Talk
rAAV meets ioNeurons: Pioneering the future of translational neurotherapies
Dr Martina Esposito Soccoio, AviadoBio, presented a study evaluating rAAV vector delivery in ioGlutamatergic Neurons. Learn how she established a robust in vitro model for translational gene therapy research.
Dr Martina Esposito Soccoio, AviadoBio, presented a study evaluating rAAV vector delivery in ioGlutamatergic Neurons. Learn how she established a robust in vitro model for translational gene therapy research.
Recombinant adeno-associated virus (rAAV) vectors are widely used tools in gene therapy and neuroscience research, owing to their ability of efficiently transducing a broad range of cell types with low immunogenicity and sustained gene expression. Human-induced pluripotent stem cells (hiPSCs) offer a dynamic and reliable platform with broad differentiation capacity that can recapitulate disease-specific cellular phenotype, making them highly suitable for modelling complex diseases, testing therapeutics, and developing regenerative medicine applications. We conducted a series of experiments to evaluate the efficacy of rAAV vectors for gene delivery in ioGlutamatergic Neurons.
More specifically, we tested multiple rAAV serotypes to optimize transduction efficiency and conditions. Importantly, this was accomplished without compromising neuronal viability, morphology or development establishing a reliable cell model for translational studies and a robust platform for screening rAAV vector candidates prior to in vivo evaluation. Overall, our findings demonstrate the importance of hiPSCs technology in assessing rAAV-based gene delivery. In doing so, we provide a reliable, consistent and disease-relevant system for identifying therapeutic targets and testing gene-based interventions. In addition, this integrated approach holds significant promise for accelerating translational research in neurodegeneration and expanding clinical applications based on gene therapy for many devastating nervous system disorders.
More specifically, we tested multiple rAAV serotypes to optimize transduction efficiency and conditions. Importantly, this was accomplished without compromising neuronal viability, morphology or development establishing a reliable cell model for translational studies and a robust platform for screening rAAV vector candidates prior to in vivo evaluation. Overall, our findings demonstrate the importance of hiPSCs technology in assessing rAAV-based gene delivery. In doing so, we provide a reliable, consistent and disease-relevant system for identifying therapeutic targets and testing gene-based interventions. In addition, this integrated approach holds significant promise for accelerating translational research in neurodegeneration and expanding clinical applications based on gene therapy for many devastating nervous system disorders.