Human Gene Therapy

Title Publication Date Language Citations
High Levels of Foreign Gene Expression in Hepatocytes after Tail Vein Injections of Naked Plasmid DNA1999/07/01English690
Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors2010/06/01English660
Treatment of Leber Congenital Amaurosis Due toRPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial2008/10/01English609
Episomal Vectors Rapidly and Stably Produce High-Titer Recombinant Retrovirus1996/08/01English596
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN2018/03/01English476
Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors1998/12/10English461
A Phase III Clinical Evaluation of Herpes Simplex Virus Type 1 Thymidine Kinase and Ganciclovir Gene Therapy as an Adjuvant to Surgical Resection and Radiation in Adults with Previously Untreated Glioblastoma Multiforme2000/11/20English414
A Powerful Nonviral Vector for In Vivo Gene Transfer into the Adult Mammalian Brain: Polyethylenimine1996/10/20English395
Nonviral Vectors in the New Millennium: Delivery Barriers in Gene Transfer2001/05/20English394
High-Level Transduction and Gene Expression in Hematopoietic Repopulating Cells Using a Human Imunodeficiency Virus Type 1-Based Lentiviral Vector Containing an Internal Spleen Focus Forming Virus Promoter2002/05/01English391
Activation of the Complement System by Synthetic DNA Complexes: A Potential Barrier for Intravenous Gene Delivery1996/08/01English391
Characterization of 911: A New Helper Cell Line for the Titration and Propagation of Early Region 1-Deleted Adenoviral Vectors1996/01/20English373
Innate Immune Mechanisms Dominate Elimination of Adenoviral Vectors FollowingIn VivoAdministration1997/01/01English362
Quantitative Analysis of the Packaging Capacity of Recombinant Adeno-Associated Virus1996/11/10English353
Insect Cells as a Factory to Produce Adeno-Associated Virus Type 2 Vectors2002/11/01English330
Adenoviral Gene Therapy Leads to Rapid Induction of Multiple Chemokines and Acute Neutrophil-Dependent Hepatic Injury in Vivo1999/04/10English327
Detailed Analysis of Structures and Formulations of Cationic Lipids for Efficient Gene Transfer to the Lung1996/09/10English321
Nonviral Gene Therapy: The Promise of Genes as Pharmaceutical Products1995/09/01English307
New Helper Cells and Matched Early Region 1-Deleted Adenovirus Vectors Prevent Generation of Replication-Competent Adenoviruses1998/09/01English305
The Innate Immune Response to Adenovirus Vectors2004/12/01English297
Systematic Determination of the Packaging Limit of Lentiviral Vectors2001/10/10English297
Gene Therapy by Intramuscular Injection of Plasmid DNA: Studies on Firefly Luciferase Gene Expression in Mice1993/08/01English293
Nomenclature for Synthetic Gene Delivery Systems1997/03/20English289
PEGylation of Adenovirus with Retention of Infectivity and Protection from Neutralizing Antibody in Vitro and in Vivo1999/05/20English288
Bone Marrow Mesenchymal Stem Cells: Historical Overview and Concepts2010/09/01English284
Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA2008/05/01English275
Improved Methods of Retroviral Vector Transduction and Production for Gene Therapy1994/01/01English271
In VitroEvidence That Metabolic Cooperation Is Responsible for the Bystander Effect Observed with HSVtkRetroviral Gene Therapy1993/12/01English271
Silencing and Variegation of Gammaretrovirus and Lentivirus Vectors2005/11/01English263
Characterization of Vectors for Gene Therapy Formed by Self-Assembly of DNA with Synthetic Block Co-Polymers1996/11/10English257