Virus-based gene therapy research

Overcoming host immune responses to an AAV-delivered HIV-1 bNAb in rhesus macaques mediated by co-delivery of PD-L1

This study shows that co-delivering PD-L1 with AAV-delivered bNAb 10-1074 reduces ADA responses and improves bNAb durability in rhesus macaques.

Characterization of expression elements for an AAV delivered antibody in nonhuman primates when co-delivered with PD-L1

This study evaluates AAV transgene cassettes for expressing 10-1074 antibody in nonhuman primates and mice, showing promoter and WPRE effects on expression levels and immune responses.

NEO-AAV: an engineered extracellular vesicle-enveloped AAV platform for activation-coupled T cell transduction and CAR-T cell generation

This paper introduces NEO-AAV, an engineered AAV platform that enhances T cell transduction and CAR-T cell generation by enveloping AAV in extracellular vesicles, improving immune resistance and transduction efficiency.

Single Particle Adsorption and Response Quantification for Functional AAVx Titer and Dose Analysis

This paper introduces SPARQ, a method to quantify functional AAV vectors at single-particle resolution, revealing functional heterogeneity and improving dose analysis in gene therapy.

AAV tools enable functional modulation and readout of central and peripheral nervous systems in spiny mice

The paper identifies AAV vectors that efficiently deliver transgenes to the nervous systems of spiny mice, enabling functional studies of regeneration.

Intratumoural oncolytic HSV-1 reshapes the local and systemic immune landscape through CD8+ T cell reprogramming

This paper shows that intratumoural oncolytic HSV-1 reprograms systemic immunity by inducing specific CD8 T cell populations that correlate with clinical response to immunotherapy.

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.

Highly Efficient Lentiviral Transduction of Human iPSC-Derived Microglia and Macrophages

This paper develops a highly efficient lentiviral transduction method for human iPSC-derived microglia and macrophages using a modified promoter and plasmid design.

Low seroprevalence of neutralizing antibodies to gorilla adenovirus 32 (GRAd32) in southern African populations supports evaluation of this vector platform for HIV vaccine development

This study finds low neutralizing antibody prevalence to GRAd32 in southern African populations, suggesting its potential as an HIV vaccine vector despite prior Ad26 vaccination.

Enhancing adeno-associated virus cellular entry through receptor engineering

This study investigates the molecular mechanisms of AAV receptor AAVR and AAVR2, identifies functional motifs, and demonstrates enhanced AAV entry through engineered receptors.

Neuronal targeted AAV Micro-Dystrophin restores neurobehavioural co-morbidities, grip strength and motor coordination in mdx52 mouse model of Duchenne Muscular Dystrophy

This study evaluates neuronal-targeted AAV micro-dystrophin therapy in mdx52 mice, showing IV delivery improves neurobehavioural, motor, and neuromuscular deficits more effectively than ICV delivery.

Novel Muscle-Tropic AAV Capsids with Dramatically Enhanced Transduction and Safety Profiles in Non-Human Primates

The paper engineers AAV capsids with improved muscle transduction and reduced off-target distribution in non-human primates using the REACH platform.

An oncolytic adenovirus armed with anticancer prodrug-activating enzyme offers enhanced tumor killing and antitumor immunity.

This study develops an oncolytic adenovirus that activates a prodrug to enhance tumor killing and antitumor immunity through direct oncolysis, bystander effects, and systemic immune responses.

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.

Transgene Expression Kinetics and Replication Potential of Recombinant Adenovirus Serotype 4 in a Mouse Model and its Use as a Herpes Simplex Virus Vaccine

This paper evaluates transgene expression and viral replication of recombinant Ad4 in mice, showing protective efficacy against HSV-2 infection.

Structural dynamics insights into principles underlying the fitness of new broadly potent AAVs

The paper develops a new AAV engineering workflow, scAAVengr-Hunt, to create potent vectors and identifies a structural mechanism underlying their broad species and tissue potency.

LVV SMRTcap reveals extensive proviral variation in lentiviral vector-transduced CAR T cells

The paper uses long-read sequencing to analyze proviral variation in lentiviral vector-transduced CAR T cells, finding deletions and mutations affecting transgene function.

Engineering high-titer lentiviral vectors for robust expression of RNA-based gene circuits

This study develops high-titer lentiviral vectors for RNA-based gene circuits by optimizing gene syntax and genetic parts to enhance vector production and expression.

Oncolytic measles virus reprograms the tumor microenvironment in a vascularized mesothelioma-on-chip model

This study develops a vascularized mesothelioma-on-chip model to evaluate oncolytic measles virus effects on the tumor microenvironment.

Activation-dependent lentiviruses enable antigen-specific T cell expansion and transduction

This paper develops 4-1BB-targeting lentiviral vectors to selectively expand and transduce antigen-specific T cells, enhancing their function in cancer immunotherapy.

Optimizing Lentiviral Vector-Based Delivery of SCN1A Transgenes to Mammalian Cells

This study evaluates lentiviral vector delivery of SCN1A transgenes, finding that sequence optimization and epigenetic factors affect long-term expression.

Manipulation of CA1 neuronal subtypes through Cre-mediated viral delivery in mice

The paper describes an AAV-based strategy with CaMKII-driven Cre expression to target and study unlabeled hippocampal pyramidal neurons in mice, including protocols for viral production, delivery, and functional validation.

Optimized AAV capsids robustly transduce airway epithelial cells

This paper develops novel AAV capsids with improved transduction efficiency for airway epithelial cells, reducing required doses and enhancing therapeutic potential for respiratory diseases.

AAV2 Crosslinks Actin Filaments: Implications for AAV Gene Therapy Vector Design

This study identifies AAV2's ability to bundle actin filaments, suggesting a new class of actin-binding viruses and offering insights for improving AAV gene therapy.

Adeno-Associated Virus Co-Precipitation with Extracellular Vesicles for Genome Editing in Rodent Embryo

This paper describes a method to co-isolate AAV with extracellular vesicles for efficient genome editing in rodent embryos, achieving higher viability and targeting efficiency compared to traditional purification methods.

In vivo base editing via single myotrophic adeno-associated viruses in dystrophic mouse muscle and satellite cells

This study demonstrates that single-vector adenine base editing via MyoAAV can recode a DMD-causing mutation in mice, with optimal therapeutic outcomes achieved in juvenile animals.

Computational Rational Design of Larger AAV Icosahedral Capsids

This paper designs a larger AAV capsid with T=3 symmetry using AI, increasing its cargo capacity to up to 35 kb.

Reducing encapsidated impurity DNA derived from plasmid backbone by modifying the p5 terminal resolution site in rAAV vector production

This study modifies the p5 terminal resolution site in AAV vectors to reduce encapsidated plasmid backbone DNA, improving vector safety and yield.

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.