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Efficient approaches that overcome the physicochemical and biological barriers to getting therapeutics into the right tissues and cells are critical to therapy development.
The partial depletion of red blood cells via the administration of a low dose of anti-erythrocyte antibodies increases the circulation half-life of nanomedicines, as shown in rodent models of cancer.
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.
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.
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.
Large quantities of extracellular vesicles produced via cellular nanoporation, and loaded with endogenously transcribed therapeutic mRNAs and targeting peptides, boost therapeutic outcomes in vivo.
Translational cancer nanomedicine needs to increasingly exploit newly discovered tumour-targeting strategies as well as the further optimization of proven means to selectively increase the concentration of cytotoxic drugs in solid tumours.
Encapsulation of the therapeutic monoclonal antibody rituximab, and its molecular targeting to brain metastases of B-cell lymphoma, significantly boosts the antibody’s therapeutic effectiveness in mice.
Drug-loaded nanoparticles that specifically bind to phages infecting cancer-promoting bacteria found in colonic tumours augment the effects of chemotherapy against colorectal cancer in mice.
Widespread editing of the mutated DMD gene by CRISPR–Cas9, systemically delivered via an adeno-associated virus, restores dystrophin expression in a canine model of Duchenne muscular dystrophy.
The coupling of blood platelets bearing anti-programmed cell death protein 1 antibodies to haematopoietic stem cells enables delivery of checkpoint-blockade therapy to bone marrow to promote T-cell-mediated control of leukaemia in mice.
Two drug-loaded nanoparticle formulations that preferentially accumulate within tumour-associated macrophages induce macrophage repolarization to a tumoricidal state that leads to potent antitumour activity in multiple murine models of cancer.
Polymer-coated gold nanoparticles carrying the CRISPR components for knocking out, in the striatum of adult mice with fragile X syndrome, a gene implied in the syndrome’s pathophysiology rescue the mice from the exaggerated repetitive behaviours characteristic of the syndrome’s phenotype.
When designing translationally relevant delivery strategies to overcome the physicochemical and biological barriers to getting therapeutics into the right tissues and cells, building on tried-and-tested concepts often pays off.
Self-assembled weakly negative nanoparticles bearing an octet of short interfering RNAs, targeting ligands and endosomolytic peptides lead to efficient gene silencing in a mouse model of prostate cancer.
The sustained delivery of extracellular vesicles, secreted by induced-pluripotent-stem-cell-derived cardiomyocytes, through a hydrogel patch promotes cardiac recovery after myocardial infarction in rats.
Micelles of a superhydrophilic zwitterionic polymer covalently linked to a superhydrophobic lipid remain stable at extremely dilute conditions and enhance the delivery of hydrophobic chemotherapeutics in vivo.
Topically applied spherical nucleic acids targeting an intracellular mRNA biomarker associated with abnormal scarring enable the fluorescent detection of abnormal scars during wound healing.
Nanoparticles carrying an antibiotic and bearing a peptide that targets the bacterium Staphylococcus aureus effectively suppress staphylococcal infections in mice.
Grafting platelet-derived nanovesicles onto the surface of cardiosphere-derived cardiac stem cells enables the cells to better engraft in infarcted tissue following systemic injection in rats and pigs.
Prolonged local delivery, via an injectable hydrogel, of a miRNA known to induce cardiomyocyte proliferation stimulates the recovery of mice from myocardial infarction.
The DNA mutation that causes Duchenne muscular dystrophy in mice can be corrected, with minimal off-target effects, by gold nanoparticles carrying the CRISPR components.
A dendrimer that depletes bioavailable copper as a result of its internal make-up displays powerful anticancer activity in mice, and no observable adverse effects.
Exosomes expressing CD47 and loaded with interfering RNA dodge phagocytosis and accumulate in pancreatic tumours to silence the expression of the oncogene Kras in mice, with remarkable therapeutic efficacy.
A subcutaneous depot of a diabetes drug fused to a thermosensitive biopolymer leads to blood-glucose control, for over one week after a single injection, in animal models of type-2 diabetes.
A microfluidic device that integrates mechanical squeezing and electrical stimulation delivers DNA to the nucleus of cells at a rate of millions of cells per minute.
Platelets delivering the immunotherapeutic antibody anti-PD-L1 to the site of surgically removed tumours reduce cancer recurrence and metastatic spread in mice.
Conjugation of a diabetes drug with a brush polymer reduces the reactivity of the drug conjugate towards pre-existing polymer antibodies in human plasma and improves the drug's performance in diabetic mice.