Carmen Bertoni

Summary

Affiliation: University of California
Country: USA

Publications

  1. ncbi Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides
    Carmen Bertoni
    Department of Neurology, University of California, Los Angeles, 611 Charles Young Drive East, Boyer Hall, 508, Los Angeles, CA 90095 1570, USA
    Front Biosci 13:517-27. 2008
  2. ncbi Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305, USA
    Nucleic Acids Res 37:7468-82. 2009
  3. ncbi Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs
    Refik Kayali
    Department of Neurology, David Geffen School of Medicine, University of California at Los Angeles, CA 90095, USA
    Hum Mol Genet 19:3266-81. 2010
  4. ncbi Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305 5235, USA
    Hum Mol Genet 12:1087-99. 2003
  5. ncbi Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration
    Carmen Bertoni
    Department of Neurology, Stanford University School of Medicine, Stanford, CA 94305-5235, USA
    Proc Natl Acad Sci U S A 103:419-24. 2006
  6. ncbi Strand bias in oligonucleotide-mediated dystrophin gene editing
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305-5235, USA
    Hum Mol Genet 14:221-33. 2005
  7. ncbi Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery
    Joylette L Portlock
    Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA
    Hum Gene Ther 17:871-6. 2006
  8. ncbi Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum
    Liutao Du
    Department of Pathology and Laboratory Medicine, The David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1732, USA
    Hum Mol Genet 20:3151-60. 2011
  9. ncbi Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy
    Refik Kayali
    Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA
    Hum Mol Genet 21:4007-20. 2012
  10. ncbi Nonaminoglycoside compounds induce readthrough of nonsense mutations
    Liutao Du
    Department of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA
    J Exp Med 206:2285-97. 2009

Collaborators

Detail Information

Publications11

  1. ncbi Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides
    Carmen Bertoni
    Department of Neurology, University of California, Los Angeles, 611 Charles Young Drive East, Boyer Hall, 508, Los Angeles, CA 90095 1570, USA
    Front Biosci 13:517-27. 2008
    ..Ultimately the need for production of oligonucleotides in large scale and the cost of treatment for each individual patient will play a big role in the feasibility of these approaches in DMD...
  2. ncbi Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305, USA
    Nucleic Acids Res 37:7468-82. 2009
    ....
  3. ncbi Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs
    Refik Kayali
    Department of Neurology, David Geffen School of Medicine, University of California at Los Angeles, CA 90095, USA
    Hum Mol Genet 19:3266-81. 2010
    ..The use of PNA-ssODNs has important implications in terms of both efficacy and duration of the repair process in muscles and may have a role in advancing the treatment of DMD...
  4. ncbi Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305 5235, USA
    Hum Mol Genet 12:1087-99. 2003
    ..As such, chimeraplast-mediated exon skipping has the potential to be used to transform a severe DMD phenotype into a much milder BMD phenotype...
  5. ncbi Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration
    Carmen Bertoni
    Department of Neurology, Stanford University School of Medicine, Stanford, CA 94305-5235, USA
    Proc Natl Acad Sci U S A 103:419-24. 2006
    ..These data demonstrate the importance of both the level and distribution of dystrophin expression to achieve therapeutic efficacy, and that the efficacy can be enhanced by targeted plasmid integration...
  6. ncbi Strand bias in oligonucleotide-mediated dystrophin gene editing
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305-5235, USA
    Hum Mol Genet 14:221-33. 2005
    ....
  7. ncbi Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery
    Joylette L Portlock
    Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA
    Hum Gene Ther 17:871-6. 2006
    ..These results suggest the possible utility of the phiC31 integrase system to treat ischemic disease...
  8. ncbi Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum
    Liutao Du
    Department of Pathology and Laboratory Medicine, The David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1732, USA
    Hum Mol Genet 20:3151-60. 2011
    ..Taken together, these results highlight the therapeutic potential of (RXRRBR)(2)XB-AMOs in A-T and other neurogenetic disorders...
  9. ncbi Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy
    Refik Kayali
    Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA
    Hum Mol Genet 21:4007-20. 2012
    ..These studies establish the therapeutic potential of RTC13 in vivo and advance this newly identified compound into preclinical application for DMD...
  10. ncbi Nonaminoglycoside compounds induce readthrough of nonsense mutations
    Liutao Du
    Department of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA
    J Exp Med 206:2285-97. 2009
    ..The two compounds also demonstrated readthrough activity in mdx mouse myotube cells carrying a nonsense mutation and induced significant amounts of dystrophin protein...
  11. ncbi Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides
    Carmen Bertoni
    Department of Neurology and Neurological Sciences, Medical Center, Stanford University School of Medicine, Stanford, CA 94305, USA
    Hum Gene Ther 13:707-18. 2002
    ..These results, taken together, further demonstrate that chimeraplast-mediated gene repair may be effective as an approach to gene therapy for muscular dystrophies due to point mutations...