Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
《人类基因治疗》是一本涵盖基因治疗各个方面的多学科期刊。该杂志通过提供最新的研究和技术突破，深入报道DNA、RNA和细胞疗法。人类基因治疗为科学和临床信息提供了一个中心论坛，包括伦理、法律的、监管、社会和商业问题，这使得治疗程序的进步和发展能够改善患者的结局，并最终治愈疾病。
Immunoengineering of the Vascular Endothelium to Silence MHC Expression During Normothermic Ex Vivo Lung Perfusion.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.117
Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with no Detectable Immunity.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.234
Virally Mediated Overexpression of Glial-Derived Neurotrophic Factor Elicits Age- and Dose-Dependent Neuronal Toxicity and Hearing Loss.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.028
Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.059
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
来源期刊：Human Gene TherapyDOI：10.1089/hum.2018.098
Utility of self-destructing CRISPR/Cas constructs for targeted gene editing in the retina.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.021
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.006
Dysregulated Expression of microRNA-21 and Disease-Related Genes in Human Patients and in a Mouse Model of Alport Syndrome.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.205
Impact of the Assembly-Activating Protein on Molecular Evolution of Synthetic Adeno-Associated Virus Capsids.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.085
Dual ABCA4-AAV vector treatment reduces pathogenic retinal A2E accumulation in a mouse model of autosomal recessive Stargardt Disease.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.132
Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.229
Lentiviral CRISPR Epigenome Editing of Inflammatory Receptors as a Gene Therapy Strategy for Disc Degeneration.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.005
Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.178
CAR T Cells Generated Using Sleeping Beauty Transposon Vectors and Expanded with an EBV-Transformed Lymphoblastoid Cell Line Display Antitumor Activity In Vitro and In Vivo.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.218
Application of CRISPR-Cas9 for Long Noncoding RNA Genes in Cancer Research.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.063
In vivo gene therapy for Mucopolysaccharidosis type III (Sanfilippo syndrome): A new treatment horizon.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.217
Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing
来源期刊：Human Gene TherapyDOI：10.1089/hum.2019.157
Establishment of a High-Yield Recombinant Adeno-Associated Virus/Human Bocavirus Vector Production System Independent of Bocavirus Nonstructural Proteins.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.173
Public acceptability of gene therapy and gene editing for human use: A systematic review.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.197
Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.151
Active Immunoprophylaxis and Vaccine Augmentations Mediated by a Novel Plasmid DNA Formulation
来源期刊：Human Gene TherapyDOI：10.1089/hum.2018.241
Silencing of GAS5 alleviates glaucoma in rat models by reducing retinal ganglion cell apoptosis.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.056
Artificial MicroRNA-Mediated Tgfbr2 and Pdgfrb Co-Silencing Ameliorates Carbon Tetrachloride-Induced Hepatic Fibrosis in Mice.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.047
New Frontiers of Corneal Gene Therapy.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.026
Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.009
Generation and clinical application of gene modified autologous epidermal sheets in Netherton syndrome - lessons learned from a phase 1 trial.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.049
Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings
来源期刊：Human Gene TherapyDOI：10.1089/hum.2019.016
Therapeutic Delivery of miR-143 Targeting Tumor Metabolism in Poorly Differentiated Thyroid Cancer Xenografts and Efficacy Evaluation Using 18F-FDG MicroPET-CT.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.160
Long-Term Effects of Gene Therapy in a Novel Mouse Model of Human MFRP-Associated Retinopathy.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.192
Specific overexpression of Mitofusin-2 in hepatic stellate cells ameliorates liver fibrosis in mice model.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.153
Engineering of Human Mesenchymal Stem/Stromal Cells with Vascular Endothelial Growth Factor-Encoding Minicircles for Angiogenic Ex Vivo Gene Therapy.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.154
Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.113
Systemic Safety of a Recombinant AAV8 Vector for Human Cocaine Hydrolase Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.233
Multiplex Epigenome Editing of Dorsal Root Ganglion Neuron Receptors Abolishes Redundant Interleukin 6, Tumor Necrosis Factor Alpha, and Interleukin 1β Signaling by the Degenerative Intervertebral Disc.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.032
Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.233
Gene therapy for Pompe disease: the time is now.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.109
The Oncolytic Adenovirus XVir-N-31 as a Novel Therapy in Muscle-Invasive Bladder Cancer.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.026
In vivo Exon Replacement in Mouse Atp7b Gene by CRISPR/Cas9 System.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.037
Conditional immortalization of lymphoid progenitors via tetracycline-regulated LMO2 expression.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.212
Validation and Safety of Visual Restoration by Ectopic Expression of Human Melanopsin in Retinal Ganglion Cells.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.009
Breathing New Life into TRAIL for Breast Cancer Therapy: Co-delivery of pTRAIL and Complementary siRNAs using Lipopolymers.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.096
Effect of Genetic Modifications on Physical and Functional Titers of Adenoviral Cancer Gene Therapy Constructs.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.240
Single Plasmid-Based, Upgradable, and Backward-Compatible Adenoviral Vector Systems.
来源期刊：Human gene therapyDOI：10.1089/hum.2018.258
Nonviral delivery of GRIM-19 gene inhibits tumor growth with reduced local and systemic complications.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.134
The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE
来源期刊：Human Gene TherapyDOI：10.1089/HUM.2018.217
Adenoviral gene transfer of Gremlin modulates VEGF-A induced angiogenesis in porcine myocardium.
来源期刊：Human gene therapyDOI：10.1089/hum.2019.229
Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy
来源期刊：Human Gene TherapyDOI：10.1089/HUM.2019.041
Rational Engineering and Preclinical Evaluation of Neddylation and SUMOylation Site Modified Adeno-Associated Virus Vectors in Murine Models of Hemophilia B and Leber Congenital Amaurosis
来源期刊：Human Gene TherapyDOI：10.1089/hum.2019.164
Pro)renin Receptor RNA Interference Silencing Attenuates Diabetic Cardiomyopathy Pathological Process in Rats
来源期刊：Human Gene TherapyDOI：10.1089/HUM.2018.155
