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Negatively charged nanoparticles of about 50 nm in size permit the oral delivery of insulin and other peptide drugs by temporally enhancing the permeability of the intestinal wall.
In an adult mouse model of tyrosinaemia, a base editor correcting an A-to-G splice-site mutation in the Fah gene restores the translation of the functional enzyme, promoting the repopulation of the liver with the corrected cells.
An injectable biomaterial vaccine encapsulating antigens associated with acute myeloid leukaemia, dendritic-cell-targeting pro-inflammatory cytokines and an adjuvant protects mice from the disease.
The therapeutic dose of small interfering RNA can be reduced by endogenously expressing and packaging the RNA into extracellular vesicles through its integration with the backbone of a highly enriched pre-microRNA.
Large quantities of extracellular vesicles produced via cellular nanoporation, and loaded with endogenously transcribed therapeutic mRNAs and targeting peptides, boost therapeutic outcomes in vivo.
The overexpression of the transcription factor c-Jun improves chimeric-antigen-receptor T-cell functionality and enhances the killing of low-antigen-expressing liquid and solid cancers in mice.
