Virus-based gene therapy research

This study shows that lentiviral vector transduction of airway basal cells does not impair their differentiation or function, suggesting potential for long-term gene therapy in lung diseases.

The paper identifies satellite subgenomic particles (SBGs) as regulators of AAV life cycle by balancing gene expression and packaging efficiency.

This study uses gene editing in hematopoietic stem cells to treat Pyruvate Kinase Deficiency Hemolytic Anemia, restoring RPK function and ATP levels in erythroid cells.

The paper shows Tax rescues HIV-1 replication by recruiting NF-kB to unintegrated viral DNA, enabling transcription and epigenetic changes, and describes effective IN-lentiviral vectors.

This study examines immune responses to AAV8 delivery via suprachoroidal route in nonhuman primates, finding mild inflammation and immune cell infiltration with reduced systemic viral capsid antibody responses.

This paper introduces a transposase-based protocol for nanopore sequencing of ssDNA without second-strand synthesis, demonstrating its utility for characterizing AAV vectors.

The paper identifies enhancers for targeting specific neuronal subtypes across species and demonstrates their utility in circuit manipulation.

This paper introduces a genetic switch called iOn that enables the identification of genomic integration events by triggering gene expression upon insertion, improving transgenesis in stem cells and model organisms.

OvirusTdb is a manually curated repository of oncolytic viruses used in cancer treatment, providing comprehensive information on 5927 virus records and their therapeutic applications.

The paper analyzes polyclonal vector integration sites using Nanopore sequencing to provide a scalable and cost-effective method for tracking gene-modified cells.

This study compares rAAV production methods using different host cells, finding chemically and functionally distinct vectors with differences in PTMs, methylation, and potency.

This study uses AAV-PHP.S to deliver chemogenetic receptors to knee-innervating DRG neurons, showing that inhibitory DREADDs reduce inflammation-induced pain in mice.

The paper describes a method using safe nucleic acid dyes to quickly and reliably quantify Adeno-Associated Virus (AAV) titers.

This study shows AAV2.retro efficiently transports gene cargo retrogradely in rhesus macaque brains, enabling widespread transduction of brain circuits relevant to Huntington's disease.

The paper identifies small herpesvirus latency-associated promoters that enable long-term, pan-neuronal transgene expression in the CNS using AAV vectors.

This study uses directed evolution to create AAV vectors with enhanced gene delivery to the outer retina in primates.

This study demonstrates that AAV1 preferentially spreads transsynaptically through glutamatergic and GABAergic synapses, enabling the labeling of neurons based on their input sources and molecular identities.

This paper demonstrates that gene replacement therapy using AAV-PHP.B vector improves survival and corrects metabolic and behavioral deficits in a mouse model of Leigh syndrome.

The paper shows that helper virus concentrations significantly affect transsynaptic tracing efficiency and nonspecific labeling in monosynaptic tracing experiments.

The paper develops a method using adeno-associated virus vectors to generate somatic-transgenic mice for non-invasive monitoring of signaling pathways in disease models.

The paper identifies enhancer elements that drive subclass-specific gene expression in neocortex across mouse, primate, and human species using comparative epigenetics and AAV-based functional assays.

The paper introduces BRAVE, a method for rational AAV vector evolution that maps peptide binding motifs in vivo using barcoding and clustering.

The paper discusses strategies for targeted neuronal manipulation using adeno-associated virus (AAV) and other technologies, emphasizing best practices and future directions.

The paper identifies LY6A as a novel receptor for AAV-PHP.B vectors, enabling efficient gene delivery across the blood-brain barrier in mice.

The paper develops a barcoded AAV capsid library to identify novel gene therapy vectors with improved transduction efficiency in human islets and hepatocytes.

The paper evaluates clade F AAVs for homologous recombination-based genome editing without DSBs, finding no detectable editing without ZFNs.

The paper develops synthetic promoter AAVs for cell-type-specific gene delivery in mice, non-human primates, and humans, showing 11% of AAVs target neuronal and glial cell types in multiple species.

This paper reports a case of clonal hematopoiesis in a rhesus macaque following lentiviral transduction of HSPC with a strong retroviral promoter, leading to gene overexpression and aberrant splicing.

This paper develops a non-integrating lentiviral gene therapy for refractory focal epilepsy, showing reduced seizure frequency in a rat model.

This study shows that AAV-mediated GRN overexpression in mice causes hippocampal degeneration and T cell infiltration, raising safety concerns for GRN-based CNS gene therapies.