To facilitate model-informed drug development (MIDD) of adeno-associated virus (AAV) therapy, here we have developed a physiologically based pharmacokinetic (PBPK) model for AAVs …

With the recent difficulties in selecting safe and effective doses for AAV-based gene therapies seen clinically, PBPK models allowing for in-depth exploration of AAV pharmacology could be …

2024年1月1日 · Unprecedented PK data and the novel PBPK model developed here provide the foundation for quantitative systems pharmacology (QSP) investigations of AAV-mediated gene …

2021年3月1日 · The most promising variant, AAV-PHP.B, delivered more than 40-fold AAV genomes to the CNS compared to AAV9 and transduced >50% of all neurons and astrocytes …

2023年7月31日 · Interest and efforts to use recombinant adeno-associated viruses (AAV) as gene therapy delivery tools to treat disease have grown exponentially. However, gaps in …

2023年7月31日 · When characterizing the PK of rAAV-mediated gene therapy, the vector and/or transgene DNA, transgene mRNA, and protein are the main analytes of interest and can …

Pathway analysis of the PBPK model suggested that liver, muscle, and heart are the main contributors for the secreted transgene mAb. Unprecedented PK data and the novel PBPK …

Download scientific diagram | Example schematic of systemic AAV PBPK distribution with suggested applicable compartments based on AAV tropism and frequently targeted tissues of …

2023年10月17日 · Example schematic of systemic AAV PBPK distribution with suggested applicable compartments based on AAV tropism and frequently target tissues of AAV therapy. …

2024年1月1日 · To facilitate model-informed drug development (MIDD) of adeno-associated virus (AAV) therapy, here we have developed a physiologically based pharmacokinetic (PBPK) …