31 results on '"Furling D"'
Search Results
2. FP.44 Exploring the role of MuscleBlind-Like proteins in the regulation of CaVB1 isoform expression in adult skeletal muscle
3. MYASTHENIA & RELATED DISORDERS
4. O.32Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in muscle of DM1 mice
5. Proteomic evaluation of Pip6a-PMO treatment for myotonic dystrophy type 1
6. MYASTHENIA & RELATED DISORDERS: P.31 Identification of new recessive mutations in synaptotagmin-2 responsible for severe and early presynaptic forms of congenital myasthenic syndrome
7. Survival in myotonic dystrophy type 1 predicted by the new DM1 survival risk score
8. CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1
9. Association between mutation size and cardiac involvement in myotonic dystrophy type 1: an analysis of the DM1-heart registry
10. Development of a MBNLΔ decoy-based gene therapy for myotonic dystrophy
11. Correlation between mutation size and cardiac involvement in myotonic dystrophy type 1: An analysis of the DM1-heart registry
12. D10 - Proteomic evaluation of Pip6a-PMO treatment for myotonic dystrophy type 1
13. P.279 - Development of a MBNLΔ decoy-based gene therapy for myotonic dystrophy
14. P.278 - CRISPR/Cas9-mediated genome editing corrects splicing alterations in myotonic dystrophy type 1
15. P.270 - Survival in myotonic dystrophy type 1 predicted by the new DM1 survival risk score
16. P.269 - Association between mutation size and cardiac involvement in myotonic dystrophy type 1: an analysis of the DM1-heart registry
17. P22 High content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC independent pathway in myotonic dystrophy cell lines
18. P.362 - Correlation between mutation size and cardiac involvement in myotonic dystrophy type 1: An analysis of the DM1-heart registry
19. P74 Observations on oligo-based therapy for Myotonic Dystrophy
20. P81 Compound screening in myotonic dystrophy
21. O.6 Antisense approach for myotonic dystrophy
22. G.P.12.01 Immunodetection of myotubularin in human tissues: A diagnostic tool for X-linked myotubular myopathy
23. D.P.4.08 P16 triggers premature senescence of congenital DM1 myoblasts
24. D.P.4.09 Defective mRNA in myotonic dystrophy accumulates at the periphery of nuclear splicing speckles
25. D.P.4.10 Muscleblind-like proteins: Similarities and differences in normal and myotonic dystrophy muscle
26. G.P.3.01 The use of immortalised human fibroblasts from a DMD patient to test exon skipping in vivo
27. G.P.14.09 Functional characterization of skeletal muscles in DM1 mice
28. T.O.4 Ribozyme-based gene therapy reverses muscle atrophy in a mouse model of myotonic dystrophy
29. Decreased levels of myotonic dystrophy protein kinase (DMPK) and delayed differentiation in human myotonic dystrophy myoblasts
30. G.P.14.10 Inhibition of prostaglandin E2 (PGE2) production restores the differentiation of congenital human dystrophy myotonic type 1 (CDM1) myoblasts
31. 440P Nonclinical data for PGN-EDODM1 demonstrated nuclear delivery, mechanistic and meaningful activity for the potential treatment of DM1.
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