RNA therapeutics, such as mRNA, siRNA, and CRISPR–Cas9, present exciting avenues for treating diverse diseases. However, their potential is commonly hindered by vulnerability to degradation and poor cellular uptake, requiring effective delivery systems. Lipid nanoparticles (LNPs) have emerged as a lea...
Insufficient accumulation of lipid nanoparticles (LNPs)‐based mRNA vaccines in antigen presenting cells remains a key barrier to eliciting potent antitumor immune responses. Herein, we develop dendritic cells (DCs) targeting LNPs by taking advantage of mannose receptor‐mediated endocytosis....
mRNA requires safe, effective and stable delivery systems that protect the nucleic acid from degradation and that allow cellular uptake and mRNA release. Lipid nanoparticles have successfully entered the clinic for the delivery of mRNA; in particular, ...
encapsulation of nanoparticles into KGS-NE. Initially, the EDX spectrum, SAED pattern, and EDX were utilized to identify the presence of nanoparticles. We observed the highest Au element peak at 2.1 keV with multiple points and the appearance of a Cu peak at 8 keV was attributed to the coppe...
nanoparticles produced a five-fold improvement in the delivery of mRNA to liver endothelial cells [185]. Similarly, Pattipeiluhuet al. reported that replacement of neutral DSPC with anionic DSPG in the formulation of patisiran nanoparticles significantly enhanced transfection of liver endothelial cells ...
Sahay and fellow researchers including Kelvin MacDonald, an OHSU physician who treatscystic fibrosis patients, successfully tested the mRNA-loaded nanoparticles in culture with patient-derived bronchial epithelial cells and also in mice that had had their CFTR gene knocked out. ...
The use of lipid nanoparticles as a drug delivery approach improves the payloads' stability, pharmacokinetics, bio-distribution and therapeutic benefit while reducing side effects. Moreover, a wide variety of targeting moieties allow a precise and modular manipulation of gene expression, together with ...
Here, we show that intravenously delivered human OTC (hOTC) mRNA by lipid nanoparticles (LNP) was an effective treatment for OTCD by restoring the urea cycle. We observed a homotrimer conformation of hOTC proteins produced by the mRNA-LNP in cells by cryo-electron microscopy. The ...
Liposomes and lipid nanoparticles are common lipid-based drug delivery systems and play important roles in cancer treatment and vaccine manufacture. Although significant progress has been made with these lipid-based nanocarriers in recent years, efficien
Liposomes and lipid nanoparticles are common lipid-based drug delivery systems and play important roles in cancer treatment and vaccine manufacture. Although significant progress has been made with these lipid-based nanocarriers in recent years, efficien