TL;DR
The history and evolution of various baculovirus systems for rAAV production are outlined and the most modern and promising platforms for assembling recombinant AAV particles are described.
This review provides a comprehensive overview of the current status of viral and non-viral vector systems for in vivo and ex vivo applications, and key comparisons are made across safety, efficacy, scalability, and immune responses.
A 140% increase in pHelper plasmid production is reported, while the most successful ITR variant increased pTransgene plasmid yields by 57% and transgene expression by 28%, without reducing rAAV yields or transduction efficiency.
AAV vector production with robust plasmid DNA in cellulo replication (AAVPCR) proves effective across serotypes and transgenes, offering broad potential to improve the safety, efficacy, and affordability of AAV gene therapies.
Recombinant adeno-associated virus (rAAV) vectors are widely used for gene delivery but show limited efficiency in immune cells, including T lymphocytes and natural killer (NK) cells. To overcome this barrier, we have developed a CD7-targeted rAAV vector (CD7-AAV6/9) featuring a nanobody-fused hybrid capsid derived fro...
A detailed protocol for the production of high-titer, third-generation LVs with a dual-expression cassette for Cas13d and a customizable guide RNA is described, which can be used for stable transduction and efficient RNA knockdown in a broad range of mammalian cell types.
A streamlined three-dimensional spheroid platform optimized for evaluating AAV potency, transgene expression kinetics, and serotype-specific transduction efficacy across diverse cell lines is described and establishes its utility as a scalable and physiologically relevant system for preclinical gene therapy evaluation...
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