Cincinnati Children's scientists have successfully grown functional human intestinal organoids that mimic key development stages of the human fetus. These lab-grown tissues accurately replicate the formation of specific cell types and tissue structures, providing a valuable resource for studying fetal intestine development and potentia...
A new therapy delivery method, using modified viruses engineered with fusogens Myomaker and Myomerger, shows promise as a treatment for Duchenne muscular dystrophy. The vector can deliver a vital gene needed for muscle function to cells, potentially providing a lifelong supply of the missing gene.
Researchers found that a combination of IL-22 and IL-1R inhibitors could be a promising treatment for inflammatory bowel diseases like Crohn's disease and ulcerative colitis. The study suggests that targeting both pathways may lead to more effective treatment outcomes.
Researchers found that consuming vitamin A or its analogs can help prevent painful pancreas inflammation in children and young adults with acute lymphoblastic leukemia (ALL) during chemotherapy treatment. The study revealed that only 1.4% of patients developed pancreatitis when taking vitamin A, compared to 3.4% without it.
Researchers discovered a new approach to treating ependymoma, a rare childhood brain tumor, by targeting YAP fusion proteins. The study found that blocking the function of BRD4 gene expression regulator can prevent tumors from forming.
A team from Cincinnati Children's Hospital Medical Center discovered how segmentation clock genes instruct the tempo of spine formation, opening doors to new basic science research. By inducing segment formation in zebrafish without biological clocks, the researchers aimed to understand the origins of birth defects in humans.
A new driver training program has been shown to reduce the frequency of long, distracted glances away from the road among teen drivers with ADHD. The FOCAL+ training program improved teen drivers' attention to the roadway and reduced crash risk by 40% compared to a control group.
Scientists have developed a detailed 'atlas' of human fetal brain development, revealing the origin of aggressive medulloblastomas. The study identifies a collection of progenitor cells that give rise to these tumors and provides potential targets for therapy.
Researchers have identified two subgroups of Ewing sarcoma that respond differently to targeted drug therapy, with the first group responding to Linsitinib and AZD1775 treatment. The study suggests that repeated biopsies may be needed over time to determine the best course of treatment for each patient.
A study by Cincinnati Children's Hospital Medical Center has identified two genes, ZEB1 and ALDH1A1, as critical in producing malignant cells in MPNST. The research found that disrupting these genes can slow tumor growth and reduce proliferation in mice, suggesting potential new treatments for the disease.
A collaborative network led by Cincinnati Children's Hospital Medical Center has received an $8 million grant to identify and validate biomarkers for acute rheumatic fever. This will enable the development of a sensitive and specific diagnostic test, crucial for diagnosing rheumatic heart disease, which is caused by a slow but progress...
Scientists at Cincinnati Children's develop a 'plug-and-play' method to consistently produce large numbers of lab-grown small intestines, colons, and stomachs. The new process overcomes the bottleneck of inconsistent organoid production, enabling faster and more accessible research.
A study from Cincinnati Children's Hospital Medical Center found that early antibiotic use disrupts the developing immune system of newborns, increasing risks of severe pneumonia and life-long health issues. Fecal transplants may offer a solution to mitigate these effects.
Researchers discovered that pregnant women produce super antibodies to protect their newborns by altering the structure of sugars attached to the antibodies. This molecular change allows immunoglobulin G to stimulate immunity through receptors that respond specifically to deacetylated sugars, providing expanded protective role.
Scientists discovered that commensal fungi like Candida albicans need to be alive and actively producing proteins to elicit a beneficial immune response. The fungus oscillates between high and low expression of the UME6 gene, signaling its benefits to the body.
A study published in Nature Communications found that BMP9 treatment restored capillary density and improved alveolarization in mice with ACDMPV. The treatment showed promise for improving care for BPD and CDH, two conditions that also affect lung development.
Researchers at Cincinnati Children's Hospital Medical Center discovered that a combination of two anti-inflammatory drugs may prevent lung damage and asthma in infants born prematurely due to chorioamnionitis. The study used animal models, but further research is needed to confirm the results.
Researchers at Cincinnati Children's Hospital Medical Center have identified a protein crucial to AML cell survival and found a small molecule that blocks its function, killing cancer cells in lab dishes and mouse models. This potential breakthrough could lead to novel therapies for AML and other conditions.
A new study from Cincinnati Children's Hospital Medical Center found that medical-legal partnerships, which connect pediatric patients with advocates from Legal Aid Society, significantly reduce hospital admissions for children. The program, launched in 2008, has resulted in a 38% reduction in hospitalizations among referred cases.
A new study suggests that the timing of steroid treatment may be crucial to its effectiveness in patients with Duchenne muscular dystrophy. Researchers found that weekly doses of prednisone were more effective when administered at a specific time of day, promoting muscle function and reducing side effects.
Researchers found that a diet rich in butyrate sharply reduced the risk of biliary atresia in mouse pups, and identified glutamine as a key compound with similar protective effects. The findings suggest a potential role for maternal microbiome manipulation in preventing liver disease.
The study reveals that the Cdc42 gene is essential for proper alignment and function of Sertoli cells, which support sperm cell development. Disruptions in this gene can lead to reduced testicular function and infertility. Researchers hope to develop non-invasive diagnostic tests to identify specific causes of male infertility.
A study by Cincinnati Children's Hospital Medical Center has identified a common biological response platform that could help alleviate allergic inflammation. The research suggests that inhibiting this pathway may provide a unique opportunity to counteract type 2 immunity and reduce allergic reactions.
Researchers at Cincinnati Children's grow functional biliary organoids to reveal previously unknown cell defects and a potential repair approach. The study identifies structural defects in cells that form bile ducts, which can lead to liver fibrosis and failure.
A study by Cincinnati Children's Hospital Medical Center reveals that minor cells with mutations in the DDX41 gene can trigger a subset of inherited MDS cases. The research suggests that targeting these cells could lead to new treatment options for patients.
Researchers at Cincinnati Children's Hospital Medical Center are exploring telehealth as a treatment option for adolescents with suicidal thoughts, following a $7.5 million PCORI award. The study aims to evaluate the safety and effectiveness of suicide prevention treatment delivered by telehealth during the pandemic.
Researchers discovered a three-step molecular process behind cardiac atrophy, which can lead to fatal heart failure. The study suggests potential new ways to prevent or treat the problem by eliminating or reducing the activity of specific genes.
Researchers developed a nanoparticle delivery system to stimulate blood vessel growth in the newborn lung, improving survival rates from 70% to 35%. The treatment involves delivering an engineered nanoparticle carrying a STAT3 gene vector, which prompts blood vessel growth and improves lung function.
Scientists at Cincinnati Children's are developing an automated risk assessment system using natural language processing and machine learning to analyze student interviews and predict violent outcomes.
A Cincinnati Children's Hospital Medical Center study found that a combination of two early reading programs improved kindergarten readiness by 15.4 percentage points over three years. The Reach Out and Read/Imagination Library program boosted preliteracy skills, improving school success.
A team of scientists at Cincinnati Children's Hospital Medical Center has discovered a promising new approach to preventing obesity-driven liver damage. By controlling a subgroup of immune system cells that trigger non-alcoholic fatty liver disease (NAFLD), researchers have identified a potential treatment target.
A team of scientists at Stanford University and Cincinnati Children's have found a potentially effective compound, AG1296, to treat pulmonary arterial hypertension (PAH). The compound was identified using human, PAH-specific stem cells as a drug screening platform.
Researchers at Cincinnati Children's Hospital Medical Center developed a new framework to understand how a child's brain is wired to learn to read. The 'eco-bio-developmental' model identifies risk factors for reading difficulties in infancy, emphasizing the importance of early screening and intervention.
Researchers at Cincinnati Children's Hospital Medical Center have developed a 'map' of bone marrow tissue, providing new insights into how tiny blood vessels organize the bone marrow and regulate blood cell production. This discovery advances understanding of how to control the production of specific blood cells at will.
A new approach inhibiting SCD and blocking FOSB function blunts acquired resistance and extends survival in mouse models of glioblastoma, with potential applicability to other cancers like melanoma.
A new screening tool, The Reading House (TRH), assesses early literacy skills in preschool-age children and has been validated as a potential identifier of reading difficulties. TRH measures core skills predictive of reading success and identifies relationships between screened skills and early brain development in the brain.
Researchers at Cincinnati Children's Hospital Medical Center have discovered a novel method to boost the effectiveness of mTOR inhibitors against acute myeloid leukemia (AML) by targeting alternative signaling pathways. The study suggests using a combination therapy that combines an mTOR inhibitor with other drugs inhibiting CDK9, c-My...
Researchers used bioengineered models of patient-specific skin to study how inherited DNA defects cause skin damage and deadly squamous cell carcinoma in children with Fanconi anemia. The study identified key biological connections important to skin formation and function, leading to accelerated blistering and disease progression.
The PEDSnet report analyzes over 135,000 medical records to reveal disparities in COVID-19 outcomes among children. Children from lower-income families, Black, Latinx, and Asian groups are more likely to be infected, while those with diabetes or cancer fare significantly worse than others.
A study by Cincinnati Children's Hospital Medical Center found that targeting the BCAP protein could help control overactive immune responses and promote tissue repair in inflammatory diseases. The researchers suggest that modulating BCAP activity could be used to dampen inflammation and accelerate response against tumors.
A new software tool can accurately predict motor development disorders in preterm infants based on early MRI scan data. The tool quantifies diffuse white matter abnormality (DWMA) volume, which serves as a biomarker for brain developmental issues.
The FDA has approved Nucala for use in treating patients with hypereosinophilic syndrome, a life-threatening group of blood disorders. The drug reduces disease flares and improves health without substantial side effects.
Human intestinal organoids grown from stem cells help identify peptide YY as a possible therapeutic for severe malabsorption. The hormone allows 80% of mice to survive with just one injection per day, showing promise for treating congenital disorder.
New research reveals viruses, especially endogenous retroviruses, shape evolution by driving gene expression and fine-tuning species-specific transcriptomes. This has significant implications for clinical practice, particularly in understanding male infertility and birth defects.
Scientists have developed a gene test that can predict the risk of certain medications causing liver injury. The test uses a polygenic risk score, which takes into account over 20,000 gene variants to identify individuals at higher risk. This could lead to more accurate prescribing and safer drug development.
A study of over 180 girls found that those entering puberty early have a longer window of susceptibility to breast cancer, with higher growth hormone levels and slower pubertal progression. This can be mitigated through healthy lifestyle habits, such as exercise, balanced diet, and reduced exposure to endocrine disruptors.
Researchers found that deleting AMPK from astrocyte brain cells disrupts glucose and lactate metabolism in neurons, leading to spontaneous seizures. The study suggests that metformin, an antidiabetic drug, may mitigate epileptic seizures by targeting AMPK.
Scientists from Cincinnati Children's Hospital Medical Center and RIKEN report discovering signals that control the formation of human organs from fetal foregut. The study provides a roadmap for growing more complex and functional organoids using human stem cells.
A new study has identified a potential therapeutic target, fibronectin (FN1), to improve endocardial function and regenerate cardiac valves, septum, and coronary vessels in children with hypoplastic left heart syndrome. This discovery offers hope for increasing heart chamber size and reducing the need for multiple surgeries.
Two common gene variants in the KIF3A gene increase the risk of young children developing atopic dermatitis and food allergies. The study provides new insights into the genetic mechanisms underlying this condition, which affects up to 20% of all children.