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 AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice
    Kristin N Heller
    The Ohio State University, Columbus, OH, USA
    Mol Ther 21:520-5. 2013
  7. 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
  8. 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
  9. ncbi 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
  10. ncbi 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

Collaborators

Detail Information

Publications20

  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 AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice
    Kristin N Heller
    The Ohio State University, Columbus, OH, 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...
  7. 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...
  8. 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...
  9. ncbi 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...
  10. ncbi 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)...
  11. pmc Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates
    Louis G Chicoine
    1 Department of Pediatrics, The Ohio State University and Nationwide Children s Hospital, Columbus, Ohio, USA 2 Centers for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA
    Mol Ther 22:713-24. 2014
    ..These experiments demonstrate successful transduction of rhesus macaque muscle with rAAVrh74.MCK.GALGT2 after vascular delivery and induction of molecular changes thought to be therapeutic in several forms of muscular dystrophy. ..
  12. pmc AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy
    Zarife Sahenk
    1 Department of Pediatrics, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio, USA 2 Center for Gene Therapy at The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA 3 Department of Neurology, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio, USA 4 Department of Pathology, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio, USA
    Mol Ther 22:511-21. 2014
    ..These studies of intramuscular (i.m.) delivery of rAAV1.NT-3 serve as a template for future CMT1A clinical trials with a potential to extend treatment to other nerve diseases with impaired nerve regeneration. ..
  13. ncbi request reprint Eteplirsen for the treatment of Duchenne muscular dystrophy
    Jerry R Mendell
    Departments of Pediatrics, Ohio State University, Columbus, OH Neurology the Ohio State University, Ohio State University, Columbus, OH Gene Therapy Center, Nationwide Children s Hospital Ohio State University, Columbus, OH Paul D Wellstone Center, Nationwide Children s Hospital Ohio State University, Columbus, OH
    Ann Neurol 74:637-47. 2013
    ..The present study used a double-blind placebo-controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6-minute walk test (6MWT)...
  14. 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
    ....
  15. ncbi 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...
  16. 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, USA
    Muscle Nerve 47:731-9. 2013
    ..In normal muscle, up-regulation of miR-1 and miR-206 facilitates transition from proliferating SCs to differentiating myogenic progenitors...
  17. 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...
  18. 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
    ..Finding a more satisfactory treatment should focus on maintaining long-term efficacy with a minimal side effect profile...
  19. 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...
  20. 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...

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). ..