Virus-based gene therapy research

Tailored Cell Cycle Modulation Enhances AAV Manufacturing: Balancing Arrest with Adaptive Stress Responses

This study evaluates how cell-cycle arrest and stress pathways affect rAAV production, finding that tailored modulation improves yields.

Comparative Study of BBB-Targeting AAV Capsids on CentralNervous System Delivery Efficiency

This study compares BBB-targeting AAV capsids for CNS delivery efficiency and safety, finding CNSRCV300 offers the best balance between brain targeting and biosafety.

Deep learning-guided design of cell type-specific AAV promoters

This study designs cell type-specific AAV promoters using deep learning, outperforming rational approaches in mouse retinal cells and showing promise for human applications.

Engineered muscle tissues with enhanced maturation enable the identification of clinically relevant rAAV capsids

This study uses engineered human muscle tissues to evaluate AAV capsid efficiency, finding that more mature tissues show improved transduction by AAV9 and MyoAAV variants.

Designing AAV Capsid Protein with viability-guided Diffusion Model

This paper introduces AAVDiffusion, a viability-guided diffusion model for designing viable AAV capsid proteins, showing improved performance in generating functional sequences.

Preparation and application of YFV-17D-derived Anterograde Trans-neuronal Viral Vectors for Neuroscience Research

The paper describes the preparation and application of anterograde trans-neuronal viral vectors derived from YFV-17D for mapping brain connectivity.

Enhanced lentiviral gene delivery to mammalian cells via paired cell surface and viral envelope engineering

This paper improves lentiviral gene delivery to mammalian cells by engineering virus-envelope and cell-surface interactions using FITC labeling and chimeric envelopes.

Adeno-associated viruses (AAVs) induce dose-dependent neonatal ventriculomegaly following intracerebroventricular administration

This study investigates the dose-dependent effects of AAV serotypes on neonatal ventriculomegaly and CNS inflammation following intracerebroventricular administration in mice.

Decoupling Efficacy from Toxicity: Engineering Spatial Control in AAV-Mediated Gene Therapy

This study shows that using tissue-specific AAV variants and local delivery reduces hepatic toxicity while maintaining therapeutic efficacy in gene therapy.

Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo

This paper demonstrates efficient and durable gene activation using Cas9-mediated epigenome editing in a mouse model of LAMA2-CMD, showing sustained therapeutic effects beyond one year.

LISA: A Comprehensive R Package for Lentiviral Integration Site Analysis in Gene Therapy Safety Assessment

LISA is an R package for analyzing lentiviral integration sites in gene therapy to assess safety by identifying risk-associated genomic regions and tracking clonal dynamics.

Genetically encoded SpyTag enables modular AAV retargeting via SpyCatcher-fused ligands for targeted gene delivery.

This paper introduces a modular AAV retargeting platform using SpyTag/SpyCatcher for targeted gene delivery, demonstrating specific cancer cell transduction and therapeutic potential.

Targeted gene transfer into developmentally defined cell populations of the primate brain

This paper introduces a method for targeted gene transfer into specific cell populations of the fetal primate brain using ultrasound-guided viral injections.

Aptamer-Fortified VV-GMCSF-Lact for Systemic Glial Tumor Virotherapy

This study shows that aptamer-fortified oncolytic viruses reduce glioblastoma tumor growth and improve mouse survival in xenograft models.

Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system

This study uses AAV9-mediated PIGA gene replacement to rescue PIGA deficiency phenotypes in mice, improving survival, brain structure, and neurological function.

Engineered AAV Capsids with Enhanced Extracellular Vesicle Loading via Rational Design and Directed Evolution

The paper engineers AAV9 capsids to enhance extracellular vesicle loading through rational design and directed evolution, achieving up to threefold increased EV-AAV yield and improved transduction efficiency.

A compact glutamic acid decarboxylase 67 promoter enables inhibitory neuron-targeted AAV gene therapy for treatment-resistant epilepsy

This paper develops a compact promoter for AAV gene therapy targeting inhibitory neurons, showing efficacy in treating epilepsy by restoring GABA function.

Administration of barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport

The paper tests AAV capsid variants in non-human primates to identify those with improved retrograde transport properties.

Insights into the AAV packaging mechanism: Cryo-EM Structure of the AAV2 Rep-Capsid Packaging Complex

This study determines the cryo-EM structure of the AAV2 Rep-capsid packaging complex, revealing how Rep proteins interact with the capsid and contribute to genome packaging.

Gene supplementation in Vanishing White Matter mice ameliorates the disease

This study shows that gene supplementation in astrocytes of Vanishing White Matter mice improves motor skills and CNS pathology without side effects.

VCAP-102 Achieves Exceptional BBB Penetration in Marmosets After Ten Years of AAV Capsid Evaluation

The paper evaluates BBB-penetrant AAV capsids in marmosets over a decade and identifies VCAP-102 as a superior candidate for systemic gene therapy.

Inner-ear delivery of AAV2-retro robustly labels lateral olivocochlear efferents but results in off-target transfection in the contralateral cochlea and the brain

This study shows that AAV2-retro injected into the inner ear labels the lateral olivocochlear pathway but causes off-target labeling in the contralateral cochlea and brain.

3D co-cultures of primary human hepatocytes and Kupffer-like cells to address innate immune responses to rAAV

This study develops a 3D co-culture model of human hepatocytes and Kupffer-like cells to investigate innate immune responses to rAAV vectors.

AAV-mediated neuronal expression of FOXG1 restores oligodendrocyte maturation, myelination, and hippocampal structure in mouse models of FOXG1 Syndrome

This study shows that delivering FOXG1 to neurons via AAV vectors improves oligodendrocyte maturation, myelination, and hippocampal structure in mouse models of FOXG1 syndrome.

AAV vectors with enhancer-controlled ITR promoters

This paper designs AAV vectors with enhancer-controlled ITR promoters to improve payload capacity and enable tissue-specific gene editing with a single AAV.

Tripartite AAV Systems for EYS Retinal Gene Therapy

This paper develops a tripartite AAV system to deliver the large EYS gene using recombination, showing successful in vitro and in vivo expression of full-length EYS protein.

Selective targeting of hippocampal fast-spiking basket cell interneurons via noninvasive enhancer-AAV delivery

The paper develops a non-invasive method to specifically target fast-spiking basket cell interneurons in the mouse hippocampus using enhancer-AAV delivery.

Targeted and efficient AAV therapy for neuroblastoma via direct capsid-antibody coupling

This paper develops AAV-STITCH, a method to target neuroblastoma cells using anti-GD2 ScFv coupled to AAV capsids, showing selective transduction and tumor suppression in mouse models.

Peptide-MHC-targeted retroviruses enable in vivo expansion and gene delivery to tumor-specific T cells

This study develops pMHC-targeted retroviruses for in vivo gene delivery to tumor-specific T cells, enhancing their expansion and anti-tumor activity in mouse models.

Next-generation enhancer AAVs for selective interspecies targeting of midbrain dopaminergic neurons

The paper develops and benchmarks enhancer-AAVs for selective targeting of midbrain dopaminergic neurons across species, identifying a specific enhancer and optimizing AAV backbones to improve specificity and efficiency.