| scholarly article | Q13442814 |
| P819 | ADS bibcode | 2002PNAS...99.9456D |
| P356 | DOI | 10.1073/PNAS.142302299 |
| P932 | PMC publication ID | 123162 |
| P698 | PubMed publication ID | 12077324 |
| P5875 | ResearchGate publication ID | 11298350 |
| P50 | author | Barbara Berarducci | Q130280513 |
| P2093 | author name string | Giulio Cossu | |
| Enzo Ricci | |||
| Fernanda Gabriella De Angelis | |||
| Giuliana Galluzzi | |||
| Irene Bozzoni | |||
| Olga Sthandier | |||
| Silvia Toso | |||
| P2860 | cites work | The low abundance of U7 snRNA is partly determined by its Sm binding site | Q24564682 |
| Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs | Q24569504 | ||
| 3' end processing of mouse histone pre-mRNA: evidence for additional base-pairing between U7 snRNA and pre-mRNA | Q24605803 | ||
| Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human beta-thalassemic mutations | Q28137724 | ||
| Multiple processing-defective mutations in a mammalian histone pre-mRNA are suppressed by compensatory changes in U7 RNA both in vivo and in vitro | Q28296716 | ||
| Advanced mammalian gene transfer: high titre retroviral vectors with multiple drug selection markers and a complementary helper-free packaging cell line | Q29547239 | ||
| A compensatory base change in U1 snRNA suppresses a 5' splice site mutation | Q29618292 | ||
| Tethering ribozymes to a retroviral packaging signal for destruction of viral RNA. | Q30466758 | ||
| Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. | Q33868227 | ||
| Antisense effects in the cell nucleus: modification of splicing. | Q34332181 | ||
| The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization. | Q34361133 | ||
| Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides | Q34470860 | ||
| Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse | Q34582428 | ||
| Ribozyme-mediated inhibition of HIV 1 suggests nucleolar trafficking of HIV-1 RNA. | Q35192070 | ||
| The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion | Q35197608 | ||
| Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model | Q35573761 | ||
| Persistence in muscle of an adenoviral vector that lacks all viral genes | Q36018705 | ||
| Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides | Q36684554 | ||
| Identification of the sequences responsible for the splicing phenotype of the regulatory intron of the L1 ribosomal protein gene of Xenopus laevis | Q36962943 | ||
| U1 small nuclear RNA chimeric ribozymes with substrate specificity for the Rev pre-mRNA of human immunodeficiency virus | Q37495569 | ||
| Molecular mechanisms of antisense drugs: RNase H. | Q38337536 | ||
| Carrier detection and prenatal diagnosis in Duchenne and Becker muscular dystrophy | Q38738023 | ||
| Transfection of large plasmids in primary human myoblasts. | Q40778614 | ||
| Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells | Q40790512 | ||
| The fate of individual myoblasts after transplantation into muscles of DMD patients | Q40883106 | ||
| The Rev protein is able to transport to the cytoplasm small nucleolar RNAs containing a Rev binding element. | Q40935808 | ||
| A novel U2-U6 snRNA structure is necessary for mammalian mRNA splicing | Q41354468 | ||
| Multiplex Western blotting system for the analysis of muscular dystrophy proteins | Q42108368 | ||
| Reversible immortalization of human myogenic cells by site-specific excision of a retrovirally transferred oncogene. | Q42812685 | ||
| Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts | Q43465378 | ||
| Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route | Q45866613 | ||
| Sequences required for 3' end formation of human U2 small nuclear RNA. | Q48376864 | ||
| Genetic evidence for base pairing between U2 and U6 snRNA in mammalian mRNA splicing | Q59095420 | ||
| Frequency of Duchenne muscular dystrophy carriers | Q66694732 | ||
| RNA as an enzyme | Q69068654 | ||
| Biochemical complementation with RNA in the Xenopus oocyte: A small rna is required for the generation of 3′ histone mRNA termini | Q72714199 | ||
| Restoration of correct splicing of thalassemic beta-globin pre-mRNA by modified U1 snRNAs | Q74254054 | ||
| P433 | issue | 14 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 9456-9461 | |
| P577 | publication date | 2002-06-20 | |
| P1433 | published in | Proceedings of the National Academy of Sciences of the United States of America | Q1146531 |
| P1476 | title | 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 | |
| P478 | volume | 99 |
| Q44385966 | A brief history of novel drug discovery technologies |
| Q44992027 | A new vector, based on the PolII promoter of the U1 snRNA gene, for the expression of siRNAs in mammalian cells |
| Q34267400 | Advances in Duchenne muscular dystrophy gene therapy |
| Q44879918 | Alternative splicing in disease and therapy |
| Q41944520 | Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense |
| Q34574529 | Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. |
| Q34264478 | Challenging the dogma: the hidden layer of non‐protein‐coding RNAs in complex organisms |
| Q90530644 | Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy |
| Q44960581 | Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells |
| Q41671504 | Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations. |
| Q28290763 | Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes |
| Q34278933 | Dystrophin and mutations: one gene, several proteins, multiple phenotypes |
| Q36960845 | Emerging strategies for cell and gene therapy of the muscular dystrophies |
| Q39392603 | Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy |
| Q24631244 | Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy |
| Q79120762 | Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle |
| Q33722582 | Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA. |
| Q41136576 | Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts |
| Q56990656 | Exon Skipping in a Dysf-Missense Mutant Mouse Model |
| Q39692533 | Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping |
| Q45863474 | Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse |
| Q37519650 | Gene therapy for muscular dystrophy: current progress and future prospects. |
| Q33712930 | Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles |
| Q38516522 | Genome mapping and expression analyses of human intronic noncoding RNAs reveal tissue-specific patterns and enrichment in genes related to regulation of transcription |
| Q50131827 | Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders |
| Q34343780 | In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse |
| Q38823382 | Longitudinal MRI quantification of muscle degeneration in Duchenne muscular dystrophy. |
| Q36179776 | MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy. |
| Q58413953 | Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model |
| Q92489474 | Recent advances in Duchenne muscular dystrophy |
| Q34762623 | Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy. |
| Q38079633 | Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies |
| Q30514014 | Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping |
| Q34004566 | Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing |
| Q37211004 | Splice-switching antisense oligonucleotides as therapeutic drugs |
| Q45007187 | Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides |
| Q24680286 | Telomerase RNA level limits telomere maintenance in X-linked dyskeratosis congenita |
| Q39480191 | The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery |
| Q35808837 | The interplay between microRNAs and the neurotrophin receptor tropomyosin-related kinase C controls proliferation of human neuroblastoma cells. |
| Q34473336 | The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy |
| Q34982869 | Therapeutic approaches to muscular dystrophy. |
| Q35952733 | Therapeutic strategies for Duchenne and Becker dystrophies |
| Q38289261 | Viral Vector-Mediated Antisense Therapy for Genetic Diseases |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
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