Virus-based gene therapy research

Multi-omics analysis reveals integrin α3-dependent mechanisms of Zika virus oncolytic activity in pediatric neural tumor

Efficient NK cell transduction with VSV-G-pseudotyped lentiviral vectors

This study optimizes VSV-G-pseudotyped lentiviral transduction of NK cells by improving activation, construct design, and enhancer use, achieving high efficiency without compromising cell function.

Pancreatic tumor microenvironment reprogramming via alloantigen-expressing virotherapy elicits tumor rejection and improves immunotherapy response

Oncolytic virus-based delivery of an alloantigen improves antitumor immunity and synergizes with immune checkpoint inhibitors in pancreatic cancer.

AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome

This paper demonstrates the use of AAV9 vectors to deliver AntagoNATs targeting a natural antisense transcript in a DS mouse model, improving survival and reducing seizures.

Mapping AAV capsid sequences to functions through function-guided in silico evolution

The paper introduces ALICE, a platform that uses function-guided in silico evolution to design AAV capsids with tailored properties, demonstrating improved targeting and tropism in mice.

Defining a Midgestational Window for In Utero Genome Editing of the Fetal Murine Cortex

The paper establishes a method for in utero genome editing in fetal mouse cortex at midgestation, demonstrating efficient editing with reduced off-target effects.

Technical and biological sources of noise confound multiplexed enhancer AAV screening

The paper presents a barcoded multiplex strategy to screen enhancer AAVs using single-cell RNA sequencing and taxonomy mapping, but finds significant technical and biological noise complicates accurate expression pattern analysis.

Hepatic gene replacement restores energy metabolism and doubles the survival in mouse model of GRACILE syndrome, a neonatal mitochondrial disease

This study uses gene replacement in a mouse model of GRACILE syndrome to show improved energy metabolism and doubled survival through liver-specific correction of a mitochondrial complex III deficiency.

Identification of AAV Capsids with Enhanced Intravitreal Transduction and Favorable Safety Profile in Non-Human Primates

The paper identifies AAV capsid variants with improved intravitreal transduction and safety in non-human primates.

Cardiac-Detargeted MyoAAV Enables Systemic Nrl-Mediated Fast Myofiber Remodeling and Hypertrophy Across Multiple Skeletal Muscles

This paper develops a modified MyoAAV vector that detargets cardiac expression, enabling safe systemic induction of fast myofiber remodeling and hypertrophy in skeletal muscles.

Combination of engineered cell type-specific promoters and a high-efficiency AAV capsid restores hearing in adult DFNB1 mice model with demonstrated safety in nonhuman primate

This paper demonstrates hearing restoration in adult DFNB1 mice using engineered promoters and an AAV capsid, with safety confirmed in nonhuman primates.

Recombinant measles virus equipped with BNiP3, a pro-apoptotic gene, targets β-catenin pathway in triple negative breast cancer cells

This paper investigates how a recombinant measles virus with BNiP3 reduces β-catenin expression and downstream targets, inhibiting migration in triple-negative breast cancer cells.

Controlled linking of AAV capsids enables coordinated multi-vector delivery

This paper introduces a method to link AAV capsids for coordinated delivery of genetic cargo, improving efficiency and uniformity in gene delivery.

CHARIOT-AAV: Conjugation of diverse vectors to adeno-associated viruses for delivery of large genes

CHARIOT-AAV conjugates diverse vectors to AAVs to enable tissue-specific delivery and expanded cargo capacity for large genes.

Optimization of AAV tools to target Müller glial cells for retinal gene therapy

This study optimizes AAV serotypes and promoters to specifically target Müller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.

SG33, a vaccine strain of myxoma virus with oncolytic potential, exploits macropinocytosis and clathrin-mediated endocytosis for entry into pancreatic cancer cells.

This study identifies the entry mechanisms of SG33, an oncolytic myxoma virus, into pancreatic cancer cells, showing it uses macropinocytosis and clathrin-mediated endocytosis.

MuteFree: A novel AAV vector system featuring mutation-free ITRs

MuteFree eliminates ITR mutations in AAV transfer plasmids, achieving 0% mutation rates and resolving production issues in GMP manufacturing.

Ultra-large targeted DNA integrations in primary human cells

This paper identifies methods to achieve ultra-large DNA integrations (>10 kb) in human cells with high efficiency using optimized templates and delivery strategies.

Neuritin1 Cis-Regulatory Elements Enable Gene Expression Preferentially in Retinal Ganglion Cells

The paper identifies Neuritin1 cis-regulatory elements that enable efficient and injury-resilient gene expression specifically in retinal ganglion cells using AAV vectors.

Enabling CAR-T Cell Immunotherapy in Glioblastoma by Modifying Tumor Microenvironment via Oncolytic Adenovirus Encoding Bispecific T Cell Engager

This paper demonstrates that combining oncolytic adenovirus encoding bispecific T cell engager (OV-BiTE) with CAR-T cells enhances T cell recruitment and tumor eradication in glioblastoma xenograft models.

AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a mouse model of congenital generalised lipodystrophy

This study evaluates AAV-mediated delivery of leptin and adiponectin in a mouse model of congenital generalized lipodystrophy, finding that leptin improves metabolic health while adiponectin does not.

Precision DNA Impurity Reduction Approaches for Ultra-Pure rAAV Manufacturing

The paper evaluates DNA impurity reduction strategies in rAAV manufacturing, finding that TelN/TelROL and I-SceI reduce plasmid backbone DNA to 20-40%, CRISPR/Cas9 to 10-20%, and Cre/LoxP eliminates it without affecting vector titers.

Swine reporter model for preclinical evaluation and characterization of gene delivery vectors

This paper develops a swine reporter model to evaluate gene delivery vectors in a large animal system and demonstrates its utility for assessing biodistribution, safety, and administration methods.

Achieving cell-type specific transduction with adeno-associated viral vectors in pigeons

This study validates adeno-associated viral vectors for precise gene expression in pigeons, demonstrating cell-type-specific transduction using promoters and enhancers.

Novel Engineered AAV Variants Demonstrate Superior Blood-Brain Barrier Penetration and Safety in Non-Human Primates

The paper develops novel AAV variants with improved BBB penetration and safety in NHPs, showing higher brain transduction and reduced organ toxicity.

Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle-Adeno-Associated Virus Vectors

The paper develops an assay to distinguish pseudotransduction from true transduction in EV-AAV vectors, revealing cell-type-specific effects of EV modifications on transduction efficiency.

Architecture Matters: Design Rules for Multigene IDO1/PD L1 Cassettes in Human Skin Cells

This study identifies design rules for multigene cassettes in human skin cells to avoid promoter interference and improve immunomodulatory function.

Tumor-Intrinsic IL-17 Signaling Correlates with Multimodal Resistance Phenotypes Following Oncolytic Adenovirus Challenge

This study identifies IL-17 signaling as a tumor-intrinsic factor associated with resistance to oncolytic adenovirus therapy, linked to stemness, metabolic changes, and reduced cell death.

Comprehensive investigation of AAV tropism across human iPSC-derived neuronal subtypes

This study evaluates AAV transduction efficiency and toxicity across human iPSC-derived neuronal subtypes, identifying optimal serotypes for gene delivery in vitro.

Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids

The paper solves cryo-EM structures of AAV capsids and their complexes with AAVR to design variants with reduced liver targeting and altered brain cell tropism.