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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.
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.
The simultaneous removal of endogenous T-cell receptor α-chains and β-chains, and the orthotopic placement of an exogenous receptor in human T cells via CRISPR gene-editing, prevents the mispairing between endogenous and transgenic receptors while preserving the cells’ function.
For genome-wide screens and other applications that require the processing of a large number of cells, the immunomagnetic sorting of cells on a microfluidic chip is a scalable, rapid and cost-efficient alternative to fluorescence-activated cell sorting.
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.
A suite of microelectrodes integrated into glass pipettes, for the simultaneous recording of intracellular and extracellular data in the brains of living mice and rats, yields ground-truth data for the validation of the performance of spike-sorting algorithms.
A viscoelastic adhesive cardiac patch with optimal mechanical behaviour, determined using a computational model, restores heart function and slows down pathological remodelling following myocardial infarction in rodents.
Smartphone-controlled optofluidic neural implants with replaceable and replenishable plug-like drug cartridges enable the selective wireless manipulation of brain circuits in rodents via chronic pharmacology and photostimulation.
The localization of target proteins, at subcellular resolution, in fixed patient-derived tissues can now be achieved via antibodies conjugated with tetrahedral DNA nanostructures self-assembled in situ.
Modelling human tissues in microphysiologically relevant ‘chips’ will increasingly help to unravel mechanistic knowledge underlying disease, and might eventually accelerate the productivity of drug development and predict how individual patients will respond to specific drugs.
The safety and security of medical devices driven by software, the software-development processes, and the need for data collection and privacy, all offer challenges and opportunities for device regulation and clinical care.
Plasmids coding for a toxin gene that is only expressed in the presence of a virulence-associated transcription factor lead to the killing of only the virulent form of the bacterium Vibrio cholerae in a mixed bacterial population.
An electrical biosensor that relies on the binding of target nucleic acid sequences to Cas9 immobilized on a graphene field-effect transistor enables the rapid detection of mutations in purified samples without the need for nucleic acid amplification.
Early-stage ovarian cancer can be detected in few-microlitre plasma samples via a microfluidic chip, patterned with nanoporous herringbone structures so as to enhance the capture of extracellular vesicles from the samples.