Published in Frontiers for Young Minds
Viruses are small particles that are so tiny they cannot be seen by a regular light microscope. However, viruses can be found almost anywhere you look. Viruses specialize in carrying genetic material into all kinds of cells, including human cells. Many viruses are harmless to humans while others can cause illnesses like the cold, the flu, or COVID-...
High systemic doses of adeno-associated viruses (AAVs) have been associated with immune-related serious adverse events (SAEs). Although AAV was well tolerated in preclinical models, SAEs were observed in clinical trials, indicating the need for improved preclinical models to understand AAV-induced immune responses. Here, we show that mice dual-dose...
Glioblastoma (GBM), the most common primary malignant brain tumor, is a highly lethal form of cancer with a very limited set of treatment options. High heterogeneity in the tumor cell population and the invasive nature of these cells decrease the likely efficacy of traditional cancer treatments, thus requiring research into novel treatment options....
Published in Frontiers for Young Minds
When something is rare it means that it happens very infrequently. Did you know that most diseases are rare? There are more than 6,000 known rare diseases, each affecting fewer than 1 in every 2,000 people. But if we put all the rare diseases together, they affect about 1 in 17 of us! Given that they are individually uncommon, rare diseases are oft...
Crossing the blood-brain barrier in primates is a major obstacle for gene delivery to the brain. Adeno-associated viruses (AAVs) promise robust, non-invasive gene delivery from the bloodstream to the brain. However, unlike in rodents, few neurotropic AAVs efficiently cross the blood-brain barrier in non-human primates. Here we report on AAV.CAP-Mac...
Acknowledgements: C. O. F. would like to acknowledge the funding by the Engineering and Physical Sciences Research Council Centre for Doctoral Training in Sensor Technologies and Applications (EP/L015889/1) as well as the support by AstraZeneca in the framework of Project Beacon. A. B. P. would like to acknowledge funding from the Engineering and P...
With three FDA-approved products, lipid nanoparticles (LNPs) are under intensive development for delivering wide-ranging nucleic acid therapeutics. A significant challenge for LNP development is insufficient understanding of structure-activity relationship (SAR). Small changes in chemical composition and process parameters can affect LNP structure,...
Delivering genes to and across the brain vasculature efficiently and specifically across species remains a critical challenge for addressing neurological diseases. We have evolved adeno-associated virus (AAV9) capsids into vectors that transduce brain endothelial cells specifically and efficiently following systemic administration in wild-type mice...
The success of mRNA vaccines during the COVID-19 pandemic has greatly accelerated the development of mRNA therapy. mRNA is a negatively charged nucleic acid that serves as a template for protein synthesis in the ribosome. Despite its utility, the instability of mRNA requires suitable carriers for in vivo delivery. Lipid nanoparticles (LNPs) are emp...
Cognitive decline is a common pathological outcome during aging, with an ill-defined molecular and cellular basis. In recent years, the concept of inflammaging, defined as a low-grade inflammation increasing with age, has emerged. Infiltrating T cells accumulate in the brain with age and may contribute to the amplification of inflammatory cascades ...
