing triglyceride and HDL and total cholesterol levels identified in families with atherogenic dyslipidemia
28. Terminal antisense oligonucleotide modifications can enhance induced exon skipping
29. Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene
30. 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
31. 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
32. Molecular analysis of a spontaneous dystrophin `knockout' dog
33. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides
34. Dystrophin as a therapeutic biomarker: Are we ignoring data from the past?
35. Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
36. Analysis of HLA-DRB3 alleles and supertypical genotypes in the MHC Class II region in sporadic inclusion body myositis
37. Emerging scholarship in the geographies of disability
38. Morpholino Oligomer Peptide Therapy Improves Mitochondrial Function in mdx Cardiomyopathy
39. Splice-switching as a treament for duchenne muscular dystrophy
40. Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?
41. Diminished worlds? The geography of everyday life with HIV/AIDS
42. Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?
43. Three novel mutations and two variants in the gene for Cu/Zn superoxide dismutase in familial amyotrophic lateral sclerosis
44. The constitution of difference: Space and psyche in landscapes of exclusion
45. Power and community: Organizational and cultural responses to AIDS: D. Altman Taylor and Francis London (1994) viii + 179 pp, index and bibliography ISBN 074841938 hardback, 074841946 paperback
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