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2. Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model

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. CRISPR-mediated correction of skeletal muscle Ca2+handling in a novel DMD patient-derived pluripotent stem cell model

19. Loss-of-function mutations in SCN4A cause severe foetal hypokinesia or ‘classical’ congenital myopathy

27. 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

29. High-Throughput Digital Image Analysis Reveals Distinct Patterns of Dystrophin Expression in Dystrophinopathy Patients

30. Dystrophin involvement in peripheral circadian SRF signalling

32. Dystrophin regulates peripheral circadian SRF signalling

36. Stem cell function, self-renewal, and behavioral heterogeneity of cells from the adult muscle satellite cell niche

37. Myf5 expression in satellite cells and spindles in adult muscle is controlled by separate genetic elements

42. Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

44. Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

46. MYOBLAST THERAPY

47. CONTRIBUTORS

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