Volume 25 Issue 12, December 2019

From investigating the diversity of the vaginal microbiome to developing neuroprosthetics with sensory feedback for leg amputees, the work of the authors featured in our pages is fascinating. We asked them about their hopes for the future of medical research.

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?

Medical schools and graduate research programs embrace artificial intelligence.

Rachel Ramoni is chief research and development officer for the Department of Veterans Affairs, where she oversees 2,000 active projects at more than 100 sites.

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.

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.

Recent large-scale proteomic studies reveal the association of levels of circulating proteins with life span and many health-related outcomes.

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.

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.

Systemic chronic inflammation increases with age and is linked to the development of several diseases, as presented in this Perspective.

Inhibition of CSF-1R with recurrent activating alterations and other actionable targets identified by genomic sequencing elicits clinical activity in patients with histiocytosis.

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.

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 new assay combining genotypic and phenotypic information accelerates accurate clinical antibiotic susceptibility testing.

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.

Herpes simplex virus-1 encephalitis is linked to variants in a small nucleolar RNA, suggesting a new mechanism for antiviral immunity in cortical neurons.

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.

Prenatal androgen exposure causes transgenerational increases in offspring susceptibility to polycystic ovary syndrome in adulthood.

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 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-sensitive cell-free DNA (cfDNA) sequencing uncovers clonal hematopoiesis as a major source of somatic cfDNA variants in healthy individuals and patients with cancer, and underscores the importance of matched white blood cell DNA sequencing in liquid biopsy procedures.

The first BCL-X_L-degrading PROTAC achieves safer and more potent antitumor activity than dual BCL-X_L and BCL-2 inhibitor navitoclax because of reduced dose-limiting platelet toxicity and high target specificity.