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Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 8, p. 1341, doi. 10.1038/mt.2015.88
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- Publication type:
- Article
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 1, p. 43, doi. 10.1038/mt.2014.178
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- Publication type:
- Article
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases.
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- Molecular Therapy, 2013, v. 21, n. 2, p. 266, doi. 10.1038/mt.2013.4
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- Publication type:
- Article
Transcription Factor Rational Design Improves Directed Differentiation of Human Mesenchymal Stem Cells Into Skeletal Myocytes.
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- Molecular Therapy, 2011, v. 19, n. 7, p. 1331, doi. 10.1038/mt.2010.308
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- Publication type:
- Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 830, doi. 10.1038/mt.2011.59
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- Publication type:
- Article
Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 2, p. 345, doi. 10.1038/mt.2010.261
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- Publication type:
- Article
Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa guanidine Morpholino Oligomer Treatment.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 1, p. 159, doi. 10.1038/mt.2010.212
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- Publication type:
- Article
Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of Exon Skipping of the Human DMD Gene.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 3, p. 554, doi. 10.1038/mt.2008.287
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- Publication type:
- Article
Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 11, p. 1825, doi. 10.1038/mt.2008.186
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- Publication type:
- Article
907. Development of Recombinant Novel Adeno-Associated Viral (rAAV) Vectors Encoding Optimised Microdystrophin cDNAs for Duchenne Muscular Dystrophy (DMD).
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- Molecular Therapy, 2006, v. 13, p. S349, doi. 10.1016/j.ymthe.2006.08.997
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- Publication type:
- Article
1022. Efficient Dystrophin Production in Response to Local and Systemic Administration of Morpholino Antisense Oligonucleotides in the Mdx Mouse.
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- Molecular Therapy, 2006, v. 13, p. S393, doi. 10.1016/j.ymthe.2006.08.1117
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- Publication type:
- Article
1070. Easy Search on Clinical Gene Transfer: The Euregenethy (EC-DG Research FP5-TN) Data Base on Pre-Clinical & Clinical Gene Transfer & Therapy
- Published in:
- 2005
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- Publication type:
- Abstract
261. Recombinant Adeno-Associated Viral (rAAV) Microdystrophin Vectors as Therapeutic Tools for Duchenne Muscular Dystrophy (DMD)
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- 2005
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- Publication type:
- Abstract
258. Identification of Differentially Expressed Genes in Duchenne Muscular Dystropy Utilising RNAi Technology: Possible Targets for Gene Therapy
- Published in:
- 2005
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- Publication type:
- Abstract
G-quadruplex ligands mediate downregulation of DUX4 expression.
- Published in:
- Nucleic Acids Research, 2020, v. 48, n. 8, p. 4179, doi. 10.1093/nar/gkaa146
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- Publication type:
- Article
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.
- Published in:
- Nucleic Acids Research, 2016, v. 44, n. 22, p. 10929, doi. 10.1093/nar/gkw703
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- Publication type:
- Article
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
- Published in:
- Nucleic Acids Research, 2016, v. 44, n. 2, p. 744, doi. 10.1093/nar/gkv1464
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- Publication type:
- Article
Langerhans cells are more efficiently transduced than dermal dendritic cells by adenovirus vectors expressing either group C or group B fibre protein: Implications for mucosal vaccines.
- Published in:
- European Journal of Immunology, 2005, v. 35, n. 9, p. 2617, doi. 10.1002/eji.200425939
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- Publication type:
- Article
Application of Diffraction Gratings to Measurement of Strain of Dental Materials.
- Published in:
- Journal of Dental Research, 1968, v. 47, n. 6, p. 1104, doi. 10.1177/00220345680470061601
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- Publication type:
- Article
Dimensional Changes of Dental Amalgam Associated with Strain Release in the Silver Alloy Particles.
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- Journal of Dental Research, 1965, v. 44, n. 3, p. 620, doi. 10.1177/00220345650440033401
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- Publication type:
- Article
Some Tensile Properties of Amalgam.
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- Journal of Dental Research, 1962, v. 41, n. 4, p. 840, doi. 10.1177/00220345620410041601
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- Publication type:
- Article
SOME FACTORS AFFECTING THE DIMENSIONAL STABILITY OF THE AG-SN-(CU-ZN) AMALGAMS.
- Published in:
- Journal of Dental Research, 1955, v. 34, n. 2, p. 273, doi. 10.1177/00220345550340021601
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- Publication type:
- Article
THE MECHANISM OF HYGROSCOPIC EXPANSION IN DENTAL CASTING INVESTMENTS.
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- Journal of Dental Research, 1955, v. 34, n. 1, p. 44, doi. 10.1177/00220345550340011401
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- Publication type:
- Article
Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression.
- Published in:
- Biomedicines, 2022, v. 10, n. 7, p. N.PAG, doi. 10.3390/biomedicines10071623
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- Publication type:
- Article
How long is full-time?
- Published in:
- 2000
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- Publication type:
- Letter
Liposome-Mediated Gene Transfer into Normal and Dystrophin-Deficient Mouse Myoblasts.
- Published in:
- Journal of Neurochemistry, 1995, v. 64, n. 5, p. 2230, doi. 10.1046/j.1471-4159.1995.64052230.x
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- Publication type:
- Article
Mitochondrial Dysfunction Reveals the Role of mRNA Poly(A) Tail Regulation in Oculopharyngeal Muscular Dystrophy Pathogenesis.
- Published in:
- PLoS Genetics, 2015, v. 11, n. 3, p. 1, doi. 10.1371/journal.pgen.1005092
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- Publication type:
- Article
RAC1B modulates intestinal tumourigenesis via modulation of WNT and EGFR signalling pathways.
- Published in:
- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-22531-3
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- Publication type:
- Article
APOE ɛ3 gene transfer attenuates brain damage after experimental stroke.
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- Journal of Cerebral Blood Flow & Metabolism, 2007, v. 27, n. 3, p. 477, doi. 10.1038/sj.jcbfm.9600361
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- Publication type:
- Article
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog.
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- Journal of Gene Medicine, 2011, v. 13, n. 9, p. 497, doi. 10.1002/jgm.1602
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- Publication type:
- Article
Failure to generate atheroprotective apolipoprotein AI phenotypes using synthetic RNA/DNA oligonucleotides (chimeraplasts).
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- Journal of Gene Medicine, 2003, v. 5, n. 9, p. 795, doi. 10.1002/jgm.403
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- Publication type:
- Article
Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy.
- Published in:
- Molecules, 2012, v. 17, n. 2, p. 1138, doi. 10.3390/molecules17021138
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- Publication type:
- Article
Cytokine genes as potential biomarkers for muscle weakness in OPMD.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. 19, p. 4282, doi. 10.1093/hmg/ddw259
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- Publication type:
- Article
Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. 16, p. 3555, doi. 10.1093/hmg/ddw201
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- Publication type:
- Article
Antisense targeting of 30 end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. 8, p. 1468, doi. 10.1093/hmg/ddw015
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- Publication type:
- Article
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres.
- Published in:
- Human Molecular Genetics, 2010, v. 19, n. 11, p. 2191, doi. 10.1093/hmg/ddq098
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- Publication type:
- Article
RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology.
- Published in:
- Human Molecular Genetics, 2008, v. 17, n. 17, p. 2622, doi. 10.1093/hmg/ddn162
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- Publication type:
- Article
Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice.
- Published in:
- Human Molecular Genetics, 2006, v. 15, n. 14, p. 2266, doi. 10.1093/hmg/ddl151
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- Publication type:
- Article
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy.
- Published in:
- Nature Communications, 2015, v. 6, n. 5, p. 7205, doi. 10.1038/ncomms8205
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- Publication type:
- Article
Fusion of Ubiquitin to HIV Gag Impairs Human Monocyte-Derived Dendritic Cell Maturation and Reduces Ability to Induce Gag T Cell Responses.
- Published in:
- PLoS ONE, 2014, v. 9, n. 2, p. 1, doi. 10.1371/journal.pone.0088327
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- Publication type:
- Article
Fragmentation of SIV-gag Vaccine Induces Broader T Cell Responses.
- Published in:
- PLoS ONE, 2012, v. 7, n. 10, p. 1, doi. 10.1371/journal.pone.0048038
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- Publication type:
- Article
Fit of Porcelain Fused-to-metal Crown and Bridge Castings.
- Published in:
- Journal of Dental Research, 1979, v. 58, n. 12, p. 2360, doi. 10.1177/00220345790580120501
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- Publication type:
- Article
Physical and Chemical Properties and Wear.
- Published in:
- Journal of Dental Research, 1979, v. 58, n. 5, p. 1535, doi. 10.1177/00220345790580051901
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- Publication type:
- Article
Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.
- Published in:
- Journal of Cachexia, Sarcopenia & Muscle, 2020, v. 11, n. 3, p. 768, doi. 10.1002/jcsm.12542
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- Publication type:
- Article
Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD).
- Published in:
- Journal of Cachexia, Sarcopenia & Muscle, 2019, v. 10, n. 5, p. 1016, doi. 10.1002/jcsm.12438
- By:
- Publication type:
- Article
Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle.
- Published in:
- Human Molecular Genetics, 2002, v. 11, n. 15, p. 1719, doi. 10.1093/hmg/11.15.1719
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- Publication type:
- Article
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice.
- Published in:
- Human Molecular Genetics, 2002, v. 11, n. 7, p. 733, doi. 10.1093/hmg/11.7.733
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- Publication type:
- Article
Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides.
- Published in:
- Human Molecular Genetics, 1998, v. 7, n. 7, p. 1083, doi. 10.1093/hmg/7.7.1083
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- Publication type:
- Article
Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy.
- Published in:
- Human Molecular Genetics, 1995, v. 4, n. 8, p. 1245
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- Publication type:
- Article
Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo.
- Published in:
- Human Molecular Genetics, 1993, v. 2, n. 6, p. 717
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- Publication type:
- Article