Trends in molecular medicine最新文献

A combination of intracellular and extracellular abnormalities of the nervous system, coupled with inflammation and intestinal dysbiosis, form the hallmarks of neurodegenerative diseases (NDDs). While it is difficult to identify the precise order in which these hallmarks manifest in NDDs because of their mutualistic nature, they cumulatively result in nervous or neuronal damage that characterizes neurodegeneration. In this review we discuss the roles of microRNAs (miRNAs) in the maintenance of nervous system homeostasis and their implication for NDDs. We further highlight recent advances in, and limitations of, miRNA therapeutics in NDDs and their future potential.

In a recent study in Cancer Discovery, Maciag et al. introduce BBO-8520, a novel inhibitor targeting both the active and inactive states of KRAS^G12C. This dual inhibition shows superior target engagement and prolonged tumor suppression, offering a compelling strategy to overcome resistance development and improve outcomes in KRAS^G12C-mutant cancers.

Pulmonary arterial hypertension (PAH) and cancer may appear to be unrelated at first, but there is increasing evidence that they share many characteristics and complexities. Pulmonary vascular cells in PAH resemble cancer cells in that they display abnormal growth patterns, resistance to cell death, metabolic changes, and channelopathies. These similarities open new possibilities for researchers and clinicians to apply cancer treatment strategies to PAH and possibly reverse the condition. This review explores the complex parallels between PAH and cancer, and emphasizes their similar channelopathy-like features at the molecular, cellular, and clinical levels. We also discuss the potential implications of these similarities for developing new treatments.

Frequent spillovers and recent geospatial expansion of avian influenza virus (AIV) pose significant economic and public health threats. Recent advances in vaccine technologies, bioinformatics, and artificial intelligence will provide newer approaches to target genetically diverse and rapidly evolving AIVs. Here, we review recent advances in, and perspectives on, developing universal vaccines needed for the effective control of AIVs.

Aging and Alzheimer's disease (AD) exhibit sex differences in several biological processes, including demyelination. In a recent study, Lopez-Lee et al. uncover the contributions of sex chromosomes and gonadal hormones to sex differences in demyelination and identify Toll-like receptor 7 (TLR7) as a potential target to ameliorate tauopathy-induced demyelination in men.

Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin. In a recent study, Srivastava et al. report that patients with hemophilia A receiving F8 lentiviral HSCT gene therapy achieved lasting factor VIII restoration and clinical improvement, marking an advance that could enable broader applications of HSCT.

Over the past 30 years, significant progress has been made in understanding the genetic causes of obesity. In the coming years, catalogs that map each genetic variant to its genomic function are expected to accelerate variant-to-function (V2F) translation. Given that obesity is a heterogeneous disease, research will have to move beyond body mass index (BMI). Gene discovery efforts for more refined adiposity traits are poised to reveal additional genetic loci, pointing to new biological mechanisms. Obesity genetics research is reaching unprecedented heights and, along with a renewed interest in the development of weight-loss medication, it holds the potential to identify new drug targets. Polygenic scores (PGSs) that predict obesity risk are expected to further improve and will be particularly valuable early in life for timely prevention.

The increasing prevalence and poor prognosis of central nervous system (CNS) metastases pose a significant challenge in oncology, necessitating improved therapeutic strategies. Recent research has shed light on the complex genomic landscape of brain metastases, identifying unique and potentially actionable genetic alterations. These insights offer new avenues for targeted therapy, highlighting the potential of precision medicine approaches in treating CNS metastases. However, translating these discoveries into clinical practice requires overcoming challenges such as availability of tissue for characterization, access to molecular testing, drug delivery across the blood-brain barrier (BBB) and addressing intra- and intertumoral genetic heterogeneity. This review explores novel insights into the evolution of CNS metastases, the molecular mechanisms underlying their development, and implications for therapeutic interventions.

Translational medicine is crucial for addressing health issues and translating research findings to improve population health. This Science and Society article highlights the potential of translational medicine in Mexico. It discusses the obstacles and challenges encountered in the translation process, instilling a sense of optimism for the future of healthcare in Mexico.