Human Gene Therapy

Title Publication Date Language Citations
GDNF Delivery Using Human Neural Progenitor Cells in a Rat Model of ALS2005/04/01English174
Thymidine Kinase Gene Therapy for Human Malignant Glioma, Using Replication-Deficient Retroviruses or Adenoviruses2000/11/01English174
A Phase II, Double-Blind, Randomized, Placebo-Controlled Clinical Trial of tgAAVCF Using Maxillary Sinus Delivery in Patients with Cystic Fibrosis with Antrostomies2002/07/20English173
The Inhibitory Role of CpG Immunostimulatory Motifs in Cationic Lipid Vector-Mediated Transgene Expression in Vivo1999/09/01English172
Dynein- and Microtubule-Mediated Translocation of Adenovirus Serotype 5 Occurs after Endosomal Lysis2000/01/01English171
Efficient and Stable Adeno-Associated Virus-Mediated Transduction in the Skeletal Muscle of Adult Immunocompetent Mice1997/11/01English169
Optimizing Adoptive Polyclonal T Cell Immunotherapy of Lymphomas, Using a Chimeric T Cell Receptor Possessing CD28 and CD137 Costimulatory Domains2007/08/01English168
Basis of Pulmonary Toxicity Associated with Cationic Lipid-Mediated Gene Transfer to the Mammalian Lung1997/04/10English167
Comprehensive Analysis of the Acute Toxicities Induced by Systemic Administration of Cationic Lipid:Plasmid DNA Complexes in Mice2000/12/10English166
Tetracycline Repressor, tetR, rather than the tetR–Mammalian Cell Transcription Factor Fusion Derivatives, Regulates Inducible Gene Expression in Mammalian Cells1998/09/01English165
A Phase I Study of an Adeno-Associated Virus-CFTR Gene Vector in Adult CF Patients with Mild Lung Disease. Johns Hopkins Children's Center, Baltimore, Maryland1996/06/10English165
Plasmid DNA Is Superior to Viral Vectors for Direct Gene Transfer into Adult Mouse Skeletal Muscle1993/12/01English165
A Phase I Study of Aerosolized Administration of tgAAVCF to Cystic Fibrosis Subjects with Mild Lung Disease2001/10/10English165
RNA Interference-Mediated Gene Silencing of Pleiotrophin Through Polyethylenimine-Complexed Small Interfering RNAsIn VivoExerts Antitumoral Effects in Glioblastoma Xenografts2006/07/01English165
Cell Lines for the Production of Recombinant Adeno-Associated Virus1995/10/01English164
Treatment of Advanced Leukemia in Mice with mRNA Engineered T Cells2011/12/01English161
Transfer of a TCR Gene Derived from a Patient with a Marked Antitumor Response Conveys Highly Active T-Cell Effector Functions2005/04/01English161
Adenovirus Serotype 5 Fiber Shaft InfluencesIn VivoGene Transfer in Mice2003/05/20English159
The Presence of Human Coxsackievirus and Adenovirus Receptor Is Associated with Efficient Adenovirus-Mediated Transgene Expression in Human Melanoma Cell Cultures1998/11/01English158
Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene (ASPA) to the Human Brain2002/07/20English158
Gene Therapy for Metastatic Brain Tumors by Vaccination with Granulocyte-Macrophage Colony-Stimulating Factor-Transduced Tumor Cells1997/06/10English156
Branched Cationic Peptides for Gene Delivery: Role of Type and Number of Cationic Residues in Formation and in Vitro Activity of DNA Polyplexes1999/01/20English156
Innate Immunity to Adenovirus2014/04/01English156
Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Types 2, 5, and 6 in Cystic Fibrosis and Normal Populations: Implications for Gene Therapy Using AAV Vectors2006/04/01English156
Gene Expression Following Direct Injection of DNA into Liver1994/12/01English155
Misinterpreting the Therapeutic Effects of Small Interfering RNA Caused by Immune Stimulation2008/10/01English154
Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors2011/11/01English153
Delivery Vehicles for Small Interfering RNA In Vivo2008/02/01English153
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial2017/11/01English151
Clinical Trial to Assess the Safety, Feasibility, and Efficacy of Transferring a Potentially Anti-Arthritic Cytokine Gene to Human Joints with Rheumatoid Arthritis. University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania1996/07/01English151