Virus-based gene therapy research

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

This study develops two new AAV9 variants with improved brain targeting and reduced liver off-targeting by modifying the galactose-binding domain and displaying peptides.

This paper edits introns of HSPCs to restrict transgene expression to the myeloid lineage, demonstrating efficacy in treating metabolic diseases and reaching the brain.

The paper assesses T cell immunity to various AAV serotypes in healthy donors, finding AAV9 has the highest prevalence of pre-existing cellular immunity.

The paper develops a CRISPR-based assay to rapidly and accurately quantify AAV genome titers, enabling efficient manufacturing of rAAV vectors.

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

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

This study shows that the minute virus of mice (MVMp) selectively targets and kills mesenchymal, basal-like pancreatic cancer cells, both in vitro and in vivo, offering a potential precision therapy for aggressive pancreatic tumors.

This study presents cryo-EM structures of HAdV-5C hexon and its complex with human lactoferrin, revealing molecular interactions critical for viral entry.

This paper develops a chimeric oncolytic adenovirus that evades neutralizing antibodies and shows improved anti-glioma efficacy in mice.

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

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.

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.

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 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.

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.

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.

This study evolves MAAP variants to enhance AAV production, finding that selected variants improve vector genome packaging in multiple AAV serotypes.

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

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

This study identifies host restriction factors critical for rAAV2.5T transduction of polarized human airway epithelium, revealing KIAA0319L's role in transduction but not internalization, and WDR63's role in transduction without involvement in internalization or nuclear entry.

The paper engineers a durable AAV receptor for analyzing AAV-AAVR interactions, showing diverse binding affinities among AAV serotypes and enabling quality checks and purification of AAV vectors.

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

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.

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.

The paper inserts large protein domains into AAV capsid protein VP1 to identify engineerable hotspots and assess capsid robustness to structural modifications.

This paper uses the chicken chorioallantoic membrane model to isolate a CRISPR/Cas9-modified HSV-1 mutant expressing tumor suppressor p53, showing its potential for oncolytic virus development.

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

This study evaluates neoadjuvant oncolytic virus therapy in dogs with osteosarcoma, finding it safe and associated with increased long-term survivorship and immune gene expression.

This study demonstrates that self-complementary AAV1 with a strong promoter can enable recombinase-independent anterograde transsynaptic labeling in neural circuits.