A5032169917- RNA Interference and Gene Delivery
- Extracellular vesicles in disease
- Cell Adhesion Molecules Research
- MicroRNA in disease regulation
- Advanced biosensing and bioanalysis techniques
- CRISPR and Genetic Engineering
- Medical and Health Sciences Research
- Law and Political Science
- Neuroinflammation and Neurodegeneration Mechanisms
Karolinska Institutet
2021-2023
Extracellular vesicles (EVs) are gaining ground as next-generation drug delivery modalities. Genetic fusion of the protein interest to a scaffold with high EV-sorting ability represents robust cargo loading strategy. To address paucity such proteins, we leverage simple and reliable assay that can distinguish intravesicular proteins from surface- well non-vesicular compare potential 244 candidate proteins. We identify 24 conserved abilities across five types producer cells. TSPAN2 TSPAN3...
The toolbox for genetic engineering has quickly evolved from CRISPR/Cas9 to a myriad of different gene editors, each with promising properties and enormous clinical potential. However, major challenge remains: delivering the CRISPR machinery nucleus recipient cells in nontoxic efficient manner. In this article, we repurpose an RNA-delivering cell-penetrating peptide, PepFect14 (PF14), deliver Cas9 ribonucleoprotein (RNP). RNP-CPP complex achieved high editing rates, e.g., up 80% HEK293T...
Abstract The therapeutic and research potentials of oligonucleotides (ONs) have been hampered in part by their inability to effectively escape endosomal compartments reach cytosolic nuclear targets. Splice-switching ONs (SSOs) can be used with endosomolytic small molecule compounds increase functional delivery. So far, development these has hindered a lack high-resolution methods that correlate SSO trafficking activity. Here we present in-depth characterization two novel using combination...
ABSTRACT Extracellular vesicles (EVs) are gaining ground as next-generation drug delivery modalities. Genetic fusion of the protein interest to a scaffold with high EV-sorting ability represents robust cargo loading strategy. To address paucity such proteins we conducted large-scale comparative study involving 244 candidate proteins. Their potential was evaluated using simple but reliable assay that can distinguish intravesicular from surface and non-vesicular Notably, 24 conserved abilities...