| scholarly article | Q13442814 |
| P50 | author | Stanley F Nelson | Q90679187 |
| P2093 | author name string | Robert Damoiseaux | |
| Melissa J Spencer | |||
| Richard T Wang | |||
| M Carrie Miceli | |||
| Leonel Martinez | |||
| Ekaterina I Mokhonova | |||
| Oscar Silva | |||
| Miriana Moran | |||
| Qi L Lu | |||
| Natalia E Sejbuk | |||
| Genevieve C Kendall | |||
| Derek W Wang | |||
| P2860 | cites work | Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle | Q24308714 |
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| Dantrolene stabilizes domain interactions within the ryanodine receptor. | Q45195838 | ||
| Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse | Q45863474 | ||
| Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice | Q45867043 | ||
| Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. | Q45996402 | ||
| Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs | Q46087408 | ||
| Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology | Q46917397 | ||
| Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human | Q57562558 | ||
| Time course of changes in plasma membrane permeability in the dystrophin-deficient mdx mouse | Q58487605 | ||
| P433 | issue | 164 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 164ra160 | |
| P577 | publication date | 2012-12-01 | |
| P1433 | published in | Science Translational Medicine | Q1573955 |
| P1476 | title | Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy | |
| P478 | volume | 4 |
| Q39139178 | 2'-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping |
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| Q41479197 | Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy |
| Q64044936 | DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype |
| Q47159761 | Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease |
| Q38480698 | Drug Discovery of Therapies for Duchenne Muscular Dystrophy |
| Q38257886 | Dysregulation of calcium homeostasis in muscular dystrophies. |
| Q35051963 | Effects of Dantrolene Therapy on Disease Phenotype in Dystrophin Deficient mdx Mice. |
| Q37035750 | Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy. |
| Q91606337 | Gene Therapy for Heart Failure: New Perspectives |
| Q90628254 | Glycine Enhances Satellite Cell Proliferation, Cell Transplantation, and Oligonucleotide Efficacy in Dystrophic Muscle |
| Q36690467 | Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice |
| Q34792390 | High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics |
| Q92515290 | Mitochondrial dysfunction causes Ca2+ overload and ECM degradation-mediated muscle damage in C. elegans |
| Q39212263 | Modulation of aberrant splicing in human RNA diseases by chemical compounds. |
| Q55692383 | Multidirectional Efficacy of Biologically Active Nitro Compounds Included in Medicines. |
| Q38168863 | New developments in the use of gene therapy to treat Duchenne muscular dystrophy |
| Q38115148 | Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy. |
| Q54975630 | PAX3-FOXO1 transgenic zebrafish models identify HES3 as a mediator of rhabdomyosarcoma tumorigenesis. |
| Q50933472 | Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice. |
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| Q38160844 | Role of pseudoexons and pseudointrons in human cancer. |
| Q55230668 | Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice. |
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| Q37210936 | The delivery of therapeutic oligonucleotides |
| Q26827638 | The potential of sarcospan in adhesion complex replacement therapeutics for the treatment of muscular dystrophy |
| Q36740806 | The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides. |
| Q33816688 | The ubiquitin proteasome system in human cardiomyopathies and heart failure |
| Q37455625 | Therapeutic advances in muscular dystrophy |
| Q38996657 | Translational development of splice-modifying antisense oligomers |
| Q52756417 | Treating pediatric neuromuscular disorders: The future is now. |
| Q47147153 | Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice. |
| Q35388411 | Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy |
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