| scholarly article | Q13442814 |
| P50 | author | Tania Incitti | Q39692804 |
| Ivano Legnini | Q39692847 | ||
| P2093 | author name string | Irene Bozzoni | |
| Olga Sthandier | |||
| Fernanda G De Angelis | |||
| Chiara Pinnarò | |||
| Valentina Cazzella | |||
| P2860 | cites work | Antibodies to small nuclear RNAs complexed with proteins are produced by patients with systemic lupus erythematosus | Q24561667 |
| 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 | Q24643018 | ||
| Coupling between snoRNP assembly and 3' processing controls box C/D snoRNA biosynthesis in yeast | Q27934470 | ||
| U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping | Q28203179 | ||
| Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping | Q28291830 | ||
| Serial passaging and differentiation of myogenic cells isolated from dystrophic mouse muscle | Q29615169 | ||
| ESEfinder: A web resource to identify exonic splicing enhancers | Q29616126 | ||
| Listening to silence and understanding nonsense: exonic mutations that affect splicing | Q29618493 | ||
| Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals | Q30050310 | ||
| Becker muscular dystrophy patient with a large intragenic dystrophin deletion: implications for functional minigenes and gene therapy | Q33597923 | ||
| Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide | Q33991414 | ||
| Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells | Q34035676 | ||
| Function and genetics of dystrophin and dystrophin-related proteins in muscle | Q34120764 | ||
| Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene | Q34190227 | ||
| Very mild muscular dystrophy associated with the deletion of 46% of dystrophin | Q34371637 | ||
| Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. | Q34574529 | ||
| New aspects of calcium signaling in skeletal muscle cells: implications in Duchenne muscular dystrophy | Q35006083 | ||
| Dystrophin, its interactions with other proteins, and implications for muscular dystrophy | Q36531055 | ||
| Emerging strategies for cell and gene therapy of the muscular dystrophies | Q36960845 | ||
| HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment | Q37039472 | ||
| Tannic acid facilitates expression of the polypyrimidine tract binding protein and alleviates deleterious inclusion of CHRNA1 exon P3A due to an hnRNP H-disrupting mutation in congenital myasthenic syndrome | Q37128363 | ||
| Gene therapy for muscular dystrophy: current progress and future prospects. | Q37519650 | ||
| Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides | Q38306997 | ||
| Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites | Q38317038 | ||
| 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. | Q40655750 | ||
| The Rev protein is able to transport to the cytoplasm small nucleolar RNAs containing a Rev binding element. | Q40935808 | ||
| An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus | Q42658269 | ||
| Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice | Q42686363 | ||
| Control of myogenesis in the mouse myogenic C2 cell line by medium composition and by insulin: characterization of permissive and inducible C2 myoblasts | Q42820268 | ||
| Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. | Q43727676 | ||
| Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice | Q43769886 | ||
| Correction of disease-associated exon skipping by synthetic exon-specific activators | Q44276105 | ||
| Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides | Q45007187 | ||
| Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. | Q45394829 | ||
| Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology | Q46917397 | ||
| Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. | Q53775896 | ||
| Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. | Q54781099 | ||
| Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression | Q80141652 | ||
| Local dystrophin restoration with antisense oligonucleotide PRO051 | Q80412961 | ||
| P4510 | describes a project that uses | ImageQuant | Q112270642 |
| P433 | issue | 9 | |
| P921 | main subject | Duchenne muscular dystrophy | Q1648484 |
| P304 | page(s) | 1675-1682 | |
| P577 | publication date | 2010-06-15 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping | |
| P478 | volume | 18 |
| Q24630341 | A long noncoding RNA controls muscle differentiation by functioning as a competing endogenous RNA |
| Q42639922 | Activation of AKT signaling promotes cell growth and survival in α7β1 integrin-mediated alleviation of muscular dystrophy |
| Q38211673 | Development of therapeutic splice-switching oligonucleotides. |
| Q41671504 | Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations. |
| Q41136576 | Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts |
| Q45389853 | Exon deletion patterns of the dystrophin gene in 82 Vietnamese Duchenne/Becker muscular dystrophy patients |
| Q38314019 | In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs. |
| Q35802092 | Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping |
| Q29048207 | Novel long noncoding RNAs (lncRNAs) in myogenesis: a miR-31 overlapping lncRNA transcript controls myoblast differentiation |
| Q37867701 | Opportunities and challenges for the development of antisense treatment in neuromuscular disorders |
| Q37919973 | RNA-based therapeutic approaches for coagulation factor deficiencies |
| Q38079633 | Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies |
| Q35072755 | Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through |
| Q36577433 | The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials |
| Q36529176 | The lack of the Celf2a splicing factor converts a Duchenne genotype into a Becker phenotype |
| Q38289261 | Viral Vector-Mediated Antisense Therapy for Genetic Diseases |
| Q35913120 | mRNA transcript diversity creates new opportunities for pharmacological intervention |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
Search more.