Virus-based gene therapy research

Multi-omics Profiling Reveals an NF-κB-Driven Anti-apoptotic Network Underlying Resistance to Oncolytic VSV in Prostate Cancer Cells

This study identifies an NF-κB-driven anti-apoptotic network in PC3 prostate cancer cells that contributes to resistance against oncolytic VSV, suggesting combination therapies may enhance virotherapy efficacy.

Viral evolution of T1L mammalian orthoreovirus enhances breast cancer stem-like cell killing

This study develops novel oncolytic viruses derived from T1L mammalian orthoreovirus that enhance killing of therapy-resistant breast cancer stem-like cells.

Bovine AAV - a promising vector for pulmonary gene therapy

This study evaluates Bovine AAV as a vector for pulmonary gene therapy, showing efficient lung transduction and reduced neutralization compared to conventional AAV serotypes.

Refining gene delivery to skeletal muscle with a dual-strategy approach of muscle-tropic AAV capsids and muscle-specific promoters.

The paper investigates a dual-strategy approach using a muscle-tropic AAV capsid and muscle-specific promoter to improve gene delivery efficiency and reduce off-target expression in skeletal muscle.

Development of a recombinant adeno-associated virus vector for human T lymphocyte- and natural killer cell-targeted gene therapy

This paper develops a CD7-targeted rAAV vector for efficient gene delivery to human T and NK cells, showing reduced off-target effects and improved transgene expression through genome optimization.

Combined Disruption of Multiple Cytokine Signaling Pathways Enables One-Step Anterograde Tracing with Vesicular Stomatitis Virus

This paper develops a VSVdG-based strategy for one-step anterograde tracing in neural circuits by disrupting cytokine signaling pathways and using AAV trans-complementation.

Development of the EASY-HSV (Efficient And Simple high-Yield Herpes Simplex virus Vector) system based on HSV-1 genome-maintaining HEK293 cells

The paper develops an EASY-HSV system using HSV-1 genome-maintaining HEK293 cells to simplify and increase the yield of HSV amplicon vector production.

Alpha Protein Kinase 3 Gene Therapy Restores Heart Function in Mouse and Human Models of Cardiomyopathy

This study shows that gene therapy with AAV-ALPK3 improves heart function in mouse and human models of cardiomyopathy caused by ALPK3 variants.

TBC1D23-AAVR Interaction Drives Endosome-to-TGN Trafficking Required for rAAV Transduction

This paper identifies TBC1D23 as a host protein that binds AAVR and mediates endosome-to-TGN trafficking required for rAAV transduction.

Rab9 depletion enhances human adenovirus type 26 transduction efficiency through increased internalization and reduced late endosomal/lysosomal retention

This study shows that Rab9 depletion increases HAdV-D26 transduction efficiency by enhancing internalization and reducing late endosomal/lysosomal retention.

A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3

BG147 enhances lentivector transduction in HSPCs ex vivo and in mouse photoreceptors in vivo by degrading IFITM3.

Structural Analysis of Recombinant AAV Vector Genomes at Single-Molecule Resolution

The paper develops a pipeline to classify AAV genome structures at single-molecule resolution using long-read sequencing, revealing structural heterogeneity and its contributing factors.

AAV2 Bypasses Direct Endosomal Escape by Using AAVR to Access the Trans-Golgi Network en Route to the Nucleus.

This study shows AAV2 bypasses endosomal escape by using AAVR to access the trans-Golgi network, challenging the model of direct endosomal escape.

Oncolytic Adenovirus Armed with cGAS Activates STING Pathway and Enhances Antitumor Immunity in Lung Cancer with Superior Combined Efficacy of PD-L1 Therapy

This study develops oncolytic adenoviruses expressing cGAS to activate the STING pathway and enhance antitumor immunity in lung cancer, showing improved efficacy when combined with PD-L1 therapy.

Retargeted adenoviruses for local IgA and CD47 blocker production as a novel cancer therapy

This paper develops a retargeted adenovirus system to produce IgA antibodies and CD47 blockers in tumors, enhancing innate immune responses and reducing tumor volume in mice.

Optimization of AAV tools to target Muller glial cells for retinal gene therapy

This study optimizes AAV serotypes and promoters to specifically target Muller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.

Serotype-dependent differences in AAV cellular transduction rates in the hypothalamus of Arctic ground squirrels

The paper compares AAV serotype transduction rates in the hypothalamus of Arctic ground squirrels, finding AAV1 has higher transduction rates than AAV-DJ.

Neuron-Specific WWOX Gene Therapy Produces Dose-Dependent, Durable Rescue in a Model of WWOX-Related Epileptic Encephalopathy

This study identifies optimal parameters for neuron-targeted WWOX gene therapy in a mouse model of WWOX-related epileptic encephalopathy, showing durable rescue with a defined therapeutic window and dose.

Retrograde transduction of dopaminergic cells in substantia nigra of rhesus monkey

This study compares the retrograde transduction efficiency of four viral vectors in dopaminergic neurons of rhesus monkeys after striatal injection.

Next Generation AAV-F Capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency

This study compares AAV9 and AAV-F capsids for treating PDHD in mice, finding AAV-F improves survival and enzyme activity at lower doses but causes motor impairments.

Rewiring oncogenic signaling to RNA vector replication for the treatment of metastatic cancer

This paper introduces a RNA vector that replicates specifically in cancer cells with hyperactive HER2, showing lower toxicity and better efficacy against ovarian cancer in mice, and synergizes with chemotherapy.

AAV-only targeting of ventral tegmental area dopamine neurons for optical self-stimulation studies in mice

This paper develops an AAV-only method to target VTA dopamine neurons in mice for optogenetic studies, achieving high efficiency and fidelity.

Receptor-guided AAV Tropism Engineering via MATCH

This paper introduces MATCH, a method to engineer AAV tropism by conjugating targeting proteins, achieving efficient transduction of T cells and enhanced brain delivery in vivo.

Comparative Evaluation of Adeno-Associated Virus and Lentivirus Mediated Gene Transfer in Adult Rat Optic Nerve

This study compares AAV and lentivirus gene transfer efficiency in adult rat optic nerve, finding AAV-PHP.eB with CAG promoter achieves high oligodendrocyte transduction.

EctoShed: A novel Gene Delivery Platform for Functional Analysis of Adipocyte-Shed Proteoforms

EctoShed is a gene delivery platform that enables expression of both membrane-bound and soluble proteoforms in adipocytes, revealing their functional roles in metabolic regulation.

Efficient NK cell transduction with VSV-G-pseudotyped lentiviral vectors

This study optimizes VSV-G-pseudotyped lentiviral transduction of NK cells by improving activation, construct design, and enhancer use, achieving high efficiency without compromising cell function.

Pancreatic tumor microenvironment reprogramming via alloantigen-expressing virotherapy elicits tumor rejection and improves immunotherapy response

Oncolytic virus-based delivery of an alloantigen improves antitumor immunity and synergizes with immune checkpoint inhibitors in pancreatic cancer.

AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome

This paper demonstrates the use of AAV9 vectors to deliver AntagoNATs targeting a natural antisense transcript in a DS mouse model, improving survival and reducing seizures.

Mapping AAV capsid sequences to functions through function-guided in silico evolution

The paper introduces ALICE, a platform that uses function-guided in silico evolution to design AAV capsids with tailored properties, demonstrating improved targeting and tropism in mice.

Technical and biological sources of noise confound multiplexed enhancer AAV screening

The paper presents a barcoded multiplex strategy to screen enhancer AAVs using single-cell RNA sequencing and taxonomy mapping, but finds significant technical and biological noise complicates accurate expression pattern analysis.