Hum Gene Ther

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  Publication Venue For

  • Ex Vivo Gene Delivery to Porcine Cardiac Allografts Using a Myocardial-Enhanced Adeno-Associated Viral Vector..  34:303-313. 2023
  • Letter to the Editor: mRNA Vaccines in Urological Malignancies..  33:1101-1102. 2022
  • Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease..  33:479-491. 2022
  • Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease..  33:492-498. 2022
  • Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood..  32:405-419. 2021
  • Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development..  32:31-42. 2021
  • Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy..  31:839-851. 2020
  • Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis..  31:828-838. 2020
  • Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease..  30:855-864. 2019
  • Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease..  30:57-68. 2019
  • Gene Therapy for Modulation of T-Cell-Mediated Immune Response Provoked by Corneal Transplantation..  29:467-479. 2018
  • Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector..  28:385-391. 2017
  • Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy..  28:286-294. 2017
  • Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates..  27:19-31. 2016
  • Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease..  26:743-750. 2015
  • High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline..  26:614-621. 2015
  • Responsible Use of Human Gene-Editing Technologies..  26:411-412. 2015
  • Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade..  26:26-35. 2015
  • Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013..  25:488-497. 2014
  • An emerging adeno-associated viral vector pipeline for cardiac gene therapy..  24:906-913. 2013
  • Adeno-associated viral vectors based on serotype 3b use components of the fibroblast growth factor receptor signaling complex for efficient transduction..  23:1031-1042. 2012
  • Recognition of glioma stem cells by genetically modified T cells targeting EGFRvIII and development of adoptive cell therapy for glioma..  23:1043-1053. 2012
  • X-linked inhibitor of apoptosis protein-mediated attenuation of apoptosis, using a novel cardiac-enhanced adeno-associated viral vector..  23:635-646. 2012
  • Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease..  23:460-472. 2012
  • Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia..  23:407-418. 2012
  • A novel conditionally replicating "armed" adenovirus selectively targeting gastrointestinal tumors with aberrant wnt signaling..  22:427-437. 2011
  • Genetic engineering of mesenchymal stem cells and its application in human disease therapy..  21:1513-1526. 2010
  • Transduction of human antigen-presenting cells with integrase-defective lentiviral vector enables functional expansion of primed antigen-specific CD8(+) T cells..  21:1029-1035. 2010
  • Enhanced pancreatic cancer gene therapy by combination of adenoviral vector expressing c-erb-B2 (Her-2/neu)-targeted immunotoxin with a replication-competent adenovirus or etoposide..  21:157-170. 2010
  • Aptamers in immunotherapy..  19:443-450. 2008
  • Selective modification of antigen-specific T cells by RNA electroporation..  19:511-521. 2008