| scholarly article | Q13442814 |
| P2093 | author name string | Jeffrey Rosenfeld | |
| Steve D Wilton | |||
| Terence A Partridge | |||
| Qi Long Lu | |||
| Fang Lou | |||
| HaiFang Yin | |||
| Adam Rabinowitz | |||
| Julia Alter | |||
| P2860 | cites work | Screening for antisense modulation of dystrophin pre-mRNA splicing | Q34807136 |
| Antisense technologies. Improvement through novel chemical modifications | Q35106663 | ||
| Systemically delivered antisense oligomers upregulate gene expression in mouse tissues | Q44212614 | ||
| Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. | Q44521172 | ||
| Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse | Q45863474 | ||
| Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. | Q52547202 | ||
| Morpholino antisense oligomers: design, preparation, and properties | Q28243074 | ||
| Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping | Q28291830 | ||
| Dystrophin: the protein product of the Duchenne muscular dystrophy locus | Q29618077 | ||
| Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles | Q33715654 | ||
| Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements | Q33980928 | ||
| Systemic delivery of genes to striated muscles using adeno-associated viral vectors. | Q34347199 | ||
| Very mild muscular dystrophy associated with the deletion of 46% of dystrophin | Q34371637 | ||
| P433 | issue | 2 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | pathology | Q7208 |
| P304 | page(s) | 175-177 | |
| P577 | publication date | 2006-01-29 | |
| P1433 | published in | Nature Medicine | Q1633234 |
| P1476 | title | Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology | |
| P478 | volume | 12 |
| Q34175938 | A Human-Specific Deletion in Mouse Cmah Increases Disease Severity in the mdx Model of Duchenne Muscular Dystrophy |
| Q26863684 | A chemical view of oligonucleotides for exon skipping and related drug applications |
| Q38360072 | A deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment. |
| Q39444689 | A deficit of brain dystrophin impairs specific amygdala GABAergic transmission and enhances defensive behaviour in mice |
| Q37374581 | A gain of function mutation causing skeletal overgrowth in the rapunzel mutant |
| Q41877911 | A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides |
| Q34241874 | A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse |
| Q45878156 | AAV-dependent targeting of myostatin function: follistatin strikes back at muscular dystrophy. |
| Q36774636 | Alternative splicing: an emerging topic in molecular and clinical oncology |
| Q90240748 | An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement |
| Q95780698 | An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models |
| Q37165337 | An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models |
| Q28084979 | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| Q28475196 | Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient |
| Q37859009 | Antisense drug discovery and development |
| Q36718923 | Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice |
| Q42122236 | Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD) |
| Q27480935 | Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries |
| Q55043089 | Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. |
| Q55018458 | Antisense oligonucleotides in neurological disorders. |
| Q47344432 | Antisense oligonucleotides: the next frontier for treatment of neurological disorders |
| Q27007056 | Antisense oligonucleotides: treating neurodegeneration at the level of RNA |
| Q33289993 | Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy |
| Q38340328 | Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. |
| Q37910743 | Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain |
| Q24682555 | Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications |
| Q34883011 | Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy |
| Q33416809 | Blastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice |
| Q42520819 | CD90-positive cells, an additional cell population, produce laminin alpha2 upon transplantation to dy(3k)/dy(3k) mice. |
| Q37807346 | CPP-Directed Oligonucleotide Exon Skipping in Animal Models of Duchenne Muscular Dystrophy |
| Q92523166 | CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors |
| Q57061444 | Cardiac Management of the Patient With Duchenne Muscular Dystrophy |
| Q37889770 | Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions |
| Q33713471 | Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse |
| Q37013966 | Cell penetrating peptide conjugates of steric block oligonucleotides |
| Q37629659 | Cell penetrating peptides: overview and applications to the delivery of oligonucleotides |
| Q37944530 | Cell-matrix interactions in muscle disease |
| Q46375612 | Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. |
| Q37631779 | Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells. |
| Q37907407 | Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. |
| Q34556906 | Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice |
| Q90530644 | Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy |
| Q37389308 | Clinical uses of microbubbles in diagnosis and treatment |
| Q33931629 | Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice |
| Q33637391 | Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency |
| Q24679378 | Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy |
| Q35600455 | Could exon skipping help dystrophic boys to run, hop, and jump? |
| Q37277411 | Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy |
| Q89220022 | Current and Emerging Therapies for Duchenne Muscular Dystrophy |
| Q34994545 | Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. |
| Q28082625 | Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy |
| Q39225878 | Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy |
| Q37154271 | Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy |
| Q38369597 | Delivery of therapeutic oligonucleotides with cell penetrating peptides. |
| Q38357434 | Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. |
| Q36078437 | Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides |
| Q37276200 | Development of a general methodology for labelling peptide-morpholino oligonucleotide conjugates using alkyne-azide click chemistry |
| Q37105210 | Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy |
| Q33908369 | Differential requirement for utrophin in the induced pluripotent stem cell correction of muscle versus fat in muscular dystrophy mice |
| Q43275640 | Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino |
| Q58826477 | Drug discovery for spinal muscular atrophy |
| Q36482835 | Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy. |
| Q35052506 | Duchenne muscular dystrophy gene therapy: Lost in translation? |
| Q33325470 | Dynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides |
| Q33608478 | Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies |
| Q58716650 | Dystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision Medicine |
| Q33930110 | Dystrophin isoform induction in vivo by antisense-mediated alternative splicing. |
| Q43124526 | Dystrophin knockdown mice suggest that early, transient dystrophin expression might be enough to prevent later pathology |
| Q43243962 | Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP-AON complexes |
| Q44264343 | Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice |
| Q36897347 | Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer |
| Q33594805 | Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy |
| Q46087408 | Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs |
| Q34675818 | Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice |
| Q36340978 | Elusive sources of variability of dystrophin rescue by exon skipping |
| Q41390937 | Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle |
| Q33796975 | Emerging genetic therapies to treat Duchenne muscular dystrophy |
| Q36960845 | Emerging strategies for cell and gene therapy of the muscular dystrophies |
| Q33278100 | Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon |
| Q33280999 | Environmental enrichment effects on development of retinal ganglion cell dendritic stratification require retinal BDNF. |
| Q39692533 | Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping |
| Q38014906 | Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? |
| Q34204198 | Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice |
| Q38186137 | Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases |
| Q35731229 | Exon-skipping therapy: a roadblock, detour, or bump in the road? |
| Q35740521 | Expanding the action of duplex RNAs into the nucleus: redirecting alternative splicing |
| Q34508301 | Experimental models of duchenne muscular dystrophy: relationship with cardiovascular disease |
| Q36297940 | Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. |
| Q37254647 | Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice |
| Q39044313 | Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. |
| Q42412768 | Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. |
| Q34180144 | Gene Therapy for Muscular Dystrophies: Progress and Challenges |
| Q33831193 | Gene and cell-mediated therapies for muscular dystrophy. |
| Q37171838 | Gene doping: the hype and the reality. |
| Q34016744 | Gene knockdowns in adult animals: PPMOs and vivo-morpholinos |
| Q38829627 | Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy |
| Q37767924 | Gene therapy for muscle disease |
| Q37519650 | Gene therapy for muscular dystrophy: current progress and future prospects. |
| Q37396408 | Gene therapy in large animal models of muscular dystrophy |
| Q34576814 | Gene therapy progress and prospects: Duchenne muscular dystrophy |
| Q58223916 | Generation and Characterization of Transgenic Mice with the Full-length HumanDMDGene |
| Q36690467 | Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice |
| Q28472268 | Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening |
| Q41949953 | Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy. |
| Q36972091 | Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. |
| Q64934843 | In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient. |
| Q35582583 | In vitro stability of therapeutically relevant, internally truncated dystrophins |
| Q34123466 | In vivo MRI characterization of progressive cardiac dysfunction in the mdx mouse model of muscular dystrophy. |
| Q36499173 | In vivo correction of a Menkes disease model using antisense oligonucleotides |
| Q39502175 | In vivo myogenic potential of human CD133+ muscle-derived stem cells: a quantitative study |
| Q34343780 | In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse |
| Q90089689 | Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy |
| Q35014916 | Interventions for muscular dystrophy: molecular medicines entering the clinic |
| Q84480135 | Less is more: therapeutic exon skipping for Duchenne muscular dystrophy |
| Q47288511 | Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping |
| Q36964393 | Linking cytoarchitecture to metabolism: sarcolemma-associated plectin affects glucose uptake by destabilizing microtubule networks in mdx myofibers |
| Q24643018 | 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 |
| Q64254546 | Long Noncoding RNA and Epithelial Mesenchymal Transition in Cancer |
| Q89717338 | Long non-coding RNAs in immune regulation and their potential as therapeutic targets |
| Q42507352 | Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models |
| Q33573648 | Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers |
| Q42257279 | Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino |
| Q28304022 | MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction |
| Q34497055 | Mammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications |
| Q36270889 | Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo |
| Q45883797 | Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer. |
| Q36866549 | Modulating the expression of disease genes with RNA-based therapy. |
| Q37799373 | Molecular Mechanisms and Treatment Options for Muscle Wasting Diseases |
| Q59358387 | Molecular Therapies for Muscular Dystrophies |
| Q36593241 | Molecular mechanisms of muscular dystrophies: old and new players |
| Q37142004 | Molecular-targeted therapy for Duchenne muscular dystrophy: progress and potential |
| Q80501077 | Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse |
| Q37562102 | Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice |
| Q34206305 | Morpholino-mediated increase in soluble Flt-1 expression results in decreased ocular and tumor neovascularization |
| Q39342422 | Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy |
| Q36369845 | Multiple exon skipping strategies to by-pass dystrophin mutations |
| Q57286631 | Muscle membrane integrity in Duchenne muscular dystrophy: recent advances in copolymer-based muscle membrane stabilizers |
| Q53343843 | Muscling in: Gene therapies for muscular dystrophy target RNA. |
| Q52560217 | Mutation of IPO13 causes recessive ocular coloboma, microphthalmia, and cataract. |
| Q64068599 | Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy |
| Q42378896 | Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle |
| Q37329544 | Myotonic dystrophy: therapeutic strategies for the future |
| Q37783315 | NMD: RNA biology meets human genetic medicine |
| Q58549842 | NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy |
| Q33326762 | Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice |
| Q79431189 | Neuromuscular disorders: therapeutic advances |
| Q37814437 | New insights in gene-derived therapy: the example of Duchenne muscular dystrophy |
| Q50283542 | Nitric oxide donors improve prednisone effects on muscular dystrophy in the mdx mouse diaphragm |
| Q28544896 | Non-invasive MRI and spectroscopy of mdx mice reveal temporal changes in dystrophic muscle imaging and in energy deficits |
| Q64104371 | Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results |
| Q36925191 | Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents |
| Q37255013 | Novel therapeutic modalities to address nondrugable protein interaction targets |
| Q33713525 | Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice |
| Q34621772 | One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice |
| Q37867701 | Opportunities and challenges for the development of antisense treatment in neuromuscular disorders |
| Q96126745 | Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy |
| Q39882118 | Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. |
| Q34171630 | Optimizing exon skipping therapies for DMD. |
| Q37419563 | Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides. |
| Q30235525 | Overcoming cellular barriers for RNA therapeutics |
| Q48620752 | PMO Delivery System Using Bubble Liposomes and Ultrasound Exposure for Duchenne Muscular Dystrophy Treatment |
| Q36141218 | Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy |
| Q36939725 | Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice |
| Q93138367 | Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases |
| Q81825063 | Peptide-conjugated antisense therapy takes a skip ahead |
| Q37347451 | Peptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: quantitative evaluation of overall efficacy employing easy to handle reporter systems |
| Q41854668 | Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice |
| Q37831858 | Perspectives on gene therapy in myotonic dystrophy type 1. |
| Q34463222 | Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity. |
| Q34473309 | Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy. |
| Q35084227 | Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. |
| Q41890992 | Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. |
| Q37338790 | Polymersome delivery of siRNA and antisense oligonucleotides. |
| Q34580135 | Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy. |
| Q39880997 | Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model |
| Q37767638 | Present and future of antisense therapy for splicing modulation in inherited metabolic disease |
| Q30493685 | Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping |
| Q30626615 | Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice |
| Q36773242 | Progress in gene therapy of dystrophic heart disease |
| Q34734708 | Progress in muscular dystrophy research with special emphasis on gene therapy |
| Q37609948 | Progress in therapeutic antisense applications for neuromuscular disorders |
| Q37908904 | Progress in therapy for Duchenne muscular dystrophy. |
| Q37021623 | Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy |
| Q33398588 | Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm. |
| Q37676955 | Proteomic profiling of x-linked muscular dystrophy |
| Q46849509 | Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency |
| Q29615183 | RNA and disease |
| Q37079038 | RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice |
| Q34248493 | RNA therapeutics: beyond RNA interference and antisense oligonucleotides |
| Q36714202 | Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy |
| Q38079633 | Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies |
| Q55000730 | Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse. |
| Q30514014 | Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping |
| Q35750055 | Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy |
| Q45033314 | Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study |
| Q35070602 | Shielding the messenger (RNA): microRNA-based anticancer therapies |
| Q29039090 | Silencing disease genes in the laboratory and the clinic |
| Q37211004 | Splice-switching antisense oligonucleotides as therapeutic drugs |
| Q27009581 | Splicing therapy for neuromuscular disease |
| Q37165327 | Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. |
| Q37116446 | Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. |
| Q28478166 | Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development |
| Q37909000 | Targeting RNA to treat neuromuscular disease |
| Q39154733 | Targeting Splicing in the Treatment of Human Disease |
| Q34459257 | Targeting nuclear RNA for in vivo correction of myotonic dystrophy |
| Q37082470 | Technology insight: therapy for Duchenne muscular dystrophy-an opportunity for personalized medicine? |
| Q45758649 | Temperature dependent behavior of ultrasound contrast agents |
| Q38257312 | The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice |
| Q38772751 | The Role of Reactive Oxygen and Nitrogen Species in the Expression and Splicing of Nitric Oxide Receptor |
| Q35799722 | The evolution of heart gene delivery vectors |
| Q62109034 | The macrophage as a Trojan horse for antisense oligonucleotide delivery |
| Q28656377 | The mdx mouse model as a surrogate for Duchenne muscular dystrophy |
| Q36963079 | The potential of exon skipping for treatment for Duchenne muscular dystrophy |
| Q34473336 | The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy |
| Q35881677 | The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse |
| Q37383091 | The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies |
| Q36897394 | The versatility of oligonucleotides as potential therapeutics |
| Q34926082 | Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations |
| Q49961986 | Therapeutic Targeting of Long Non-Coding RNAs in Cancer |
| Q34982869 | Therapeutic approaches to muscular dystrophy. |
| Q53263857 | Therapeutic effects of exon skipping and losartan on skeletal muscle of mdx mice. |
| Q37363171 | Therapeutic potential of splice-switching oligonucleotides |
| Q36549699 | Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy. |
| Q33678893 | Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy |
| Q36841572 | Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy |
| Q38996657 | Translational development of splice-modifying antisense oligomers |
| Q89763505 | Triazine-cored polymeric vectors for antisense oligonucleotide delivery in vitro and in vivo |
| Q42827488 | Use of vivo-morpholinos for control of protein expression in the adult rat brain |
| Q35388411 | Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy |
| Q83334456 | [Assessment of tumour angiogenesis using contrast-enhanced ultrasound] |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
Search more.