A new class of KRAS inhibitors has shown at least as much potential as FDA-approved MEK inhibitors in shrinking NF1 tumors, preventing new ones from forming, and extending survival in mice. The treatment has the potential to be less severe in terms of side effects, making it an attractive alternative or replacement for MEK inhibitors.
A new study suggests that indigo light, which is abundant in sunlight but poorly represented in standard indoor lighting, may prevent myopia. The study used tree shrews as a model and found that exposure to indigo light completely prevents nearsightedness from developing.
Researchers developed a new method to study long-distance DNA regulation in rare immune cells, uncovering previously hidden connections relevant to autoimmune diseases. The approach prioritized over 100 candidate genes linked to Crohn's disease risk, including CLN3, which was not typically associated with the condition.
A gene therapy approach has restored several disease-relevant traits in a mouse model of fragile X syndrome, including reduced seizure susceptibility and improved brain activity markers. The study's findings suggest that restoring the FMRP protein could improve symptoms such as sensory hypersensitivity and repetitive behavior.
A 10-year study published in NEJM found hydroxyurea treatment significantly reduces serious complications, hospitalizations, and death from sickle cell anemia. The study demonstrated improved growth and development in Ugandan children with the disease.
Researchers identified a potential new way to relieve chronic pain linked to neurofibromatosis type 1 (NF1). Blocking MAPK signaling with a MEK inhibitor lowered GDNF levels in Schwann cells and reduced pain-like responses in mice.
Researchers at Cincinnati Children's Hospital Medical Center have developed a new method to produce large, functional human gut organoids with nerve cells, growing them twice as fast as previous methods. These organoids can now be used for patching damage or restoring diminished functions of the small intestine, stomach, or colon.
Researchers identified a practical strategy to reduce bias in AI systems used in children's mental health care. By analyzing training data, they found that targeted improvements can significantly reduce disparities without diminishing overall model performance.
A single-cell study reveals how immune memory cells are molecularly programmed to respond faster when encountering a familiar threat. The study found that memory CD4⁺ T cells have their DNA primed to activate key defense genes within hours, providing a head start in responding to known pathogens.
A study by Cincinnati Children's Hospital Medical Center and the eMERGE network found that sharing genetic disease risk information with patients can be done responsibly, with a 70% success rate. The team shared results with over 24,000 people using various methods, including one-to-one conversations for those with higher-risk findings.
A study found that babies who develop severe E. coli infections lack sufficient germ-fighting antibodies, which mothers transfer to their babies. The research suggests that these antibodies protect against infection, and developing a screening test to identify high-risk newborns could prevent severe illnesses.
Scientists at Cincinnati Children's Hospital Medical Center have discovered a way to reduce the risk of a lethal side effect in cancer patients treated with immune checkpoint inhibitors. By targeting CD8 T cell-derived tumor necrosis factor, they found that blocking TNF signaling can prevent inflammatory cycle in the heart.
A study published in Stem Cell Reports suggests that transferring young microbiota to the intestines of older mice can speed up healing and improve the regenerative potential of intestinal stem cells. This approach may have implications for treating gut-related conditions such as inflammatory bowel disease.
A new study from Cincinnati Children's reveals how Fanconi anemia disrupts energy pathways and affects glucose metabolism, paving the way for targeted dietary interventions to improve health and reduce cancer risk. The research uses isotope tracing metabolomics to track nutrient metabolism in people with FA.
Researchers at Cincinnati Children's Hospital Medical Center have discovered a shared mechanism that may help improve healing across several types of muscle injury. Macrophages, immune cells known as microscopic garbage trucks, deliver ions to muscle fibers, facilitating rapid repair after an injury.
Researchers at Cincinnati Children's discovered a specific gene mutation in 'cold' tumors that block immune checkpoint inhibitors. Rapamycin was found to make these tumors responsive again by inhibiting mTORC1 signaling.
Researchers at Cincinnati Children's propose a unified approach to understand blood cell formation, identifying rare cell populations and gene regulatory networks. This breakthrough advances targeted therapies and stem cell engineering.
A new study demonstrates the safety and tolerability of enteral ventilation in a first-in-human trial, using rectal delivery of perfluorocarbon liquid. The researchers aim to improve oxygen levels in patients with blocked airways or severely limited lung function.
Sing Sing Way has made significant contributions to understanding the immune system's response to pregnancy, maternal-fetal dyad, and immunity in newborn babies. He is recognized for his outstanding professional achievement and commitment to service, bridging fundamental biology and translational medicine.
Researchers at Cincinnati Children's and Roche developed a patient-specific, immune-competent liver model to predict which drugs cause immune reactions. The miniaturized liver system built from stem cells and a patient's own immune cells reproduces hallmark signs of immune-mediated liver toxicity.
Researchers at Cincinnati Children's Hospital Medical Center have successfully grown liver tissue that can produce its own internal blood vessels. This breakthrough could lead to new treatments for people living with hemophilia and those experiencing acute or chronic liver failure, as the liver organoids can secrete coagulation factors.
Researchers found that a specific molecule called inosine, produced by beneficial gut bacteria, plays a critical role in developing immune cells. Supplementing antibiotic-exposed infants with inosine restored normal T cell development and enhanced infection resistance in mice.
A new study led by Cincinnati Children's reports that preterm infants born to women with preeclampsia or high blood pressure show signs of brain injury soon after birth and worse neurodevelopment at age 2. The study found decreased cognitive and language scores among preterm-born toddlers of mothers affected by high blood pressure.
Researchers discover a link between gut health and blood cancer risk, finding that age-related gut changes can accelerate the growth of pre-leukemic blood cells. A specific bacterial sugar, ADP-heptose, plays a key role in this process, and a new blood test detects its activity.
A team of experts at Cincinnati Children's reports achieving a milestone in growing human liver organoid tissue that faithfully reproduces key zones, nearly doubling rodent survival rate. This breakthrough paves the way for studying human liver biology and disease, accelerating drug development and potentially leading to repair tissues.
A new consensus report on anaphylaxis has been released, providing updated clinical criteria and guidelines for diagnosing and treating the condition. The report includes distinct infant findings to improve management in young children, addressing a significant burden on patients and caregivers worldwide.
A recent study published in JAMA Network Open reveals that using race-neutral lung function equations can identify more Black children with reduced lung function and increase asthma diagnosis rates. This is crucial as under-diagnosis can lead to delayed treatment and long-term health issues.
A new study suggests that an anti-inflammatory antibody treatment can be used to prevent heart transplant rejection. The treatment was found to block an innate immune response that drives dangerous inflammation in transplanted hearts, leading to prolonged organ survival.
Researchers have identified a crucial molecular signaling pathway in maintaining the skin barrier, which could lead to new treatments for inflammatory skin diseases. The study's findings also suggest that this pathway may contribute to certain types of cancer.
Two studies published in The New England Journal of Medicine show mixed results in treating EoE. Dupilumab is found to be effective in children under 12, while benralizumab fails to provide significant relief for patients. These findings challenge the long-held assumption that eosinophils are the primary cause of the disease.
Researchers have created the world's first human mini-brain that incorporates a fully functional blood-brain barrier, mimicking human neurovascular development. This breakthrough model promises to accelerate understanding and treatment of brain disorders like stroke, cerebral vascular disorders, and Parkinson's disease.
Researchers have discovered a small molecule compound called ESI1 that can regenerate vital myelin coatings, potentially treating multiple sclerosis and age-related cognitive deficits. The treatment promotes healing by clearing a roadblock in the repair process, allowing oligodendrocytes to produce myelin sheaths.
A study by Cincinnati Children's Hospital Medical Center found that children aged 3-5 consume a significant portion of their daily calories during the hour after picking up from childcare, with high amounts of added sugar and salt. Experts recommend preparing healthy snacks in advance to instill healthier habits.
A study published in Cell Reports found that early-life pain experiences can lead to genetic changes in macrophage cells, resulting in more intense pain reactions later in life. The researchers suggest targeting these genetic changes could help prevent long-lasting pain memories.
Scientists at Cincinnati Children's Hospital Medical Center have created the most detailed atlas of human blood stem cells, identifying over 80 distinct subsets. The study provides new insights into the development of leukemia and potential biomarkers for clinical monitoring.
A study published in Nature found that the response to hematopoietic insults differs across the skeleton, with certain bones specialized to respond to specific stresses. The research uses confocal imaging microscopy to count different cell types and provides new insights into blood cell production, potentially leading to improved treat...
The COVID-19 pandemic disrupted learning and development among school-aged children, with disadvantaged families feeling the effects most severely. Researchers found that only 30% of students were assessed as kindergarten-ready in 2021, compared to 40% in 2018.
A new computational tool, SNAF, has been developed to identify shared splicing neoantigens that can help a patient's immune system fight cancer. The tool has already uncovered promising targets for melanoma and other cancers.
Researchers have discovered how pioneer transcription factors, such as FOXA and OCT4, coordinate with epigenetic repressors to safeguard cell fate, enabling precise manipulation of cell fate in cellular programming and reprogramming. This breakthrough has important implications for scaling up organoid and tissue engineering technology.
Researchers analyzed data from over 1,500 infants, revealing a correlation between reports of food insecurity and difficulties maintaining benefits, leading to behavioral dysfunction. The study emphasizes the importance of early intervention and highlights the role of pediatricians in connecting families with support programs.
Scientists have identified a population of progenitor cells that strongly promote new blood vessel growth in ischemic limbs, showing promise as a treatment for preventing amputations. The discovery was made through single-cell transcriptomics and confirmed in mice with limb ischemia, where the treatment rescued limbs from amputation.
A study by Cincinnati Children's explores how systemic hesitances contributed to COVID-19 risks, including inadequate disease surveillance and top-down command and control. The authors emphasize the need for integrated systems, flexible approaches, and rapid learning to overcome these challenges and prevent future public health crises.
A six-year study analyzing data from over 15,000 lower-income children ages 2 to 6 reveals higher-than-expected levels of significant behavioral dysfunction. The research highlights the importance of early interventions to prevent behavior problems from escalating.
A new study published in JAMA Network Open found live vaccinations safe and effective for children who have received liver or kidney transplants, offering protection against life-threatening conditions. The study showed high antibody levels and minimal adverse events following vaccination.
Researchers have developed a human vascular organoid model that accurately mimics the damage caused by SARS-CoV-2, revealing clues for a potential COVID-19 treatment. The study identified a long-acting monoclonal antibody targeting factor D as a promising approach to mitigate severe vascular damage and thrombosis associated with COVID-19.
Researchers discovered a cell signaling pathway that allows effector memory T cells to drive innate cytokine storms in autoimmune diseases. Targeting the STING pathway may prevent inflammation chain reactions, offering new hope for treating conditions like type 1 diabetes and rheumatoid arthritis.
Researchers found that mothers' bodies retain a long-term supply of immune suppressive T cells that recognize the next fetus by the same couple, instructing the rest of the immune system to stand down. This 'memory' effect persists in women even after childbirth.
Researchers isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore and found that preventing its function stops disease progression. A potential treatment strategy involves targeting the mitochondrial pore with a nontoxic inhibitor, which could provide benefits independently or in co...
Researchers have identified specific cellular signatures for kidney transplant rejection, which could lead to the development of more effective anti-rejection therapies. The study's findings suggest that treatments often do not thoroughly eliminate T cells responsible for rejection, and new drug targets may be able to address this issue.
A clinical trial found that benralizumab effectively depleted eosinophils, but it did not alleviate symptoms or improve gut tissue health in patients with eosinophilic gastritis. The study's findings suggest a new therapeutic target for eosinophilic gastrointestinal diseases and highlight the need to reconsider remission definitions.