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Initial results from phase I/II lentiviral gene therapy trials provide early evidence supporting its safety and efficacy in treating patients with X-linked chronic granulomatous disease.
CRISPR–Cas9-mediated gene editing restores dystrophin expression in both pig and human induced pluripotent stem cell models of Duchenne muscular dystrophy, with beneficial effects on skeletal muscle and cardiac function.
iPSC-derived midbrain dopaminergic neurons from patients with young-onset Parkinson’s disease exhibit molecular abnormalities, including elevated α-synuclein and lysosomal dysfunction, which can be normalized with phorbol ester treatment.
Inactivating mutations in a protein kinase, NEK10, cause a genetic bronchiectasis syndrome in humans that is characterized by short motile cilia and impaired mucociliary transport.
In a first-in-human, phase I trial in patients with B-cell lymphoma,
CD19 CAR T cells with fully human binding domains exhibit lower neurologic toxicity,
but similar clinical activity, to previously tested CD19 CAR T cells with murine
binding domains.
A newly developed PET tracer allows visualization of AMPA receptors in the living human brain, providing a new tool to study their potential involvement in neurological or psychiatric disorders.
Most infants with cystic fibrosis have poor early linear growth in their first year despite nutritional supplementation and treatment. Intestinal dysbiosis in these infants is associated with low length, suggesting a path for intervention.
Although examples of algorithms designed to improve healthcare delivery abound, for many, clinical integration will not be achieved. The deployment cost of machine learning models is an underappreciated barrier to success. Experts propose three criteria that, assessed early, could help estimate the deployment cost.
Among the many promises of big data, one of the most exciting could be the potential to unlock the detection of cancer before advanced malignancy ensures, which means opening up a whole new understanding of the disease.
A statistical model based on an analysis of routinely collected data from 1980 to 2017 predicts 1,601 excess injury deaths per year in the contiguous USA if average temperatures rise by 1.5 °C.