63 results on '"Aartsma-Rus, Annemieke"'
Search Results
2. Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
3. Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
4. The therapeutic potential of soluble activin type receptor IIB treatment in a limb girdle muscular dystrophy type 2D mouse model
5. The mRNA Binding Proteome of Proliferating and Differentiated Muscle Cells
6. Implications of increased S100β and Tau5 proteins in dystrophic nerves of two mdx mouse models for Duchenne muscular dystrophy
7. Longitudinal follow-up of verbal span and processing speed in Duchenne muscular dystrophy
8. 226th ENMC International Workshop
9. Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and caregivers’ perspective
10. 238th ENMC International Workshop: Updating management recommendations of cardiac dystrophinopathyHoofddorp, The Netherlands, 30 November - 2 December 2018
11. Evidence-Based Consensus and Systematic Review on Reducing the Time to Diagnosis of Duchenne Muscular Dystrophy
12. Voluntary exercise improves muscle function and does not exacerbate muscle and heart pathology in aged Duchenne muscular dystrophy mice
13. Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017
14. Meeting report of the “Regulatory Exchange Matters” session at the 5th International TREAT-NMD Conference
15. 227 th ENMC International Workshop
16. Genetic therapies for spinal muscular atrophy type 1
17. Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
18. Stakeholder collaboration for spinal muscular atrophy therapy development
19. Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy
20. Environmental 24-hr Cycles Are Essential for Health
21. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
22. 504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy
23. New Momentum for the Field of Oligonucleotide Therapeutics
24. Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
25. Response to: Studying the role of dystrophin-associated proteins in influencing Becker muscular dystrophy disease severity
26. Response to: Evaluation of the serum matrix metalloproteinase-9 as a biomarker for monitoring disease progression in Duchenne muscular dystrophy
27. Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy
28. Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
29. Response
30. The risks of therapeutic misconception and individual patient (n=1) “trials” in rare diseases such as Duchenne dystrophy
31. Biomarkers and surrogate endpoints in Duchenne: Meeting report
32. Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases
33. Low dystrophin levels in heart can delay heart failure in mdx mice
34. A Novel Feed-Forward Loop between ARIH2 E3-Ligase and PABPN1 Regulates Aging-Associated Muscle Degeneration
35. Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy
36. The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice
37. Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor
38. Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy
39. Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides
40. 194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8–10 December 2012, Naarden, The Netherlands
41. Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon
42. Advances in therapeutic RNA-targeting
43. Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
44. Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)
45. Assessment of cardiac function in three mouse dystrophinopathies by magnetic resonance imaging
46. Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains
47. Splice Modulating Therapies for Human Disease
48. Phage display screening without repetitious selection rounds
49. Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models
50. Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.