Ohio State - Nationwide Children's Hospital - Research For Muscle Biology and Disease
 

Louise R. Rodino-Klapac, Ph.D.Louise R. Rodino-Klapac, Ph.D.

Associate Professor

The Ohio State University School of Medicine
Department of Pediatrics and Center for Gene Therapy
Nationwide Children's Research Institute
700 Children's Drive, Rm. WA3015
Columbus, OH  43205

Phone: (614) 355-2893
Email: Louise.Rodino-Klapac@nationwidechildrens.org

Education & Training:
King’s College, Wilkes-Barre, PA, 2000 BS in Biology
The Ohio State University 2005, Ph.D. in Molecular Genetics
The Research Institute at Nationwide Children’s Hospital, 2010, Postdoctoral Fellow

Research Interest:
My laboratory is focused on developing gene therapy based vectors for the treatment of neuromuscular disorders. With emphasis on translating laboratory benchside research to the bedside, we are interested in addressing key issues such as transgene delivery, efficacy of gene expression, and immunogenicity; which all have the potential to affect clinical outcomes. One of our goals is to deliver recombinant adeno-associated virus (rAAV) mediated vectors by a vascular route to target multiple muscle groups. One such vector is micro-dystrophin as a potential treatment for Duchenne muscular dystrophy (DMD). Gene therapy based therapeutics for other forms of muscular dystrophy, Limb girdle muscular dystrophy type 2B [dysferlin (DYSF) deficiency] and the related type 2L (Anoctamin 5 (AN05) deficiency] are also a key initiative in my lab. Development of a gene delivery vector for DYSF deficiency using a novel approach with AAV5 is currently underway and future studies will focus on translating these pre-clinical studies to the clinic. We are testing the efficacy of gene transfer using a membrane repair assay in muscle isolated from animals treated with AAV5.DYSF. Pre-Clinical studies for AN05 are well underway including characterization of AN05 function and understanding loss of function mechanisms in both zebrafish and mouse models.

Selected Publications:

  • Pozsgai, ER, Griffin, DA, Heller, KN, Mendell, JR and Rodino-Klapac, LR. (2017). Systemic AAV- Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Molecular Therapy. 25: PMID: 28284983
  • Pozsgai, ER, Griffin, DA, Heller, KN, Mendell, JR and Rodino-Klapac, LR. (2016). β-Sarcoglycan Gene Transfer Decreases Fibrosis and Restores Force in LGMD2E Mice. Gene Therapy. 23: 57-66. PMID: 26214262
  • Griffin, DA, Johnson, RW, Whitlock, JM, Pozsgai, ER, Heller, KH, Grose, WE, Arnold, WD, Sahenk, Z; Hartzell HC and Rodino-Klapac, LR. (2016). Defective membrane fusion and repair in anoctamin5- deficient muscular dystrophy. Human Molecular Genetics. Epub PMID: 26911675
  • Mendell, JR, Sahenk, Z, Malik, V, Gomez, AM, Flanigan, KM, Lowes, LP, Alfano, LN, Berry, K, Meadows, E, Lewis, S, Braun, L, Shontz, K, Rouhana, M, Clark, KR, Quintero-Rosales, X, Al-Zaidy, S, Govoni, A, Rodino-Klapac, LR, Hogan, MJ, and Kaspar, BK. (2015) A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy. Molecular Therapy. 23: 192-201 PMID: 25322757
  • Sondergaard, PC, Griffin, DA, Pozsgai, ER, Johnson, RW, Grose, WE, Heller, KN, Shontz, KM, Montgomery, CL, Liu, J, Clark, KR, Sahenk, Z, Mendell, JR, and Rodino-Klapac, LR. (2015) Dysferlin Restoration using Overlapping AAV Vectors Restores Function in Animal Models of Dysferlinopathies. Annals of Clinical and Translational Neurology. 2: 256-270. PMID: 25815352
  • Heller, KN, Montgomery, CL, Shontz, KM, Janssen, PML, Clark, KR, Mendell, JR, Rodino-Klapac, LR. (2015). Human α7 integrin gene (ITGΑ7) delivered by adeno-associated virus reverses the phenotype of the double knock out (MDX/UTRN -\-) mouse devoid of dystrophin and utrophin. Human Gene Therapy. 10: 647-656. PMID: 26076707
  • Sondergaard, PC, Griffin, DA, Pozsgai, ER, Johnson, RW, Grose, WE, Heller, KN, Shontz, KM, Montgomery, CL, Liu, J, Clark, KR, Sahenk, Z, Mendell, JR, and Rodino-Klapac, LR. (2015) Dysferlin Restoration using Overlapping AAV Vectors Restores Function in Animal Models of Dysferlinopathies. Annals of Clinical and Translational Neurology. 2: 256-270. PMID: 25815352
  • Heller, KN, Montgomery, CL, Shontz, KM, Janssen, PML, Clark, KR, Mendell, JR, Rodino-Klapac, LR. (2015). Human α7 integrin gene (ITGΑ7) delivered by adeno-associated virus reverses the phenotype of the double knock out (MDX/UTRN -\-) mouse devoid of dystrophin and utrophin. Human Gene Therapy. In Press. PMID: 26076707
  • Chicoine, LG, Montgomery, CL, Bremer, WG, Shontz, KM, Griffin, DA, Heller, KN, Lewis, S, Malik, V, Shilling, CJ, Campbell, KJ, Preston, TJ, Coley, BD, Martin, PT, Walker, CM, Clark, KR, Sahenk, Z, Mendell, JR, and Rodino-Klapac, LR. (2014). Plasmapheresis eliminates the negative impact of AAV antibodies on micro-dystrophin gene expression following vascular delivery. Molecular Therapy. 22:338-347. PMID: 24196577
  • Heller, KN, Montgomery, CL, Janssen, PML, Clark, KR, Mendell, JR, Rodino-Klapac, LR. (2013) AAV mediated overexpression of Human α7 Integrin leads to histological and functional improvement in dystrophic mice. Molecular Therapy. 21:520-525. PMID: 23319059

My NCBI Link: http://www.ncbi.nlm.nih.gov/sites/myncbi/louise.rodino- klapac.1/bibliography/48568735/public/?sort=date&direction=ascending