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To celebrate the end of our 25th anniversary year, we asked thought leaders and experts in the field to answer one question: What will shape the next 25 years of medical research?
Julie Makani is an associate professor in the Department of Haematology and Blood Transfusion at Muhimbili University of Health and Allied Sciences in Dar es Salaam, where she helped Tanzania establish one of the world’s largest single-center study cohorts for sickle cell disease. She received the 2011 Royal Society Africa Award on the translation of genomics to health benefits.
To promote effective translational medicine, academia must take the lead in creating a conducive environment for the first steps into translation, urge Roch Ogier and colleagues.
The recent report of an antisense oligonucleotide therapy designed for only one patient spotlights regulatory, economic and ethical issues that must be grappled with in an era of accelerated development of ‘ultra-personalized’ drugs.
Herpes simplex virus-1 encephalitis is linked to variants in a small nucleolar RNA, suggesting a new mechanism for antiviral immunity in cortical neurons.
Aptamer-based proteomic analysis of plasma from healthy individuals aged 18–95 years reveals wave-like patterns of protein expression that are associated with age-related diseases and phenotypic traits.
Enteroviral infection persistence as determined by host genetic susceptibility, rather than independent, short-lived infections, may contribute to islet autoimmunity and be a precursor to the development of type 1 diabetes.
There is a transgenerational increase in the susceptibility of female offspring to developing PCOS that occurs via the female germline and is linked to fetal exposure to excess androgen.
Imaging of muscle fibrosis with a handheld device could potentially serve as a biomarker for disease progression and response to therapy in patients with Duchenne muscular dystrophy.
Analysis of known viruses in stool samples from young children with high genetic risk for type 1 diabetes shows that sustained enterovirus B (EV-B) infections, rather than independent, short-duration EV-B infections, might be involved in the development of islet autoimmunity, but not type 1 diabetes.
Large-scale aptamer-based scanning of plasma proteins coupled with machine learning demonstrates proof-of-concept and feasibility of an individualized health check using a single blood sample.
A combination of high-resolution imaging and modeling approaches facilitates the study of the mechanisms and clinical progression of non-alcoholic fatty liver disease in humans.
The first BCL-XL-degrading PROTAC achieves safer and more potent antitumor activity than dual BCL-XL and BCL-2 inhibitor navitoclax because of reduced dose-limiting platelet toxicity and high target specificity.
Analysis of fully clinically annotated and sequenced melanoma tumor samples collected before anti-PD1 treatment suggests that determinants of response differ on the basis of previous anti-CTLA4 therapy, and that tumor mutational burden may not be a strong predictor of response across melanoma subtypes.
Ultra-deep sequencing of paired plasma-circulating free DNA and white blood cells allows the identification of tumor-derived somatic mutations with high accuracy by filtering out variants consistent with clonal hematopoiesis.
With the growing number of efficient gene therapies on the market, now is the time to take actions to ensure reasonable pricing of gene therapy products. Among these, we propose to incentivize gene therapy companies to adopt a status that translates their corporate social responsibility into concrete commitments.