Virus-based gene therapy research

The paper investigates leaky expression of 3' transgenes in bicistronic vectors and shows that 5' transgenes can drive promoter-like activity, leading to Cre-independent expression.

The paper develops all-in-one virus-like particles for co-delivering Cas9 mRNA and single guide RNA and improves lentiviral vectors with aptamers for better gene expression.

The paper introduces a DIO/USS system to enhance Cre-dependent transgene expression specificity by preventing Cre-independent recombination during vector production.

This paper introduces third-generation rabies viral vectors with single deletions that enable more efficient, non-toxic retrograde targeting of projection neurons compared to previous generations.

The paper develops a method to label neurons receiving monosynaptic inputs from two upstream regions in mice using intersectional gene expression and AAV1.

The paper improves the production of CVS-N2c-ΔG virus for retrograde tracing, showing higher efficiency and broader application in neural circuit studies.

The paper compares viral strategies for targeting locus coeruleus norepinephrine neurons, finding variability in transgene expression and no behavioral differences in cre-expressing mice.

The paper evaluates AAV serotypes 1, 5, and 8 for transducing astrocytes in the rat brain cortex with an optogenetic construct and finds serotype 8 has the highest transduction efficiency.

The paper identifies and validates two novel human genomic sites for safe and predictable gene expression in therapeutic applications.

The paper evaluates two engineered AAV capsid variants for transducing human cortical neurons derived from iPSCs, finding that AAV2-NNPTPSR efficiently transduces both glutamatergic and GABAergic neurons, while AAV9-NVVRSSS also transduces astrocytes.

This paper introduces ENTER-seq, a method using engineered lentiviral particles to systematically discover receptor-ligand interactions and map TCR specificity in single cells.

This study shows that gene therapy using an AAV vector to restore WWOX expression in neurons rescues multiple neurological defects in Wwox-null mice.

The paper investigates off-target expression of Cre-dependent AAVs in wild-type mice and finds extensive Cre-independent viral expression despite vendor, serotype, and promoter variations.

This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.

This study shows that juvenile AAV9-MCT8 gene therapy improves neurological and behavioral deficits in an AHDS mouse model by restoring TH levels and gene expression.

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

The paper identifies a microglia-specific promoter of the human HexB gene that can be used for AAV gene therapy targeting microglia in the CNS.

This study shows that AAV triggers dendritic complexity loss in the cortex via TLR9, independent of capsid or transgene, and identifies TLR9 inhibitors as potential safety improvements.

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 shows that using a secretable TATk-CDKL5 protein in AAV gene therapy improves CDKL5 replacement and functional outcomes in Cdkl5 KO mice compared to native CDKL5 expression.

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.

The paper develops a method to measure transduction efficiency and specificity of viral vectors in complex cell populations using single-cell RNA sequencing.

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 machine learning to predict AAV capsid mutations that improve production fitness, finding threefold more effective mutations than random.