Virus-based gene therapy research

The paper identifies MAAP as a novel AAV egress factor that facilitates secretion of AAV particles through Rab11+ vesicles and EV markers.

The paper optimizes AAV2/6 for microglial targeting in the retina, showing enhanced transduction during photoreceptor degeneration through capsid modification and Cre-dependent cassette validation.

ExBoX uses Boolean exclusion logic to achieve specific gene expression in neurons by combining activating and inhibiting recombinases, demonstrated in vitro and in vivo.

This study optimizes AAV6 transduction for ex vivo genome editing of human lymphocytes, achieving higher editing rates with reduced vector usage and toxicity.

This study improves AAV vector genome packaging efficiency by engineering Rep hybrid proteins, reducing empty capsids and increasing full capsid yields across non-AAV2 serotypes.

This study shows that rAAV causes dose-dependent cell death in adult murine hippocampal NPCs and DGCs, with no recovery of neurogenesis at 3 months.

This study develops a high-throughput method to engineer 5' UTRs that enhance protein expression in non-viral gene therapies, identifying synthetic 5 UTRs that outperform existing plasmids.

The paper introduces NEPTUNE, an in utero nanoinjection method to study gene function in mouse embryos, and shows that Sptbn2 silencing causes neural tube defects and embryo resorption.

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.