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The preclinical performance of subretinal or intracorneal delivery of Cas9 nucleases encoded in RNA foreshadows safer and effective one-and-done gene therapies for eye diseases.
By using CRISPR and transposon constructs, pigs have been genetically modified to inactivate endogenous retroviruses and to enhance the compatibility of their organs with the human immune and coagulation systems.
A polyvalent STING agonist prolongs the activation of innate-immunity pathways through the formation of STING condensates, and leads to synergistic therapeutic outcomes in vivo when combined with the STING ligand cGAMP.
A deep learning model trained on raw pixel data in hundreds of thousands of echocardiographic videos for the prediction of one-year all-cause mortality outperforms clinical scores and improves predictions by cardiologists.
Input–output models can predict multiregional brain network dynamics in response to temporally varying patterns of ongoing direct electrical stimulation, with variabilities in prediction accuracy explained by at-rest functional connectivity.
A neuroprosthetic interface comprising a muscle actuator coupled to a natively pedicled skin flap in a cuff-like architecture elicits graded contact and vibratory afferent signals analogous to those elicited by mechanical stimulation of intact skin.
The adsorption of coagulation factors on microspheres placed within extracorporeal blood-purification devices allows for transient blood thinning and faster recovery of haemostasis.
Cytidine base editors delivered to the liver of mice with phenylketonuria via adeno-associated viruses or lipid nanoparticles do not lead to detectable off-target edits in the RNA and DNA of hepatocytes.
The DNA methylation of the promoter region of the oncogenic Yes-associated protein is reversibly regulated by the stiffness of the extracellular matrix.
The capacity of mesenchymal stromal cells to promote vascular regeneration in vivo can be enhanced by the combination of specific small-molecule inhibitors and the right waveform of mechanical stretch.
A minimally invasive microneedle patch detects specific protein biomarkers in dermal interstitial fluid at high sensitivity via a needle-integrated immunoassay coupled with an ultrabright fluorescent label.
A microneedle patch that samples and quantifies target protein biomarkers in interstitial fluid allows for longitudinal monitoring of the levels of a range of disease-relevant biomarkers, as shown in live mice.
Human mesenchymal stem cells can be mechanically and pharmacologically conditioned to enhance vascular regeneration in vivo, as shown in mice with hindlimb ischaemia.
Mimicking the molecular architecture of channel-forming membrane proteins of a target microbe can be used to design host defence peptides that specifically target a particular pathogen, as shown here for Mycobacterium tuberculosis.
Lentiviruses co-packaging SpCas9 mRNA and an expression cassette encoding for a guide RNA targeting the Vegfa gene prevent mice from developing wet age-related macular degeneration induced by Vegfa. [Summary amended to correct the description of the packaged cargo.]
A mathematical model of the time course of tumour responses to immunotherapy predicts tumour burden and treatment sensitivity across cancer types and drug combinations.