| scholarly article | Q13442814 |
| P2093 | author name string | Brian K Kaspar | |
| Jerry R Mendell | |||
| K Reed Clark | |||
| Vinod Malik | |||
| Zarife Sahenk | |||
| Christopher M Walker | |||
| Louise Rodino-Klapac | |||
| P2860 | cites work | Quadrupling muscle mass in mice by targeting TGF-beta signaling pathways | Q21090110 |
| A mutation in the myostatin gene increases muscle mass and enhances racing performance in heterozygote dogs | Q21090153 | ||
| Myostatin mutation associated with gross muscle hypertrophy in a child | Q24297666 | ||
| Follistatin gene delivery enhances muscle growth and strength in nonhuman primates | Q24598350 | ||
| Clinical characteristics of aged Becker muscular dystrophy patients with onset after 30 years | Q73101996 | ||
| Diagnosis of Duchenne dystrophy by enhanced detection of small mutations | Q74428673 | ||
| Limb-girdle muscular dystrophy in the United States | Q79189595 | ||
| Local dystrophin restoration with antisense oligonucleotide PRO051 | Q80412961 | ||
| Pain and activity limitations in children with Duchenne or Becker muscular dystrophy | Q81604652 | ||
| Evidence-based path to newborn screening for Duchenne muscular dystrophy | Q83749785 | ||
| Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study | Q24616493 | ||
| 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 | ||
| Double muscling in cattle due to mutations in the myostatin gene | Q24648136 | ||
| The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein | Q28118471 | ||
| Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member | Q28237287 | ||
| A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy | Q28272415 | ||
| Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals | Q30050310 | ||
| Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping | Q30493685 | ||
| Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery | Q33730464 | ||
| Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice | Q33871190 | ||
| Rapid direct sequence analysis of the dystrophin gene | Q33905096 | ||
| Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers | Q34003192 | ||
| PTC124 targets genetic disorders caused by nonsense mutations | Q34003720 | ||
| Systemic administration of PRO051 in Duchenne's muscular dystrophy | Q34172661 | ||
| Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. | Q34266296 | ||
| Very mild muscular dystrophy associated with the deletion of 46% of dystrophin | Q34371637 | ||
| Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy | Q34501945 | ||
| Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse | Q34582428 | ||
| Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy | Q35135888 | ||
| Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model | Q35573761 | ||
| Limb-girdle muscular dystrophies--from genetics to molecular pathology | Q35714351 | ||
| Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy. | Q35754170 | ||
| A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy | Q36151226 | ||
| E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice | Q36562319 | ||
| Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors | Q36670010 | ||
| Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity | Q39686097 | ||
| Clinical investigation in duchenne dystrophy: 2. Determination of the ?power? of therapeutic trials based on the natural history | Q41449126 | ||
| Dystrophin immunity in Duchenne's muscular dystrophy. | Q41590387 | ||
| Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy | Q43047105 | ||
| Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins | Q43268180 | ||
| A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C. | Q45360452 | ||
| Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice | Q45731049 | ||
| AAV-mediated gene therapy to the isolated limb in rhesus macaques | Q45865970 | ||
| Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy | Q45883916 | ||
| Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs | Q46087408 | ||
| Family function in families of children with Duchenne muscular dystrophy. | Q50910784 | ||
| Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. | Q53252919 | ||
| Duchenne muscular dystrophy: ethical and emotional considerations in long-term management | Q58668865 | ||
| P433 | issue | 2 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| muscular dystrophy | Q1137767 | ||
| lessons learned | Q1673259 | ||
| P304 | page(s) | 90-99 | |
| P577 | publication date | 2012-05-17 | |
| P1433 | published in | Neuroscience Letters | Q7002625 |
| P1476 | title | Gene therapy for muscular dystrophy: lessons learned and path forward | |
| P478 | volume | 527 |
| Q28611318 | 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands |
| Q39737661 | A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform |
| Q28283362 | AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice |
| Q36422465 | Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy. |
| Q42859445 | Adaptive Immune Response Impairs the Efficacy of Autologous Transplantation of Engineered Stem Cells in Dystrophic Dogs |
| Q35167319 | Advances in therapeutic development for spinal muscular atrophy |
| Q28084979 | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| Q64977765 | Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. |
| Q39016484 | Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicing |
| Q39065043 | Computer experience and further developments in the respiratory function laboratory (author's transl) |
| Q28082625 | Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy |
| Q37164477 | Development, clinical utility, and place of ivacaftor in the treatment of cystic fibrosis |
| Q34555477 | Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients |
| Q26784259 | Duchenne Muscular Dystrophy: From Diagnosis to Therapy |
| Q42175045 | Duchenne muscular dystrophy gene therapy in the canine model |
| Q35641673 | Duchenne muscular dystrophy gene therapy in the canine model. |
| Q35780806 | Duchenne muscular dystrophy: current cell therapies |
| 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 |
| Q45871995 | ECM-Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies |
| Q36412412 | Effects of exogenous neurotrophin-3 on myocyte apoptosis and Ca(2+)-ATP enzyme levels following nerve injury in rats |
| Q40363171 | Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors. |
| Q30383884 | Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method. |
| Q26786571 | Emerging therapeutic options for sporadic inclusion body myositis |
| Q38848060 | Evolving lessons on nanomaterial-coated viral vectors for local and systemic gene therapy |
| Q38260248 | Gene replacement therapy for genetic hepatocellular jaundice |
| Q38152406 | Gene-based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation |
| Q38523086 | Genetic manipulation of cardiac ageing |
| Q91916997 | Glucocorticoids counteract hypertrophic effects of myostatin inhibition in dystrophic muscle |
| Q26750393 | Human induced pluripotent stem cells for monogenic disease modelling and therapy |
| Q90571083 | Increasing lean muscle mass in mice via nanoparticle-mediated hepatic delivery of follistatin mRNA |
| Q33736192 | Influence of immune responses in gene/stem cell therapies for muscular dystrophies |
| Q99239337 | Intra-tracheal delivery of AAV6 vectors results in sustained transduction in murine lungs without genomic integration |
| Q47345657 | Laminin-deficient muscular dystrophy: Molecular pathogenesis and structural repair strategies. |
| Q92535136 | Linker Protein Repair of LAMA2 Dystrophic Neuromuscular Basement Membranes |
| Q44861538 | Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery |
| Q45857273 | Modeling functional decline over time in sporadic inclusion body myositis. |
| Q91594164 | Muscular dystrophies |
| Q26992271 | Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy |
| Q42240676 | Novel adeno-associated viral vector delivering the utrophin gene regulator jazz counteracts dystrophic pathology in mdx mice |
| Q34341005 | One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development |
| Q33996884 | RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies |
| Q39351028 | RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice. |
| Q33944130 | Skeletal muscle homeostasis in duchenne muscular dystrophy: modulating autophagy as a promising therapeutic strategy |
| Q27679454 | Structural Insights into Adeno-Associated Virus Serotype 5 |
| Q34001776 | Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs. |
| Q38168181 | The era of genomic medicine. |
| Q33902499 | The potential of adeno-associated viral vectors for gene delivery to muscle tissue |
| Q26827638 | The potential of sarcospan in adhesion complex replacement therapeutics for the treatment of muscular dystrophy |
| Q37455625 | Therapeutic advances in muscular dystrophy |
| Q44671291 | Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches |
| Q34202579 | Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay |
| Q27324284 | Trendelenburg-Like Gait, Instability and Altered Step Patterns in a Mouse Model for Limb Girdle Muscular Dystrophy 2i |
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