Louise R Rodino-Klapac

Summary

Affiliation: Columbus Children's Research Institute
Country: USA

Publications

  1. pmc A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus Children s Hospital, 700 Children s Dr, Columbus, Ohio 43205, USA
    J Transl Med 5:45. 2007
  2. pmc Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 68:629-38. 2010
  3. pmc Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery
    Louise R Rodino-Klapac
    Department of Pediatrics, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio 43205, USA
    Mol Ther 18:109-17. 2010
  4. pmc Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
    Janaiah Kota
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    Sci Transl Med 1:6ra15. 2009
  5. doi request reprint AAV-mediated gene therapy to the isolated limb in rhesus macaques
    Louise R Rodino-Klapac
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH, USA
    Methods Mol Biol 709:287-98. 2011
  6. pmc Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
    Paul T Martin
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Department of Pediatrics, The Ohio State Univ College of Medicine, 304 Hamilton Hall, 1645 Neil Ave, Columbus, OH 43210 1218, USA
    Am J Physiol Cell Physiol 296:C476-88. 2009
  7. doi request reprint Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
    Vinod Malik
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Ohio State University, Columbus, OH 43205, USA
    Ann Neurol 67:771-80. 2010
  8. doi request reprint Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 66:290-7. 2009
  9. pmc mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice
    Nicholas Beastrom
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA
    Am J Pathol 179:2464-74. 2011
  10. ncbi request reprint Gene therapy for duchenne muscular dystrophy: expectations and challenges
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus, OH, USA
    Arch Neurol 64:1236-41. 2007

Collaborators

  • Jerry R Mendell
  • K Reed Clark
  • Zarife Sahenk
  • Louis G Chicoine
  • Kevin M Flanigan
  • Lei Chen
  • Majed Dasouki
  • Lan Zhou
  • PAULUS JANSSEN
  • Vinod Malik
  • LOUISE RODINO
  • Janaiah Kota
  • Chrystal L Montgomery
  • Xiomara Q Rosales
  • Kristin N Heller
  • Sarah Lewis
  • Nicholas Beastrom
  • Brian K Kaspar
  • Laurence Viollet
  • Christopher M Walker
  • Christopher J Shilling
  • Paul T Martin
  • Julie M Gastier-Foster
  • Caroline Astbury
  • Shalini Reshmi
  • Rob Pyatt
  • Amita Sneh
  • Eric K Johnson
  • Haiyan Lu
  • Benjamin D Canan
  • Allison Macke
  • Federica Montanaro
  • Christopher M Penton
  • Roula Al-Dahhak
  • Ricardo Bien-Willner
  • John D Mahan
  • Brenda Banwell
  • Richard J Barohn
  • Carmen Serrano-Munuera
  • Kumaraswamy Sivakumar
  • Wendy King
  • Cheryl Wall
  • Victoria Watts
  • John Hayes
  • Katherine J Campbell
  • Amy Eagle
  • Danielle Tucker
  • Elaine Oglesbay
  • Steven E Weisbrode
  • Rui Xu
  • Chalonda R Handy
  • Marybeth Camboni
  • Amanda M Haidet
  • Kim Shontz
  • Walter R Therlfall

Detail Information

Publications17

  1. pmc A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus Children s Hospital, 700 Children s Dr, Columbus, Ohio 43205, USA
    J Transl Med 5:45. 2007
    ....
  2. pmc Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 68:629-38. 2010
    ....
  3. pmc Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery
    Louise R Rodino-Klapac
    Department of Pediatrics, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio 43205, USA
    Mol Ther 18:109-17. 2010
    ..In summary, an epitope-tagged micro-dystrophin cassette enhances the ability to evaluate site-specific localization and distribution of gene expression in the NHP in preparation for vascular delivery clinical trials...
  4. pmc Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
    Janaiah Kota
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    Sci Transl Med 1:6ra15. 2009
    ..Our results, together with the findings in mice, suggest that therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders...
  5. doi request reprint AAV-mediated gene therapy to the isolated limb in rhesus macaques
    Louise R Rodino-Klapac
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH, USA
    Methods Mol Biol 709:287-98. 2011
    ..We also provide methods for assessing transduction efficiency of microdystrophin.FLAG following the IFLP vascular delivery protocol...
  6. pmc Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
    Paul T Martin
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Department of Pediatrics, The Ohio State Univ College of Medicine, 304 Hamilton Hall, 1645 Neil Ave, Columbus, OH 43210 1218, USA
    Am J Physiol Cell Physiol 296:C476-88. 2009
    ..That overexpression also prevents loss of force in nondystrophic muscles suggests that Galgt2 is a therapeutic target with broad potential applications...
  7. doi request reprint Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
    Vinod Malik
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Ohio State University, Columbus, OH 43205, USA
    Ann Neurol 67:771-80. 2010
    ..Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD)...
  8. doi request reprint Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 66:290-7. 2009
    ..Gene replacement represents a strategy for correcting the underlying defect. Questions related to this approach were addressed in this clinical trial, particularly the need for immunotherapy and persistence of gene expression...
  9. pmc mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice
    Nicholas Beastrom
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA
    Am J Pathol 179:2464-74. 2011
    ....
  10. ncbi request reprint Gene therapy for duchenne muscular dystrophy: expectations and challenges
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus, OH, USA
    Arch Neurol 64:1236-41. 2007
    ..This article highlights the challenges and potential pitfalls as the field advances this treatment modality to clinical reality...
  11. pmc AAV-mediated Overexpression of Human α7 Integrin Leads to Histological and Functional Improvement in Dystrophic Mice
    Kristin N Heller
    1 Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA 2 Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA
    Mol Ther 21:520-5. 2013
    ..This therapeutic approach demonstrates promise as a viable treatment for DMD with further implications for other forms of muscular dystrophy...
  12. doi request reprint Update on the treatment of Duchenne muscular dystrophy
    Louise R Rodino-Klapac
    Department of Pediatrics, The Ohio State University, and Nationwide Children s Hospital, Columbus, OH 43210, USA
    Curr Neurol Neurosci Rep 13:332. 2013
    ..The advantages of each approach and challenges in translation are outlined in detail. Individually or in combination, all of these therapeutic strategies hold great promise for treatment of this devastating childhood disease...
  13. pmc Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation
    Xiomara Q Rosales
    Neuromuscular Center at The Research Institute at Nationwide Children s Hospital, Columbus, Ohio Department of Pediatrics and Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio, 43205 The Ohio State University, Columbus, Ohio
    Muscle Nerve 47:731-9. 2013
    ..This results in impaired regeneration and fibrosis. Muscle Nerve 47: 731-739, 2013...
  14. pmc Molecular therapeutic strategies targeting Duchenne muscular dystrophy
    Jerry R Mendell
    Center for Gene Therapy, Nationwide Children s Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA
    J Child Neurol 25:1145-8. 2010
    ..The results are modest and encumbered by side effects. The authors review 3 molecular therapeutic approaches that have been introduced into the clinic: (1) gene replacement therapy, (2) mutation suppression, and (3) exon skipping...
  15. pmc Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease
    Louise R Rodino-Klapac
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio 43205 USA
    Muscle Nerve 39:283-96. 2009
    ..These findings provide the impetus to move toward gene therapy clinical trials with delivery of AAV-FS344 to increase size and function of muscle in patients with neuromuscular disease...
  16. pmc Emerging drugs for Duchenne muscular dystrophy
    Vinod Malik
    The Ohio State University, Research Institute, Nationwide Children s Hospital and, Department of Pediatrics, Columbus, OH 43205, USA
    Expert Opin Emerg Drugs 17:261-77. 2012
    ..The review emphasizes that the goal of treatment should be to find a product at least as good as glucocorticoids with a lower side effect profile or with a significant glucocorticoid sparing effect...
  17. pmc Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy
    Vinod Malik
    The Research Institute at Nationwide Children s Hospital and Department of Pediatrics at The Ohio State University College of Medicine, Columbus, OH, USA
    Ther Adv Neurol Disord 3:379-89. 2010
    ..Here we review nonsense mutation suppression by aminoglycosides as a therapeutic strategy to treat DMD with special emphasis on gentamicin-induced readthrough of disease-causing premature termination codons...

Research Grants2

  1. Dystrophin restoration in two animal models of Duchenne Muscular Dystrophy
    LOUISE RODINO; Fiscal Year: 2007
    ..Finally, the optimal AAV serotype carrying micro-dystrophin in the mouse will be perfused into the femoral artery of the golden retriever muscular dystrophy (GRMD) dog (Aim 3). ..