academic article

• Acid Alpha-Glucosidase Gene Replacement Therapy To the Diaphragm Remodels Ventilatory Function in Severe Pompe Disease.  The FASEB's Journal.  28. 2014-04-01
• Adeno-Associated Virus-Mediated Gene Therapy for Metabolic Myopathy.  Human Gene Therapy.  24:928-936. 2013-11-01
• Intrapleural Administration of Aav9 Improves Neural and Cardiorespiratory Function in Pompe Disease.  Molecular Therapy.  21:1661-1667. 2013-09-01
• Acid Alpha-Glucosidase Gene Replacement Therapy To the Diaphragm in Ventilator-Dependent Pompe Disease: One-Year Respiratory Motor Outcomes.  Molecular Therapy.  21:S23-S24. 2013-06-01
• Phase I/II Trial of Adeno-Associated Virus-Mediated Alpha-Glucosidase Gene Therapy To the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes.  Human Gene Therapy.  24:630-640. 2013-06-01
• One Year Outcomes of Diaphragm Gene Therapy in Ventilator Dependent Children With Pompe Disease.  Molecular Genetics and Metabolism.  108:S27. 2013-02-01
• Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment.  Human Gene Therapy.  23:808-815. 2012-08-01
• Identification of Adeno-Associated Viral Vectors Suitable for Intestinal Gene Delivery and Modulation of Experimental Colitis.  American Journal of Physiology-Gastrointestinal and Liver Physiology.  302:G296-G308. 2012-02-01
• Phase I/Ii Trial of Adeno-Associated Virus Acid-Alpha-Glucosidase (Aav-Gaa) Diaphragm Gene Therapy for Ventilatory Failure in Pompe Disease.  Molecular Genetics and Metabolism.  105:S24. 2012-02-01
• Induction of Tolerance To Factor Viii By Transient Co-Administration With Rapamycin.  Journal of Thrombosis and Haemostasis.  9:1524-1533. 2011-08-01
• Characterization of a Transgenic Short Hairpin Rna-Induced Murine Model of Tafazzin Deficiency.  Human Gene Therapy.  22:865-871. 2011-07-01
• Raav1 Vector-Mediated Expression of Tafazzin in Patient Fibroblasts and Murine Model of Barth Syndrome.  Molecular Therapy.  19:901. 2011-07-01
• Clinical Manifestations of Heterozygous Expression of a Mutation That Causes Glycogen Storage Disease Type Ia in Dogs.  Journal of Veterinary Internal Medicine.  25:731-732. 2011-05-01
• Pompe Disease Gene Therapy.  Human Molecular Genetics.  20:R61-R68. 2011-04-01
• Next Generation Treatment of Pompe Disease Using Systemic Gene Transfer With Aav9.  Molecular Genetics and Metabolism.  102:S18. 2011-02-01
• Glycogen Storage Disease Type Ia in Canines: a Model for Human Metabolic and Genetic Liver Disease.  Journal of Biomedicine & Biotechnology.  2011-01-01
• Targeting Murine Small Bowel and Colon Through Selective Superior Mesenteric Artery Injection.  Microsurgery.  30:487-493. 2010-09-01
• Adeno-Associated Virus-Mediated Correction of a Canine Model of Glycogen Storage Disease Type Ia.  Human Gene Therapy.  21:903-910. 2010-07-01
• Gel-Mediated Delivery of Aav1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease.  Molecular Therapy.  18:502-510. 2010-03-01
• Gel-Mediated Delivery of Aav1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease.  Molecular Therapy.  18:502-510. 2010-03-01
• Indoleamine 2,3-Dioxygenase Attenuates Inhibitor Development in Gene-Therapy-Treated Hemophilia a Mice.  Gene Therapy.  16:724-733. 2009-06-01
• Neural Deficits Contribute To Respiratory Insufficiency in Pompe Disease.  Proceedings of the National Academy of Sciences.  106:9419-9424. 2009-06-01
• Administration of Raav2/8 and Raav2/1 Vectors Mediates Sustained Correction of a Canine Model of Glycogen Storage Disease Type Ia.  Molecular Therapy.  17:168. 2009-05-01
• Gastrointestinal Directed Adeno-Associated Virus Serotype 9 and 10 Expressing Il-10 Improves Enterocolitis in a Murine Model of Inflammatory Bowel Disease.  Molecular Therapy.  17:S314. 2009-05-01
• Rapid Raav-Mediated Improvement of Respiratory Function in An Animal Model of Pompe Disease.  Molecular Therapy.  17:S342. 2009-05-01
• Alterations in Mucosal Cytokine Levels Reflect Acute Inflammation But Not Chronic Inflammation in the Murine Dss Model of Colitis.  Inflammatory Bowel Diseases.  14:S15. 2008-12-01
• Tyrosine-Phosphorylation of Aav2 Vectors and Its Consequences On Viral Intracellular Trafficking and Transgene Expression.  Virology.  381:194-202. 2008-11-01
• Next Generation of Adeno-Associated Virus 2 Vectors: Point Mutations in Tyrosines Lead To High-Efficiency Transduction At Lower Doses.  Proceedings of the National Academy of Sciences.  105:7827-7832. 2008-06-01
• Gene Delivery To Intestinal Epithelial Cells in Vitro and in Vivo With Recombinant Adeno-Associated Virus Types 1, 2 and 5.  Digestive Diseases and Sciences.  53:1261-1270. 2008-05-01
• Gene Delivery To Intestinal Epithelial Cells in Vitro and in Vivo With Recombinant Adeno-Associated Virus Types 1, 2 and 5.  Digestive Diseases and Sciences.  53:1261-1270. 2008-05-01
• Identification of New Aav Vector Pseudotypes Suitable for Small Intestine and Colon Gene Transfer.  Inflammatory Bowel Diseases.  14:S5-S6. 2008-01-01
• Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors..  Molecular Therapy.  15. 2007
• Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II..  Human Gene Therapy.  16. 2005
• A new method for recombinant adeno-associated virus vector delivery to murine diaphragm..  Molecular Therapy.  9. 2004