Novel Cytotoxic Vectors Based on Adeno-Associated Virus [PDF]
Toxins, 2010 Vectors based on adeno-associated virus (AAV) are promising tools for gene therapy. The production of strongly toxic vectors, for example for cancer-directed gene transfer, is often unfeasible due to uncontrolled expression of toxic genes in vector ...
Johannes Kohlschütter +2 more
doaj +5 more sources
Comparison of Different Liquid Chromatography-Based Purification Strategies for Adeno-Associated Virus Vectors [PDF]
Pharmaceutics, 2021 Recombinant adeno-associated virus (rAAV) vectors have evolved as one of the most promising technologies for gene therapy due to their good safety profile, high transduction efficacy, and long-term gene expression in nondividing cells.
Ruth Rieser +7 more
doaj +4 more sources
Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases [PDF]
Biomedicines, 2023 Recombinant adeno-associated virus (rAAV) vectors are gene therapy delivery tools that offer a promising platform for the treatment of neurodegenerative diseases. Keeping up with developments in this fast-moving area of research is a challenge.
Leyao Li +11 more
doaj +2 more sources
Genome length determination in adeno-associated virus vectors with mass photometry [PDF]
Molecular Therapy: Methods & Clinical Development, 2023 Recombinant adeno-associated viruses (rAAVs) are attractive therapeutic viral vectors for gene delivery. To ensure the efficacy and safety of rAAV-based therapies, comprehensive characterization of the adeno-associated virus (AAV) capsids is essential ...
Cornelia Hiemenz +7 more
doaj +2 more sources
Modifying immune responses to adeno-associated virus vectors by capsid engineering [PDF]
Molecular Therapy: Methods & Clinical Development, 2023 De novo immune responses are considered major challenges in gene therapy. With the aim to lower innate immune responses directly in cells targeted by adeno-associated virus (AAV) vectors, we equipped the vector capsid with a peptide known to interfere ...
Martin Bentler +13 more
doaj +2 more sources
Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy [PDF]
VirusesGene therapy offers promising potential as an efficacious and long-lasting therapeutic option for genetic conditions, by correcting defective mutations using engineered vectors to deliver genetic material to host cells.
Limor Zwi-Dantsis +3 more
doaj +2 more sources
Retrograde capabilities of adeno-associated virus vectors in the central nervous system [PDF]
BioTechnologia, 2021 Adeno-associated virus (AAV) vectors delivered at the axonal terminals can be retrogradely transported toward neuronal cell bodies throughout the axons.
Magdalena Surdyka, Maciej Figiel
doaj +2 more sources
Capsid-modified adeno-associated virus vectors as novel vaccine platform for cancer immunotherapy [PDF]
Molecular Therapy: Methods & Clinical Development, 2023 Immunotherapy has significantly improved treatment outcomes in various cancer entities. To enhance immunogenicity and efficacy, and to further broaden its applicability, co-administration of anti-tumor vaccines is considered as a promising strategy. Here,
Ann-Christin Franke +10 more
doaj +2 more sources
Adeno-Associated Virus Vectors in Retinal Gene Therapy: Challenges, Innovations, and Future Directions [PDF]
BiomoleculesAdeno-associated virus (AAV) vectors have emerged as the leading platform for retinal gene therapy due to their favorable safety profile, low immunogenicity, and ability to mediate long-term transgene expression within the immune-privileged ocular ...
Jiayu Huang +3 more
doaj +2 more sources
Quantification of Empty, Partially Filled and Full Adeno-Associated Virus Vectors Using Mass Photometry [PDF]
International Journal of Molecular Sciences, 2023 Ruth Birner-Gruenberger
exaly +2 more sources