HMG Advance Access published May 4, 2011 Human Molecular Genetics, 2011 doi:[removed]hmg ddr174 R1–R8 - Alpha-glucosidase - Document - PDFSEARCH.IO - Document Search Engine

Back to Results

First Page	Meta Content
	HMG Advance Access published May 4, 2011 Human Molecular Genetics, 2011 doi:[removed]hmg/ddr174 R1–R8 Add to Reading List Document Date: 2011-05-07 23:33:11 Open Document File Size: 215,63 KB Share Result on Facebook City Gainesville / Teng / Chen / Mendelsohn / Boston / Baltimore / Bercury / / Company J.M. (2009) Worldwide / A. and Koeberl D.D. / Human Applications Laboratory / C.A.P. D.D. / B.L. and Koeberl D.D. / Florida Powell Gene Therapy Center Vector Core Laboratory / UF Toxicology Core Laboratory / Oxford University Press / Wilson / Donnelly / Fuller D.D. / Y.T. and Koeberl D.D. / IgG / Young S.P. Li / Young S.P. / Winkel L.P. / Van der Beek N.A. / Germain S.A. / Suzuki / Smith S.A. Li / Rosenberg A.S. / / Country United States / / Currency pence / / / Event FDA Phase / / Facility Hospital Boston / University of Florida / Human Applications Laboratory / The Johns Hopkins University / University of Florida Powell Gene Therapy Center Vector Core Laboratory / UF Toxicology Core Laboratory / / IndustryTerm viral vector delivery systems / suppression protocols / invaluable tool / care for the treatment of Pompe disease / therapy for lysosomal storage disease / treatment of Pompe disease / therapy for Pompe disease / injection site / tolerance protocols / cardiac magnetic resonance imaging / treatment of neurological disorders / treatment of the high rate of respiratory failure / therapy for hemophilia / organ systems / gene therapy protocols / transgene product / therapy of brain diseases / / MedicalCondition both infantile and adult human glycogen storage disease / hypertrophic cardiomyopathy / Diffuse Glycogenosis / chronic disability / infantile Pompe disease / neurological disorders / dysfunction / ALS / limb-girdle muscular dystrophy / CRIM-negative infantile Pompe disease / fatal anaphylactic reactions / anaphylactic reactions / illness / severe disease / cretinism / progressive cardiomyopathy / progressive skeletal muscle weakness / glycogenosis type II / inborn lysosomal disorder / hemophilia B. Mol / advanced Pompe disease / IgE-mediated hypersensitivity / genetic disease / respiratory failure / late-onset disease / metabolic myopathy / late-onset Pompe disease / respiratory dysfunction / Infantile-onset glycogen storage disease / glycogenosis / severe hemophilia B. Mol / 6neo/6neo Pompe disease / Glycogen disease resembling mongolism / infantile-onset disease / muscle disorder glycogen storage disease / deficiency / lethal hypersensitivity / muscular dystrophy / GAA deficiency / cardiac and skeletal muscle weakness / strain / early-onset Pompe disease / lysosomal dysfunction / alpha-glucosidase deficiency / Glycogen disease / muscle weakness / cardiomegaly / glycogen storage disease / protein deficiency disorders / lysosomal storage disease / hypersensitivity / allergic/anaphylactic reactions / infantile-onset Pompe disease / Pompe disease / enzyme deficiency / floppy baby syndrome / inherited cardioskeletal myopathy Pompe disease / primary disease / disease / R8 Pompe disease / hypotonia and severe cardiac hypertrophy / autosomal recessive metabolic myopathy / brain diseases / Generalized glycogen storage disease / Pompe’s disease / established disease / murine pompe disease / speech disorders / spinal kyphosis / acid maltase deficiency / muscular dystrophies / respiratory insufficiency / human disease / / MedicalTreatment adjunctive therapies / Surgery / Physical Therapy / adjunctive therapy / relaxation / intravenous infusion / mechanical ventilation / gene therapy / enzyme replacement therapy / / Organization American Society of Gene and Cell Therapy / The Johns Hopkins University / AAV2/ 8-LSP-hGAApA administration / Systemic administration / 2Powell Gene Therapy Center / Oxford University / American Society of Gene Therapy / Direct administration / Targeted administration / Department of Pediatrics / University of Florida / / Person Shelley W. Collins / Darin J. Falk / Stacy L. Porvasnik / Drs Kai Qiu / Cathryn S. Mah / Brian D. Cleaver / David D. Fuller / Barry J. Byrne / Van Doorn / Van den Hout / Paul Reier / Melissa E. Elder / De Klerk / Malik / V / Christina A. Pacak / Van Hove / J. Gene Med / Saleem Islam / Barbara K. Smith / Nina Raben / Roland W. Herzog / Nathalie Clement / Mai K. Elmallah / Anatole Martin / Lee Ann Lawson / Soustek / Thomas J. Conlon / Foley / / / Position D.J. / Author / King / Cao / / Product cyclophosphamide / Myozyme / POMPE PATIENTS WITH / / ProvinceOrState Bali / Y.T. / New Jersey / S.K. / Maryland / South Dakota / Florida / / PublishedMedium PLoS ONE / / SportsEvent ufl / / Technology tolerance protocols / X-ray / Cell Therapy / suppression protocols / stem cells / magnetic resonance imaging / gene expression / alpha / tomography / antibodies / gene therapy / gene therapy protocols / gene delivery / encoding human acid-alpha-glucosidase / / SocialTag Gene delivery Emerging technologies Molecular biology Applied genetics Biotechnology Glycogen storage disease type II Acid alpha-glucosidase Gene therapy Lysosomal storage disease Biology