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
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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
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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
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Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Frontiers in Immunology, 2020 Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer. However, the high homology with the parental wild-type virus, which often
Giuseppe Ronzitti +2 more
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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
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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
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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
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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
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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
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Molecular Therapy: Methods & Clinical Development, 2022 Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgensma®,
Makoto Horiuchi +10 more
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