SHOWING 1-10 OF 11 REFERENCES
Complement activation-related pseudoallergy: a stress reaction in blood triggered by nanomedicines and biologicals.
- Molecular immunology
An analogy is proposed between CARPA and the classic stress reaction, suggesting that CARPA represents a "blood stress" reaction, a systemic fight of the body against harmful biological and chemical agents via the anaphylatoxin/mast-cell/circulatory system axis, in analogy to the body's fight of physical and emotional stress via the hypothalamo/pituitary/adrenal axis. Expand
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
- Cell reports
A pseudoU-modified codon-optimized mRNA encoding human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, was generated and encapsulated into biodegradable lipid nanoparticles (LNPs) and dosing resulted in a 75%-85% reduction in plasma methylmalonic acid. Expand
Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals
- Medicine, Biology
- Molecular therapy : the journal of the American Society of Gene Therapy
It is shown that chemically unmodified mRNA can achieve sufficient protein expression and avoid activation of the innate immune system by applying sequence-engineered molecules, and has the potential to revolutionize human protein therapies. Expand
Toxicological considerations when creating nanoparticle-based drugs and drug delivery systems
- Expert opinion on drug metabolism & toxicology
This review examines and details the toxicological aspects that should be considered when planning to use nanoparticles in animals or in man for drug delivery or imaging and improves methods for assessing nanoparticle toxicity. Expand
Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge
- Nature communications
The translated antibody from a single injection of VRC01 mRNA protects humanized mice from intravenous HIV-1 challenge, demonstrating that nucleoside-modified mRNA represents a viable delivery platform for passive immunotherapy against HIV- 1 with expansion to a variety of diseases. Expand
Zika virus protection by a single low dose nucleoside modified mRNA vaccination
It is demonstrated that a single low-dose intradermal immunization with nucleoside-modified mRNA–LNP elicits rapid and durable protective immunity and therefore represents a new and promising vaccine candidate for the global fight against ZIKV. Expand
Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA.
- Biology, Medicine
It is concluded that nucleoside modifications suppress the potential of RNA to activate DCs, and the innate immune system may detect RNA lacking nucleosides modification as a means of selectively responding to bacteria or necrotic tissue. Expand
Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA.
- Biology, Medicine
- Cell stem cell
It is shown that this approach can reprogram multiple human cell types to pluripotency with efficiencies that greatly surpass established protocols and represents a safe, efficient strategy for somatic cell reprogramming and directing cell fate that has broad applicability for basic research, disease modeling, and regenerative medicine. Expand
- Am. J. Managed Care
Bright idea: Derrick Rossi created mRNA encoding bioluminescent protein
- Nat. Commun