HUMAN GENE THERAPY

Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice.

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

Utility of self-destructing CRISPR/Cas constructs for targeted gene editing in the retina.

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.

Dysregulated Expression of microRNA-21 and Disease-Related Genes in Human Patients and in a Mouse Model of Alport Syndrome.

Impact of the Assembly-Activating Protein on Molecular Evolution of Synthetic Adeno-Associated Virus Capsids.

Dual ABCA4-AAV vector treatment reduces pathogenic retinal A2E accumulation in a mouse model of autosomal recessive Stargardt Disease.

Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer.

Lentiviral CRISPR Epigenome Editing of Inflammatory Receptors as a Gene Therapy Strategy for Disc Degeneration.

Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.

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.

Application of CRISPR-Cas9 for Long Noncoding RNA Genes in Cancer Research.

In vivo gene therapy for Mucopolysaccharidosis type III (Sanfilippo syndrome): A new treatment horizon.

Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing

Establishment of a High-Yield Recombinant Adeno-Associated Virus/Human Bocavirus Vector Production System Independent of Bocavirus Nonstructural Proteins.

Public acceptability of gene therapy and gene editing for human use: A systematic review.

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.

Active Immunoprophylaxis and Vaccine Augmentations Mediated by a Novel Plasmid DNA Formulation

Silencing of GAS5 alleviates glaucoma in rat models by reducing retinal ganglion cell apoptosis.

Artificial MicroRNA-Mediated Tgfbr2 and Pdgfrb Co-Silencing Ameliorates Carbon Tetrachloride-Induced Hepatic Fibrosis in Mice.

New Frontiers of Corneal Gene Therapy.

Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid.

Generation and clinical application of gene modified autologous epidermal sheets in Netherton syndrome - lessons learned from a phase 1 trial.

Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings

Therapeutic Delivery of miR-143 Targeting Tumor Metabolism in Poorly Differentiated Thyroid Cancer Xenografts and Efficacy Evaluation Using 18F-FDG MicroPET-CT.

Long-Term Effects of Gene Therapy in a Novel Mouse Model of Human MFRP-Associated Retinopathy.

Specific overexpression of Mitofusin-2 in hepatic stellate cells ameliorates liver fibrosis in mice model.

Engineering of Human Mesenchymal Stem/Stromal Cells with Vascular Endothelial Growth Factor-Encoding Minicircles for Angiogenic Ex Vivo Gene Therapy.

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Systemic Safety of a Recombinant AAV8 Vector for Human Cocaine Hydrolase Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice.

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.

Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9.

Gene therapy for Pompe disease: the time is now.

The Oncolytic Adenovirus XVir-N-31 as a Novel Therapy in Muscle-Invasive Bladder Cancer.

In vivo Exon Replacement in Mouse Atp7b Gene by CRISPR/Cas9 System.

Conditional immortalization of lymphoid progenitors via tetracycline-regulated LMO2 expression.

Validation and Safety of Visual Restoration by Ectopic Expression of Human Melanopsin in Retinal Ganglion Cells.

Breathing New Life into TRAIL for Breast Cancer Therapy: Co-delivery of pTRAIL and Complementary siRNAs using Lipopolymers.

Effect of Genetic Modifications on Physical and Functional Titers of Adenoviral Cancer Gene Therapy Constructs.

Single Plasmid-Based, Upgradable, and Backward-Compatible Adenoviral Vector Systems.

Nonviral delivery of GRIM-19 gene inhibits tumor growth with reduced local and systemic complications.

The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE

Adenoviral gene transfer of Gremlin modulates VEGF-A induced angiogenesis in porcine myocardium.

Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy

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

Pro)renin Receptor RNA Interference Silencing Attenuates Diabetic Cardiomyopathy Pathological Process in Rats