Virus-based gene therapy research

This study demonstrates the use of adeno-associated viruses (AAVs) for efficient gene transfer in the axolotl nervous system, identifying suitable serotypes and applications for neuronal tracing.

This study demonstrates that AAV gene therapy with TN-201 restores MYBPC3 protein levels, reverses cardiac hypertrophy, and improves survival in a murine model of MYBPC3-associated HCM.

The paper identifies and validates an alternative to VSV-G for lentivector pseudotyping to improve in vivo T cell transgene delivery for tumor therapy.

The paper characterizes hepatic lentiviral vector transduction in mice, finding strain-specific variations and QTLs linked to transduction efficiency and kinetics, suggesting the need for personalized gene therapy protocols.

The paper develops a nontransgenic method using bridge proteins to target viral vectors to cells expressing endogenous μ-opioid receptors in mouse and monkey brains.

This study compares 11 AAV serotypes for transduction efficiency and specificity in murine olfactory sensory neurons using nasal inoculation.

The paper identifies HR factors that inhibit dual AAV vector transduction and shows that inhibiting these factors improves transgene reconstitution.

This study compares AAV9 and AAV-PHP.eB transduction efficiency in pigtail macaques, finding AAV-PHP.eB achieves higher rates in cortical and spinal regions.

This study identifies vinorelbine as a compound that enhances the efficacy of a GM-CSF-armed oncolytic vaccinia virus in treating ovarian cancer in mice.

This paper develops a method for targeted gene delivery using adenovirus conjugated with antibody molecular glues, showing enhanced gene transfer to specific cell types in vitro and in vivo.

This study shows that high-dose AAV injection in mice induces neutralizing antibodies that limit subsequent AAV infections, with B cells playing a key role.

This paper develops a novel lentiviral vector for gene therapy of MLD that achieves high ARSA activity with minimal genomic integrations, improving safety and efficacy in preclinical models.

This paper maps the tissue tropism of ten AAV serotypes in mice after systemic delivery, identifying liver preference and unexpected transduction in adrenals, testes, and ovaries.

The paper investigates why engineered AAV variants have lower titers and finds that DNAse digestion heat reduces their stability, leading to artificially low titers.

The paper uses a CRISPR screen to identify genes affecting rAAV production in HEK 293 cells, finding that heparan sulfate biosynthesis and vesicular trafficking genes influence yield.

The paper reports Cryo-EM structures of AAV2 Rep68 bound to DNA at the AAVS1 integration site, revealing conformational changes and DNA distortion mechanisms during replication.

The paper shows that AAV libraries packaged as pools exhibit extensive chimerism due to length and homology dependence, affecting most genomes.

The paper identifies manufacturing plasmid sequences and disrupted vector genomes in liver tissue from a patient with AAV gene therapy-related hepatitis.

This study evaluates a rationally designed ITR that reduces toxicity and transduction restriction in hESCs while maintaining gene delivery efficiency.

This study investigates how post-translational modifications on adenovirus protein VII affect its function during early and late infection stages, revealing their role in localization, transcription, and protein interactions.

The paper assesses seroprevalence and cross-reactivity of neutralizing antibodies to six adenovirus types and suggests Ad1 or Ad2 as potential vaccine vector candidates.

This study introduces depletion-AAVs to permanently eliminate lymphocyte subsets in mice, showing B cells are critical for CD4 and CD8 T cell responses to chronic viral infection.

This study demonstrates that AAV9-mediated gene therapy prevents liver dysfunction in a mouse model of DGUOK deficiency.

This study investigates AAV tropism in hippocampal CA2 neurons, finding high expression of AAV-delivered genetic material due to abundant AAVR and glycan receptors.

This study identifies SLC35A1 as a host factor essential for rAAV transduction, showing its role in vector nuclear import across multiple AAV serotypes.

This paper identifies AAV serotypes effective for transducing amphibian CNS cells and develops tools for neural circuit tracing and manipulation in multiple amphibian species.

This study identifies epigenetic and cell cycle regulators that enhance AAV production by analyzing transcriptomic responses in cell lines with varying production capacities.

The paper identifies conditions that stabilize AAV inverted terminal repeats (ITRs) in E. coli, improving rAAV yield and purity.

This paper develops an AAV vector system with optimized miR.Ts placement to achieve specific and efficient transgene expression in microglia.

This paper develops a temperature-sensitive and interferon-silent Sendai virus vector for CRISPR-Cas9 delivery, achieving high gene editing efficiency in human cells without triggering immune responses.