Virus-based gene therapy research

AAV-mediated expression of mouse or human GLDC normalises metabolic biomarkers in a GLDC-deficient mouse model of Non-Ketotic Hyperglycinemia

This study demonstrates that AAV-mediated GLDC expression reduces glycine levels and normalizes folate metabolism in a GLDC-deficient mouse model of NKH.

Optimal AAV capsid/promoter combinations to target specific cell types in the common marmoset cerebral cortex

The paper evaluates AAV capsid/promoter combinations for cell type-specific gene expression in marmoset cerebral cortex, identifying optimal pairs for neurons, astrocytes, and oligodendrocytes.

In utero adeno-associated virus (AAV)-mediated gene delivery targeting sensory and supporting cells in the embryonic mouse inner ear

This paper describes a protocol for in utero AAV-mediated gene delivery to the mouse inner ear, targeting sensory and supporting cells, and standardizes imaging procedures for reproducibility.

Standardized Nomenclature and Reporting for PacBio HiFi Sequencing and Analysis of rAAV Gene Therapy Vectors

This paper establishes standardized nomenclature and reporting for analyzing rAAV vectors using PacBio HiFi sequencing to improve quality assessment and vector design.

A Rapid Method for Producing Adeno-Associated Viral Vectors Suitable for Transducing Rodent Neurons in vitro and in vivo

This paper introduces a simplified AAV packaging protocol that produces sufficient virus for testing in rodent CNS without requiring large equipment.

Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates

The paper identifies an optimized AAV capsid, AAV-DB-3, that efficiently transduces deep brain and cortical structures in adult nonhuman primates at low doses.

Optimization of an adeno-associated viral vector for epidermal keratinocytes in vitro and in vivo

This study optimizes an AAV vector for efficient gene delivery to epidermal keratinocytes in vitro and in vivo by engineering capsid and promoter components.

A robust platform for BaEVRless-lentiviral synthesis and primary natural killer cell transduction

This paper develops a robust platform for producing BaEVRless-lentiviral vectors for transducing primary natural killer cells by optimizing production in ASCT2-KO HEK293T cells.

Combining CRISPR/Cas mediated terminal resolution with a novel genetic workflow to achieve high diversity adenoviral libraries

The paper introduces a genetic workflow called HFR to construct high-diversity adenoviral libraries using CRISPR/Cas9 mediated terminal resolution.

Harnessing the power of whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

This study evaluates AAV vectors in human liver explants under normothermic perfusion to assess their functionality and neutralization by human sera.

Highly conserved brain vascular receptor ALPL mediates transport of engineered viral vectors across the blood-brain barrier

This study identifies VCAP-102, an AAV9 variant with enhanced brain delivery across species, mediated by ALPL receptor interaction.

Noninvasive focal transgene delivery with viral neuronal tracers in the marmoset monkey

This paper demonstrates noninvasive delivery of AAV vectors to the marmoset brain using tFUS and BBB opening, enabling focal neuronal tracing.

CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells

The paper develops a CRISPR/Cas9-based gene editing strategy to correct IL7R-SCID in T cells and HSPCs, restoring IL7R expression and T cell development.

Virally induced CRISPR/Cas9-based knock-in of fluorescent albumin allows long-term visualization of cerebral circulation in infant and adult mice

This paper uses CRISPR/Cas9 to knock-in a fluorescent albumin gene in mice, enabling long-term visualization of cerebral circulation in both infant and adult mice.

Metabolic and behavioral alterations associated with viral vector-mediated toxicity in the paraventricular hypothalamic nucleus

This study shows that a specific AAV-Cre preparation causes toxicity in the PVN, leading to metabolic and behavioral changes, independent of genetic background.

The Flexible, Versatile and Fast GreGT Platform of Fully Deleted Helper-Virus Independent Adenoviral Vectors

The paper describes the development of the GreGT platform, a helper-virus-independent adenoviral vector system that enables flexible and efficient production of fully deleted Ad vectors.

Direct Pacbio sequencing methods and applications for different types of DNA sequences

This study evaluates PacBio sequencing for direct, full-length resolution of complex DNA sequences, finding it effective for high GC, poly, and repeat sequences like rAAV without pre-fragmentation.

Development of an optimized, non-stem cell line for intranasal delivery of therapeutic cargo to the central nervous system

The paper develops a non-tumorigenic cell line, LX-2, for intranasal delivery of oncolytic viruses to the brain, showing improved resistance to viral replication and safety in mice.

Precise manipulation of site and stoichiometry of capsid modification enables optimization of functional adeno-associated virus conjugates

This study shows that precise control over the site and stoichiometry of capsid modification in AAV vectors improves retargeting efficiency and activity.

AAV-mediated interneuron-specific gene replacement for Dravet syndrome

This study demonstrates that interneuron-specific AAV-mediated SCN1A gene replacement significantly rescues Dravet syndrome symptoms in mouse models.

An optimised protocol for quality control of gene therapy vectors using Nanopore direct RNA sequencing

This paper compares long-read sequencing technologies to identify RNA quality issues in lentiviral vectors, finding Nanopore direct RNA sequencing most effective for detecting incomplete RNA.

A toolbox of astrocyte-specific, serotype-independent adeno-associated viral vectors using microRNA targeting sequences.

The paper develops a toolbox of astrocyte-specific AAV vectors using miRNA targeting sequences to reduce off-target expression in neurons and endothelial cells.

Adeno-associated virus (AAV2) can replicate its DNA by a rolling hairpin or rolling circle mechanism, depending on the helper virus.

This study compares AAV2 DNA replication mechanisms under HSV-1 and AdV5 helper virus conditions, finding that HSV-1 supports rolling circle replication while AdV5 supports rolling hairpin replication.

Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and non-human primates

This study evaluates the efficacy and safety of AAV1-hOTOF gene therapy in mice and non-human primates for treating DFNB9 hearing loss.

Hidden rAAV Breakpoints Detected Using Single-Molecule, Modified Base Sequencing

The paper detects hidden DNA breakpoints in AAV vectors using modified base sequencing to improve vector design and reduce breakage.

Loss of transgene expression limits liver gene therapy in primates

This study shows that transgene expression in primate liver declines rapidly after initial transduction, even though vector DNA remains in the nucleus, suggesting that genomic integration may be needed for durable expression.

In vivo silencing of regulatory elements using a single AAV-CRISPRi vector

This paper develops an AAV-based CRISPRi system to silence gene expression in cardiac myocytes, showing dose-dependent reduction of Nppa expression in vitro and in vivo.

Genomic stability of Self-inactivating Rabies

The paper investigates the genomic stability of the self-inactivating Rabies virus (SiR) and finds that revertant mutations are rare and do not accumulate in vivo or in high-TEV protease expressing cell lines.

A blood-brain-barrier penetrant AAV gene therapy rescues neurological deficits in mucolipidosis IV mice.

Transgene Expression in Cultured Cells Using Unpurified Recombinant Adeno-Associated Viral Vectors

The paper demonstrates the use of unpurified recombinant adeno-associated viral vectors for transducing cultured cells, showing effectiveness in three previously unreported cell types.