Aurelie Goyenvalle

Summary

Affiliation: University of Oxford
Country: UK

Publications

  1. pmc Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Mol Ther 20:1212-21. 2012
  2. ncbi Splicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophies
    Rachid Benchaouir
    Equipe Biotherapies des Maladies Neuromusculaires, Unite Mixte Um76 UPMC UMR 7215 CNRS U974 Inserm Institut de Myologie, Faculte de Medecine Pierre et Marie Curie, 105 Boulevard de l Hôpital, 75013 Paris, France
    Curr Gene Ther 12:179-91. 2012
  3. pmc Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Hum Mol Genet 21:2559-71. 2012
  4. doi Engineering U7snRNA gene to reframe transcripts
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Methods Mol Biol 867:259-71. 2012
  5. pmc Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Skelet Muscle 1:8. 2011
  6. doi Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy, and Genetics, University of Oxford, South Parks Road, Oxford, UK
    Methods Mol Biol 709:179-96. 2011
  7. pmc Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Mol Ther 18:198-205. 2010
  8. pmc Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy
    Aurelie Goyenvalle
    Department of Physiology, Anatomy and Genetics, University of Oxford, UK
    Mol Ther 17:1234-40. 2009
  9. pmc Therapeutic approaches to muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Hum Mol Genet 20:R69-78. 2011

Detail Information

Publications9

  1. pmc Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Mol Ther 20:1212-21. 2012
    ....
  2. ncbi Splicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophies
    Rachid Benchaouir
    Equipe Biotherapies des Maladies Neuromusculaires, Unite Mixte Um76 UPMC UMR 7215 CNRS U974 Inserm Institut de Myologie, Faculte de Medecine Pierre et Marie Curie, 105 Boulevard de l Hôpital, 75013 Paris, France
    Curr Gene Ther 12:179-91. 2012
    ....
  3. pmc Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Hum Mol Genet 21:2559-71. 2012
    ..These findings suggest great potential for AAV-U7 in systemic treatment of the DMD phenotype...
  4. doi Engineering U7snRNA gene to reframe transcripts
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Methods Mol Biol 867:259-71. 2012
    ..Here, we outline the design of these U7snRNA constructs to achieve efficient exon-skipping and describe methods to evaluate the efficacy of such U7snRNA constructs in vitro using the dystrophin gene as an example...
  5. pmc Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Skelet Muscle 1:8. 2011
    ....
  6. doi Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy, and Genetics, University of Oxford, South Parks Road, Oxford, UK
    Methods Mol Biol 709:179-96. 2011
    ..We also describe methods to evaluate the efficiency of such U7 snRNA constructs in vitro in DMD patient cells and in vivo in the transgenic hDMD mouse model, using lentiviral and recombinant adeno-associated viral vectors, respectively...
  7. pmc Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Mol Ther 18:198-205. 2010
    ..These findings suggest great potential for PPMOs in systemic treatment of the DMD phenotype...
  8. pmc Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy
    Aurelie Goyenvalle
    Department of Physiology, Anatomy and Genetics, University of Oxford, UK
    Mol Ther 17:1234-40. 2009
    ..These new constructs are very promising for the optimization of therapeutic exon skipping for DMD, but also offer powerful and versatile tools to modulate pre-mRNA splicing in a wide range of applications...
  9. pmc Therapeutic approaches to muscular dystrophy
    Aurelie Goyenvalle
    MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK
    Hum Mol Genet 20:R69-78. 2011
    ..Here, we summarize the recent progress in therapeutic approaches to muscular dystrophies, with an emphasis on gene therapy and exon skipping for DMD...