Qi Long Lu

Summary

Affiliation: Carolinas Medical Center
Country: USA

Publications

  1. doi request reprint Systemic delivery of antisense oligomer in animal models and its implications for treating DMD
    Qi Long Lu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, NC, USA
    Methods Mol Biol 867:393-405. 2012
  2. pmc Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
    Qi Long Lu
    Muscle Cell Biology, Medical Research Council Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London W12 0NN, UK
    Proc Natl Acad Sci U S A 102:198-203. 2005
  3. pmc Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina, United States of America
    PLoS ONE 6:e19906. 2011
  4. pmc One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina 28231, USA
    Mol Ther 19:576-83. 2011
  5. ncbi request reprint Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    Julia Alter
    Muscle Cell Biology, MRC Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK
    Nat Med 12:175-7. 2006
  6. pmc Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo
    Yihong Hu
    Department of Neurology, McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, North Carolina 28231, USA
    Mol Ther 18:812-8. 2010
  7. ncbi request reprint Pluronic block copolymers: novel functions in ultrasound-mediated gene transfer and against cell damage
    Yun Chao Chen
    Ultrasound Group, Imaging Sciences Department, Clinical Sciences Centre, Imperial College, Hammersmith Hospital, London, UK
    Ultrasound Med Biol 32:131-7. 2006
  8. ncbi request reprint Gene transfer with microbubble ultrasound and plasmid DNA into skeletal muscle of mice: comparison between commercially available microbubble contrast agents
    Xinghua Wang
    Imaging Sciences Department, Hammersmith Hospital, Imperial College London, Du Cane Rd, London W12 0NN, England
    Radiology 237:224-9. 2005
  9. pmc Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina, USA
    Am J Pathol 181:392-400. 2012
  10. pmc Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, NC 28231, USA
    Proc Natl Acad Sci U S A 105:14814-9. 2008

Collaborators

Detail Information

Publications20

  1. doi request reprint Systemic delivery of antisense oligomer in animal models and its implications for treating DMD
    Qi Long Lu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, NC, USA
    Methods Mol Biol 867:393-405. 2012
    ..AO therapy will likely critically depend on adequate dosing regimens to achieve therapeutic effect...
  2. pmc Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
    Qi Long Lu
    Muscle Cell Biology, Medical Research Council Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London W12 0NN, UK
    Proc Natl Acad Sci U S A 102:198-203. 2005
    ..We conclude that a significant therapeutic effect may be achieved by further optimization in dose and regime of administration of antisense oligonucleotide...
  3. pmc Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina, United States of America
    PLoS ONE 6:e19906. 2011
    ..A combination of in vitro cell culture and a Vivo-Morpholino based evaluation in vivo systemically in the hDMD mice therefore may represent a prudent approach for selecting AO drug and to meet the regulatory requirement...
  4. pmc One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina 28231, USA
    Mol Ther 19:576-83. 2011
    ..5 g/kg PMO for 6 months. These results indicate that PMO could be used safely as effective drugs for long-term systemic treatment of DMD...
  5. ncbi request reprint Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    Julia Alter
    Muscle Cell Biology, MRC Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK
    Nat Med 12:175-7. 2006
    ..Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD...
  6. pmc Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo
    Yihong Hu
    Department of Neurology, McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, North Carolina 28231, USA
    Mol Ther 18:812-8. 2010
    ..Our results demonstrate that small-molecule compounds could enhance specific exon skipping synergistically with antisense oligomers for experimental therapy to human diseases...
  7. ncbi request reprint Pluronic block copolymers: novel functions in ultrasound-mediated gene transfer and against cell damage
    Yun Chao Chen
    Ultrasound Group, Imaging Sciences Department, Clinical Sciences Centre, Imperial College, Hammersmith Hospital, London, UK
    Ultrasound Med Biol 32:131-7. 2006
    ..L61 decreased cell viability significantly in a dose-dependent manner, whereas P85 showed mild toxicity when its concentration was at or above 0.05%...
  8. ncbi request reprint Gene transfer with microbubble ultrasound and plasmid DNA into skeletal muscle of mice: comparison between commercially available microbubble contrast agents
    Xinghua Wang
    Imaging Sciences Department, Hammersmith Hospital, Imperial College London, Du Cane Rd, London W12 0NN, England
    Radiology 237:224-9. 2005
    ..To compare three commercial microbubble contrast agents (Optison, SonoVue, and Levovist) for their effect on gene delivery in skeletal muscle in conjunction with the use of therapeutic ultrasound...
  9. pmc Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Department of Neurology, Carolinas Medical Center, Charlotte, North Carolina, USA
    Am J Pathol 181:392-400. 2012
    ..Our results demonstrate for the first time that regular 1-year administration of peptide-conjugated phosphorodiamidate morpholino can be safely applied to achieve significant therapeutic effects in an animal model...
  10. pmc Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
    Bo Wu
    McColl Lockwood Laboratory for Muscular Dystrophy, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, NC 28231, USA
    Proc Natl Acad Sci U S A 105:14814-9. 2008
    ..The high degree of potency of the oligomer in targeting all muscles and the lack of detectable toxicity and immune response support the feasibility of testing the novel oligomer in treating Duchenne muscular dystrophy patients...
  11. pmc Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice
    Bo Wu
    Department of Neurology, McColl Lockwood Laboratory for Muscular Dystrophy Laboratory, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, North Carolina 28231, USA
    Mol Ther 17:864-71. 2009
    ....
  12. ncbi request reprint Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration
    Toshifumi Yokota
    Muscle Cell Biology Group, Medical Research Council Clinical Science Centre, Hammersmith Hospital Campus, Imperial College School of Medicine, London University, Du Cane Road, London, W12 0NN, UK
    J Cell Sci 119:2679-87. 2006
    ..This expansion of revertant clusters depicts the cumulative history of regeneration, thus providing a useful index for functional evaluation of therapies that counteract muscle degeneration...
  13. ncbi request reprint Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
    Qi Long Lu
    Muscle Cell Biology, MRC Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK
    Nat Med 9:1009-14. 2003
    ..Our data establishes the realistic practicality of an approach that is applicable, in principle, to a majority of cases of severe dystrophinopathy...
  14. pmc The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy
    Qi Long Lu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, North Carolina, USA
    Mol Ther 19:9-15. 2011
    ..Two different oligonucleotide agents are now being investigated in human trials for splicing out of exon 51 with some early indications of success at the biochemical level...
  15. pmc Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions
    Lei Xu
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Carolinas HealthCare System, Charlotte, North Carolina, USA
    Mol Ther 21:1832-40. 2013
    ..Only limited FKRP transgene expression was detected in kidney and liver with no detectable toxicity. Our results provided evidence for the utility of AAV-mediated gene replacement therapy for FKRP-related muscular dystrophies...
  16. pmc Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice
    Mingxing Wang
    1 Department of Neurology, McColl Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Charlotte, NC 28231
    Hum Gene Ther 25:419-27. 2014
    ..Improved transfection efficiency and lower toxicity indicate the potential of the biodegradable PEA polymers as safe and efficient PMO delivery vectors for in vivo applications...
  17. ncbi request reprint Col1a2 enhancer regulates collagen activity during development and in adult tissue repair
    Markella Ponticos
    Muscle Cell Biology Group, MRC Clinical Science Centre, Imperial College, Hammersmith Hospital Campus, Du Cane Road, London W12 0NN, UK
    Matrix Biol 22:619-28. 2004
    ....
  18. ncbi request reprint Adeno-Associated Virus-Mediated Overexpression of LARGE Rescues α-Dystroglycan Function in Dystrophic Mice with Mutations in the Fukutin-Related Protein
    Charles H Vannoy
    1 McColl Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Carolinas HealthCare System, Charlotte, NC 28231
    Hum Gene Ther Methods 25:187-96. 2014
    ..Our findings suggest that AAV-mediated LARGE gene therapy may still be a viable therapeutic strategy for dystroglycanopathies with FKRP deficiency. ..
  19. ncbi request reprint Optimisation of ultrasound-mediated gene transfer (sonoporation) in skeletal muscle cells
    Hai Dong Liang
    Ultrasound Group, Imaging Sciences Department, Clinical Sciences Centre, Imperial College, Hammersmith Hospital, London W12 0HS, UK
    Ultrasound Med Biol 30:1523-9. 2004
    ..We conclude that low-intensity US irradiation provides a safe and effective alternative for gene delivery...
  20. ncbi request reprint Mouse models of fukutin-related protein mutations show a wide range of disease phenotypes
    Anthony Blaeser
    McColl Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular ALS Center, Carolinas Medical Center, Charlotte, NC 28231, USA
    Hum Genet 132:923-34. 2013
    ..These mutant FKRP mice are useful models for the study of disease mechanism(s) and experimental therapies. ..