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Gene therapy holds tremendous promise for treating a wide range of hereditary and acquired diseases by delivering exogenous therapeutic nucleotide sequences into specific cells or tissues. Recent advances support the notion that gene therapy could offer a long-term cure for diabetes mellitus, something that current conventional pharmacotherapies cannot achieve.
Two recent studies have unravelled novel modes of glucose-dependent insulinotropic polypeptide receptor (GIPR) signalling regulation. Kizilkaya et al. characterized the effect of changes in β-arrestin 2 coupling with naturally occurring GIPR coding variants, whereas Regmi et al. investigated GIPR expression profiles and functional regulation in adipocytes.
Thyroid nodules are common; the majority are benign and asymptomatic, and therefore the main aim of the diagnostic process is to determine their clinical significance without leading to overdiagnosis and, consequently, overtreatment, as outlined in this article.
Phaeochromocytomas and paragangliomas (PPGL) are rare endocrine tumours that can affect paediatric patients as well as adults. In this first international Consensus Statement on PPGL in paediatric patients, the authors discuss the diagnosis, management and long-term surveillance of these tumours in children and adolescents.
Medically not yet explained symptoms (MNYES) are an important consideration in patients treated for hypothyroidism who experience persistent symptoms. This Perspective brings attention to the issue of MNYES in hypothyroidism and considers effective management.
Climate change is causing human populations to be exposed to increasingly higher ambient temperatures and more frequent and extreme heatwaves than previously observed. This Perspective considers the available evidence on the endocrine effects of heat exposure, and maps out a path for future research into this field.
Nutritional imbalances (such as overnutrition in obesity) alter many properties of the neurovascular system, including neurovascular coupling and blood–brain barrier permeability. This Perspective examines how lifestyle interventions targeting weight loss, including intermittent fasting, caloric restriction and physical activity, affect the neurovascular system and neuronal networks.
Graves disease, an autoimmune disease characterized by an enlarged and overactive thyroid gland, is the most common cause of hyperthyroidism in iodine-sufficient areas. In this Review, the authors describe the epidemiology, pathogenesis, and conventional treatment of Graves hyperthyroidism and Graves orbitopathy and discuss advances that have enabled development of novel treatment modalities.
This article reviews the role of the parathyroid hormone 1 receptor (PTH1R) pathway in bone homeostasis and of osteocytes as mechanosensors and drivers of bone remodelling. It will also discuss how the PTH1R–sclerostin axis can be harnessed for the treatment of bone disease induced by diabetes mellitus.
Accurate measurement of adiposity and adipose tissue distribution is essential for the treatment of obesity and cardiometabolic disease, but can often prove challenging. This Perspective discusses the advantages and disadvantages of different methods of assessing adiposity in clinical settings.
Thermal injuries trigger stress-induced endocrine responses that are critical for survival. With age, these responses falter, which results in an inability to combat burn-induced dysfunction and greatly increased mortality. Current knowledge about the post-burn endocrine response in older adults is sparse, which leaves therapeutic gaps for this vulnerable and expanding demographic.