Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

Autor: Chicoine, LG ^**, Montgomery, CL, Bremer, WG, Shontz, KM, Griffin, DA, Heller, KN, Lewis, S, Malik, V, Grose, WE, Shilling, CJ, Campbell, KJ, Preston, TJ, Coley, BD, Martin, PT, Walker, CM, Clark, KR, Sahenk, Z, Mendell, JR ^***, Rodino-Klapac, LR ^*

Publikováno v: In Molecular Therapy February 2014 22(2):338-347

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A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.

Autor: Meadows AS; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Duncan FJ; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Camboni M; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Waligura K; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Montgomery C; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Zaraspe K; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Naughton BJ; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Bremer WG; 2 Center for Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Shilling C; 3 Drug and Device Development Service, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio., Walker CM; 2 Center for Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 4 Department of Pediatrics, College of Medicine and Public Health, Columbus, Ohio., Bolon B; 5 Comparative Pathology and Mouse Phenotyping Shared Resource, Comprehensive Cancer Center, The Ohio State University, Columbus, Ohio., Flanigan KM; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 4 Department of Pediatrics, College of Medicine and Public Health, Columbus, Ohio., McBride KL; 4 Department of Pediatrics, College of Medicine and Public Health, Columbus, Ohio.; 6 Center for Cardiovascular and Pulmonary Research, Columbus, Ohio., McCarty DM; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 4 Department of Pediatrics, College of Medicine and Public Health, Columbus, Ohio., Fu H; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 3 Drug and Device Development Service, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.

Publikováno v: Human gene therapy. Clinical development [Hum Gene Ther Clin Dev] 2015 Dec; Vol. 26 (4), pp. 228-42.

Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.

Autor: Chicoine LG; 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., Rodino-Klapac LR; 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., Shao G; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Xu R; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Bremer WG; Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Camboni M; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Golden B; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Montgomery CL; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Shontz K; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Heller KN; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Griffin DA; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Lewis S; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Coley BD; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Current address: Department of Radiology, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA., Walker CM; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Clark KR; 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., Sahenk Z; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Neurology, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [3] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Mendell JR; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Neurology, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [3] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Martin PT; 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.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2014 Apr; Vol. 22 (4), pp. 713-24. Date of Electronic Publication: 2013 Oct 22.

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.

Autor: Rodino-Klapac LR; Department of Pediatrics, The Ohio State University/Nationwide Children's Hospital, Columbus, Ohio 43205, USA., Montgomery CL, Bremer WG, Shontz KM, Malik V, Davis N, Sprinkle S, Campbell KJ, Sahenk Z, Clark KR, Walker CM, Mendell JR, Chicoine LG

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2010 Jan; Vol. 18 (1), pp. 109-17. Date of Electronic Publication: 2009 Nov 10.