| scholarly article | Q13442814 |
| P819 | ADS bibcode | 2006PNAS..103.3758D |
| P356 | DOI | 10.1073/PNAS.0508917103 |
| P932 | PMC publication ID | 1450150 |
| P698 | PubMed publication ID | 16501048 |
| P5875 | ResearchGate publication ID | 7276609 |
| P50 | author | Michela A. Denti | Q38320761 |
| Alessandro Rosa | Q41002249 | ||
| Valeria Parente | Q46447806 | ||
| Giuseppe D'Antona | Q46447827 | ||
| Alberto Auricchio | Q56950196 | ||
| Carmine Nicoletti | Q57090924 | ||
| Antonio Musarò | Q71815476 | ||
| Mariacarmela Allocca | Q91518883 | ||
| P2093 | author name string | Orietta Pansarasa | |
| Roberto Bottinelli | |||
| Fernanda Gabriella De Angelis | |||
| Irene Bozzoni | |||
| Olga Sthandier | |||
| P2860 | cites work | U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping | Q28203179 |
| Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping | Q28291830 | ||
| Cell Therapy of -Sarcoglycan Null Dystrophic Mice Through Intra-Arterial Delivery of Mesoangioblasts | Q29302095 | ||
| Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. | Q31934499 | ||
| Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles | Q33715654 | ||
| 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 | ||
| Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. | Q34190967 | ||
| Advances in Duchenne muscular dystrophy gene therapy | Q34267400 | ||
| Systemic delivery of genes to striated muscles using adeno-associated viral vectors. | Q34347199 | ||
| 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 | ||
| 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 | ||
| Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle | Q35835194 | ||
| Inhibition of human immunodeficiency virus type 1 replication by nuclear chimeric anti-HIV ribozymes in a human T lymphoblastoid cell line | Q41049538 | ||
| Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense | Q41944520 | ||
| Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. | Q43727676 | ||
| Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model | Q43829645 | ||
| The effect of ageing and immobilization on structure and function of human skeletal muscle fibres. | Q44619547 | ||
| Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides | Q45007187 | ||
| Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart | Q45507049 | ||
| Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse | Q45863474 | ||
| Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors | Q45873434 | ||
| Human circulating AC133+ stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle | Q57268519 | ||
| Sustained muscle expression of dystrophin from a high-capacity adenoviral vector with systemic gene transfer of T cell costimulatory blockade | Q64379313 | ||
| The molecular basis of muscular dystrophy in the mdx mouse: a point mutation | Q69514522 | ||
| Force-velocity properties of human skeletal muscle fibres: myosin heavy chain isoform and temperature dependence | Q71718783 | ||
| P433 | issue | 10 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| Duchenne muscular dystrophy | Q1648484 | ||
| mdx mouse | Q24976962 | ||
| P304 | page(s) | 3758-3763 | |
| P577 | publication date | 2006-02-24 | |
| P1433 | published in | Proceedings of the National Academy of Sciences of the United States of America | Q1146531 |
| P1476 | title | Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model | |
| P478 | volume | 103 |
| Q24630341 | A long noncoding RNA controls muscle differentiation by functioning as a competing endogenous RNA |
| Q37245439 | A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. |
| Q37140645 | AAV vectors for RNA-based modulation of gene expression |
| Q45878156 | AAV-dependent targeting of myostatin function: follistatin strikes back at muscular dystrophy. |
| Q37687423 | AAV-directed muscular dystrophy gene therapy |
| Q28508977 | AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis |
| Q42224211 | An engineered U1 small nuclear RNA rescues splicing defective coagulation F7 gene expression in mice. |
| Q28084979 | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| Q35888398 | Approaching a new age in Duchenne muscular dystrophy treatment |
| Q33713471 | Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse |
| Q43142245 | Characterization of the specific and sustained GH1 expression induced by rAAV2/1 in normal adult male rats |
| Q33625875 | Delivery is key: lessons learnt from developing splice-switching antisense therapies |
| Q37299072 | Designing heart performance by gene transfer. |
| Q41671504 | Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations. |
| Q36968872 | Efficient whole-body transduction with trans-splicing adeno-associated viral vectors |
| Q41136576 | Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts |
| Q33598293 | Exon exchange approach to repair Duchenne dystrophin transcripts |
| Q39692533 | Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping |
| Q40773636 | Functional and Morphological Improvement of Dystrophic Muscle by Interleukin 6 Receptor Blockade. |
| Q33831193 | Gene and cell-mediated therapies for muscular dystrophy. |
| Q37171838 | Gene doping: the hype and the reality. |
| Q37767924 | Gene therapy for muscle disease |
| Q37519650 | Gene therapy for muscular dystrophy: current progress and future prospects. |
| Q34576814 | Gene therapy progress and prospects: Duchenne muscular dystrophy |
| Q45875164 | Get personal with gene therapy for muscular dystrophy |
| Q36086772 | Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6 |
| Q47260510 | Inhibition of antigen presentation during AAV gene therapy using virus peptides |
| Q27438151 | Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression |
| Q35119851 | Intratumoral decorin gene delivery by AAV vector inhibits brain glioblastomas and prolongs survival of animals by inducing cell differentiation |
| Q33339391 | L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle |
| Q33573648 | Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers |
| Q35802092 | Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping |
| Q35612576 | Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy |
| Q36179105 | Molecular and cellular mechanisms of skeletal muscle atrophy: an update. |
| Q36593241 | Molecular mechanisms of muscular dystrophies: old and new players |
| Q79431189 | Neuromuscular disorders: therapeutic advances |
| Q37867701 | Opportunities and challenges for the development of antisense treatment in neuromuscular disorders |
| Q60054004 | Oxidative muscles have better mitochondrial homeostasis than glycolytic muscles throughout life and maintain mitochondrial function during aging |
| Q81825063 | Peptide-conjugated antisense therapy takes a skip ahead |
| Q37801839 | Physiological and tissue-specific vectors for treatment of inherited diseases |
| Q45408008 | Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors |
| Q58413953 | Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model |
| Q37609948 | Progress in therapeutic antisense applications for neuromuscular disorders |
| Q92162442 | RNA Therapeutics: How Far Have We Gone? |
| Q29615183 | RNA and disease |
| Q49196789 | RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect. |
| Q35549208 | Relationships of thigh muscle contractile and non-contractile tissue with function, strength, and age in boys with Duchenne muscular dystrophy |
| Q37719612 | Skeletal muscle fibre diversity and the underlying mechanisms. |
| Q45861904 | Soluble TNF-α receptor secretion from healthy or dystrophic mice after AAV6-mediated muscle gene transfer |
| Q59690733 | Structural and functional alterations of muscle fibres in the novel mouse model of facioscapulohumeral muscular dystrophy |
| Q37116446 | Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. |
| Q36780738 | Targeting RNA splicing for disease therapy |
| Q37082470 | Technology insight: therapy for Duchenne muscular dystrophy-an opportunity for personalized medicine? |
| Q37383091 | The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies |
| Q34926082 | Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations |
| Q34982869 | Therapeutic approaches to muscular dystrophy. |
| Q36549699 | Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy. |
| Q53775896 | Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. |
| Q38289261 | Viral Vector-Mediated Antisense Therapy for Genetic Diseases |
| Q35847178 | Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
| Q42126374 | miRNAs as serum biomarkers for Duchenne muscular dystrophy |
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