Virus-based gene therapy research

A facile chemical strategy to synthesize precise AAV-protein conjugates for targeted gene delivery

This paper describes a chemical strategy to create AAV-protein conjugates for targeted gene delivery, showing efficient and selective transduction of HER2+ cancer cells with minimal off-target effects.

Site-Specific Noninvasive Delivery of Retrograde Viral Vectors to the Brain

This study demonstrates that AAV9.retro can safely and efficiently deliver genes to neuronal projections noninvasively using FUS-BBBO, offering improved specificity compared to invasive methods.

CoreTIA: a modular, statistically robust transduction inhibition assay for AAV neutralization

coreTIA provides a modular and statistically robust assay for measuring AAV neutralizing antibodies with quantified uncertainty and consistent performance across serotypes.

Adeno-Associated Virus 2 (AAV2) - induced RPA exhaustion generates cellular DNA damage and restricts viral gene expression

This study shows that AAV2 genomes compete with host RPA for binding, causing replication stress and DNA damage, which suppresses viral gene expression.

Suppressing microtubule detyrosination augments AAV2 endosomal escape and gene delivery.

This study shows that inhibiting microtubule detyrosination improves AAV2 endosomal escape and gene delivery in cells and mouse models.

Pervasive Horizontal Transfer of Adeno-Associated Virus Capsid Genes.

The paper reveals that AAV capsid genes have undergone extensive horizontal transfer across host species and rep lineages, with some variants originating from historical adenovirus stocks.

Selective depletion of cancer cells with extrachromosomal DNA via lentiviral infection

The paper observes that lentiviral infection selectively depletes cells containing extrachromosomal DNA (ecDNA), suggesting potential therapeutic strategies for ecDNA-driven cancers.

Repeat Systemic Delivery of Cross-Neutralization Resistant Synthetic Vesiculoviruses Immunomodulates the Tumor Microenvironment

A glycoprotein-engineered vesiculovirus platform circumvents neutralizing antibodies, enabling repeat intravenous dosing and sustained anti-tumor immunity in preclinical models.

Therapeutic Knock-in Genome Editing Using Single AAV Vectors in Mouse Models of Inherited Liver Disease

This paper develops a single AAV vector system for liver-targeted gene knock-in therapy in mouse models of inherited liver diseases, achieving efficient therapeutic gene insertion without germline transmission.

Increased in vivo transduction of AAV-9 cargo in Alport podocytes

This study evaluates AAV9 transduction efficiency in Alport syndrome podocytes, finding enhanced transduction in Alport podocytes both ex vivo and in vivo.

Fully functional AAV viral vectors with highly altered structural cores and subunit interfaces using ProteinMPNN

This study uses ProteinMPNN to redesign AAV2 capsid proteins, resulting in functional variants with altered cores and interfaces while retaining transduction efficiency.

Mechanisms of AAV neutralization by human alpha-defensins

This study identifies how human alpha-defensins neutralize AAV2 and AAV6 by blocking viral entry and nuclear trafficking, revealing a common mechanism for inhibiting non-enveloped viruses.

AAV-mediated gene therapy for SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes

This study shows that AAV-mediated gene therapy in SLC13A5-deficient mice reduces extracellular citrate, normalizes electrophysiologic and sleep issues, and improves seizure resistance.

In vivo selection and glymphatic delivery of AAV5 capsids engineered to target human glial progenitor cells

The paper describes engineering AAV5 capsids to target human glial progenitor cells and uses glymphatic delivery to achieve efficient brain-wide transduction with minimal off-target effects.

Buffer Valency Engineering Enables High-concentration and Shelf-stable DNA Transfection Particles for Viral Vector Production

This paper develops a method to create high-concentration, shelf-stable DNA transfection particles for AAV production, achieving tenfold higher pDNA concentration and reduced dosing volume.

Pancreatic endocrine cells are transduced by adeno-associated virus serotypes 2 and 9 but not 6

This study evaluates the efficiency of AAV serotypes 2, 6, and 9 in transducing pancreatic endocrine cells, finding AAV2 is most effective while AAV6 causes cytotoxicity.

Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice

This study compares AAV9-driven motor neuron transduction efficiency using different CNS delivery methods in mice, finding that unilateral ICV delivery provides the highest and most consistent transduction with minimal peripheral viral copies.

Oncolytic HSV-IL27 expression improves CD8 T cell function and therapeutic activity in syngeneic glioma models

This study develops an oncolytic HSV expressing IL-27 (C027) that improves survival in syngeneic glioma models by enhancing CD8 T cell function and inducing durable immune memory.

Advancing Liver Gene Therapy: Enhanced Transduction with GalNAc-Bioconjugated rAAV Capsids

This study enhances liver gene therapy by conjugating GalNAc to rAAV capsids, improving transduction efficiency in mice while reducing required dosages.

Combining Machine Learning and Multiplexed, In Situ Profiling to Engineer Cell Type and Behavioral Specificity

The paper develops a platform combining machine learning and multiplexed in situ screening to identify enhancers with cell-type and behavioral specificity for neural circuit engineering.

Modeling Adeno-Associated Viral Vector 6-mediated In Vivo Gene Delivery to Expanded Non-Mobilized Haemopoietic Stem Cells from Transfusion dependent Thalassemia Patients in a Humanized Mouse

This study demonstrates the feasibility of using dual-ssAAV6 vectors for in vivo gene delivery to HSC from thalassemia patients in a humanized mouse model, showing efficient transduction and long-term persistence.

A Drug-Elicitable Alternative-Splicing Module (DreAM) for Tunable AAV Expression and Controlled Myocardial Regeneration

3D Quantification of Viral Transduction Efficiency in Living Human Retinal Organoids

This paper introduces a method to quantitatively assess viral transduction efficiency in living human retinal organoids using 3D imaging and deep learning.

Engineered AAV9 as in vivo gene delivery platform for the selective transduction of TME cell subsets

This paper engineers AAV9 vectors to selectively deliver genes to specific tumor cell subsets, demonstrating efficacy in preclinical cancer models and clinical relevance.

rAAV-miniBEND: A targeted vector for brain endothelial cell gene delivery and cerebrovascular malformation modeling

The paper develops a rAAV vector, rAAV-miniBEND, for specific gene delivery to brain endothelial cells and models cerebrovascular malformations like CCM and AVM.

AAV gene therapy for Cockayne syndrome

This study demonstrates that AAV9-CSA gene therapy extends lifespan and distributes human CSA in the brain and heart of Csa-/-;Xpa-/- mice, but also causes adverse effects.

STARR-CRAAVT: A platform to design cell type-specific regulatory elements for next-generation gene therapy

This study introduces STARR-CRAAVT, a platform to identify cell type-specific enhancers for gene therapy, showing promoter preference and enhancer positioning significantly affect activity.

Inter-individual variability in immune responses to AAV-mediated ocular gene delivery across species impedes reliable immunomonitoring profile

The paper shows that immune responses to AAV-mediated ocular gene delivery vary across species and individuals, complicating reliable immunomonitoring.

Induced Proteinuria Enhances Adeno-Associated Virus Transduction of Renal Tubule Epithelial Cells After Intravenous Administration

The paper shows that inducing proteinuria enhances viral vector transduction of renal tubule epithelial cells after intravenous administration.

Adaptive Machine Learning Framework enables Unprecedented Yield and Purity of Adeno-Associated Viral Vectors for Gene Therapy

This paper develops a machine learning framework using Bayesian optimization to enhance the yield and purity of AAV vectors in gene therapy.