A few months ago, Protective Services officers at Cincinnati Children's requested help from our Marketing & Communications team on a video. They wanted to be part of a lip sync challenge.
A daily hydroxyurea pill may bring relief for children living with the painful and deadly blood disease sickle cell anemia (SCA) in resource-challenged sub-Saharan Africa, where the disease is prevalent and health care is suboptimal. This is what a multinational clinical trial called REACH discovered when it tested daily hydroxyurea treatment in 606 children between the ages of 1 and 10 years old. Study data are published in the New England Journal of Medicine.
Genetic testing revealed Robin Gramke and her daughter McKenna have a complex condition called 22q.
Cincinnati Children’s Hospital Medical Center, seed investor CincyTech announced the formation and seed financing of Clarigent Health, a platform technology company leveraging artificial intelligence and natural language processing to give health care providers a new clinical decision support tool to address suicide, which in recent years has risen at alarming rates among teens.
First-of-its-kind study looks at female athletes wearing experimental neck collar
Preterm birth remains a global epidemic linked to a lifetime of potential health complications. It also is difficult to study in living creatures—especially the uniquely precise biology of preterm birth in humans. Researchers report in PLoS Biology successfully inserting just enough human DNA into transgenic laboratory mice that it allowed the team to study a unique part of human pregnancy compared to other animals.
Physicians who specialize in a devastating and aggressive immune disorder called hemophagocytic lymphohistiocytosis (HLH) report in a new study that extra care should be taken to ensure an HLH diagnosis doesn’t obscure possible underlying cancers. They caution in the journal Pediatric Blood & Cancer that expediting HLH treatment may miss underlying malignancies that could end up being fatal to the patient.
Two recent studies in the journal Leukemia present a new approach for bone marrow donation and transplant that preclinical laboratory tests suggest could make the life-saving procedure safer and more effective for patients. Researchers say their studies demonstrate that use of an experimental drug called CASIN in laboratory mice results in higher efficiency when harvesting blood stem cells from donors and less toxicity in transplant recipients.
Scientists working to bioengineer the entire human gastrointestinal system in a laboratory now report using pluripotent stem cells to grow human esophageal organoids. The newly published research in the journal Cell Stem Cell is the first time scientists have been able to grow human esophageal tissue entirely from pluripotent stem cells (PSCs), which can form any tissue type in the body.
With an estimated 1.6 million people in the U.S. dealing with inflammatory bowel disease (IBD), physicians can have a hard time telling which newly diagnosed patients have a high risk of severe inflammation or what therapies will be most effective. Now researchers report in the journal JCI Insights finding an epigenetic signature in patient cells that appears to predict inflammation risk in a serious type of IBD called Crohn’s disease.
Using new bioinformatics tools to analyze thousands of human tissue samples, researchers created a new database of daily rhythms in human gene activity—including many genes that regulate how drugs work. Reporting in Science Translational Medicine, scientists say their results could have significant implications for a growing field of study called circadian medicine.
Obesity and its related ailments like type 2 diabetes and fatty liver disease pose a major global health burden, but researchers report in Nature Communications that blocking an RNA-silencing protein in the livers of mice keeps the animals from getting fat and diabetic conditions.
Scientists may be on the road to solving the mystery of a group of mostly incurable blood diseases called myelodysplastic syndromes (MDS), which cause people to have immature, malfunctioning bone marrow cells that fuel a diverse set of health problems and can lead to leukemia. Researchers report in the journal Cancer Discovery identifying a gene that in laboratory experiments fuels the biological processes that cause the different types of MDS that physicians see in patients.
Researchers report in Nature Communications they figured out why air sacs in the lungs clog up with a thick substance called surfactant in a brutal disease called Pulmonary Alveolar Proteinosis (PAP), and they show taking cholesterol-busting pills called statins can effectively treat the disease.
Scientists report finding a potentially promising treatment target for aggressive and deadly high-grade brain cancers like glioblastoma. Publishing online June 18 in Nature Cell Biology, the study also reports the current lack of a drug that hits the molecular target keeps it from being advanced for testing as a therapeutic strategy for patients with few treatment options.
New preclinical research shows a gene already linked to a subset of people with autism spectrum disorder is critical to healthy neuronal connections in the developing brain, and its loss can harm those connections to help fuel the complex developmental condition. Scientists report in Developmental Cell their data clarify the biological role of the gene CHD8 and its protein CHD8 in developing oligodendrocytes, cells that form a protective insulation around nerves.
A study led by scientists at Cincinnati Children’s reveals one mechanism involved in regulating when nephron formation ends. If translated to clinical practice years from now, the result could be reduced need for kidney transplants and fewer deaths from kidney disease.
Scientists exploring how to tame random gene fluctuations as the embryos that become our bodies start to form have identified a control switch in the vertebrate segmentation clock of developing zebrafish. The researchers report in Cell Reports their findings could uncover methods for modulating genetic signals to prevent birth defects or cancers rooted at the earliest stages of development.
Researchers tissue-engineered human pancreatic islets in a laboratory that develop a circulatory system, secrete hormones like insulin and successfully treat sudden-onset type 1 diabetes in transplanted mice. In a study published by Cell Reports, the scientists use a new bioengineering process they developed called a self-condensation cell culture. The technology helps nudge medical science closer to one day growing human organ tissues from a person’s own cells for regenerative therapy
By studying a rare liver disease called Alagille syndrome, scientists discovered the mechanism behind a form of tissue regeneration that may someday reduce the need for organ transplants. Researchers report in Nature that when disease or injury causes a shortage in one type of liver cell, the organ can instruct another type of liver cell to change identities to provide replacement supplies. The findings one day may lead to a viable treatment for human disease.
Researchers mapped critical brain-to-spinal cord nerve connections that drive voluntary movement in forelimbs, a development that scientists say allows them to start looking for specific repair strategies. The study is an important step toward one day rehabilitating motor circuits to help motor function recover after an injury or disease damages the central nervous system, the scientists report in Cell Reports.
Researchers used an experimental molecular therapy in preclinical laboratory tests to effectively treat several types of deadly pediatric brain cancer and now propose advancing the treatment to clinical testing in children. Scientists report in the journal Molecular Cancer Therapeutics testing the small molecule 6-thio-2’deoxyguanosine (6-thio-dG) in brain cancer stem cells derived from tumor cells donated by patients. Researchers also tested the treatment in humanized mouse models of pediatric brain cancer.
A study by scientists at Cincinnati Children’s reports that the Epstein-Barr virus (EBV)—best known for causing mononucleosis—also increases the risks for some people of developing seven other major diseases. Published in Nature Genetics, researchers report EBV also plays a role in six other diseases: multiple sclerosis, rheumatoid arthritis, inflammatory bowel disease, type 1 diabetes, juvenile idiopathic arthritis, and celiac disease.
Scientists used an experimental targeted molecular therapy to block a matrix-forming protein in heart cells damaged by heart attack, reducing levels of scarred muscle tissue and saving mouse models from heart failure. Researchers at the Cincinnati Children’s Heart Institute report in the journal Circulation
Scientists searching for a therapy to stop the deadly and mostly untreatable lung disease, idiopathic pulmonary fibrosis (IPF), found a new molecular target that slows or stops the illness in preclinical laboratory tests. Researchers at Cincinnati Children’s Hospital Medical Center report their data in the journal Cell Reports. Studying mice with IPF and donated human cells, they identified a gene called FOXF1 that inhibits the IPF disease.
A few months into her pregnancy, Anisah Brown had a routine ultrasound that detected a large hole in her baby’s diaphragm. Her baby had been diagnosed with a congenital diaphragmatic hernia, or CDH. The hole would let the liver, stomach and other internal organs compress the lungs, leaving them little room to develop. Her doctor told her there was no hope. But that was before she came to the Cincinnati Fetal Center.
Researchers at Cincinnati Children’s report in the journal Pediatrics a link between parents impacted by adverse childhood experiences and increased risk for delayed development of their children at age two.
The Cincinnati Drug and Poison Information Center at Cincinnati Children's is seeing an increasing number of calls regarding drug overdoses as the nation copes with the opioid epidemic.
In findings that one day may help people sleep better, scientists have uncovered the first molecular evidence that two anciently conserved proteins in the brains of insects and mammals share a common biological ancestry as regulators of body temperature rhythms crucial to metabolism and sleep. Researchers publish their data in the journal Genes & Development.
Genomic profiling of mostly untreatable and deadly nerve sheath tumors led scientists to test a possible therapeutic strategy that inhibited tumor growth in lab tests on human tumor cells and mouse models, according to research in the journal Cancer Cell.
When the body attacks its own healthy tissues in an autoimmune disease, peripheral nerve damage handicaps people and causes persistent neuropathic pain when insulation on healing nerves doesn’t fully regenerate. Unfortunately, there are no effective ways to treat the condition. Now scientists describe in Nature Medicine an experimental molecular therapy that restores insulation on peripheral nerves in mice, improves limb function, and results in less observable discomfort.
Researchers used 3D imaging with molecular testing to uncover new insight into the earliest stages of mammalian pregnancy—offering clues to unsolved questions in pregnancy. Investigators report Feb. 9 in Nature Communications they demonstrated in mice that glands in the uterus must link and communicate directly with the embryo so it will implant and begin pregnancy.
Before medical science can bioengineer human organs in a lab for therapeutic use, two remaining hurdles are ensuring genetic stability—so the organs are free from the risk of tumor growth—and producing organ tissues of sufficient volume and size for viable transplant into people. Scientists report in Stem Cell Reports achieving both goals with a new production method for bioengineered human gut and liver tissues.
Scientists discovered how a gene mutation affects T cell function to promote immune disorders and then tested a treatment based on the discovery—successfully fixing donated immune cells from a 16-year-old boy with an abnormally low level of white blood cells called lymphopenia. Researchers report their findings Jan. 30 in Nature Communications
New research published in the Journal of Experimental Medicine highlights the pernicious effect of obesity on the long-term health of blood-making stem cells (hematopoietic stem cells). Published Dec. 27 and conducted largely in genetic models of obese mice, the study shows obesity causes durable and harmful changes to the hematopoietic stem cell compartment – the blood-making factory in our bodies.
Women exposed to air pollution just prior to conception or during the first month of pregnancy face an increased risk of their children being born with birth defects, such as cleft lip or palate or abnormal hearts.
Researchers report creating a biologically accurate mass-production platform that overcomes major barriers to bioengineering human liver tissues suitable for therapeutic transplant into people.
Birth defects in babies born infected with Zika virus remain a major health concern. Now, scientists suggest the possibility that some women in high-risk Zika regions may already be protected and not know it. New research in PLOS Pathogens on Nov. 16, performed in mice, shows women who develop symptom-free Zika infections may be able to acquire immunity that would protect them from future infections and their offspring in a future pregnancy.
Seven years ago Cincinnati Children’s scientists first used pluripotent stem cells to mimic natural human development and grow working human intestine in a lab. Today medical center doctors can bioengineer the gastrointestinal tissues of sick children to find clues about a child’s disease and how to treat it. Cincinnati Children’s is building on this early research advancement in personalized medicine by launching the Center for Stem Cell and Organoid Medicine (CuSTOM).
A new study explores how herpes simplex virus might change when passed from one individual to another, information that may prove useful in future development of therapeutics and vaccines.
Early research results suggest scientists might be on to a way to preserve heart function after heart attacks or for people with inherited heart defects called congenital cardiomyopathies. Researchers at the Cincinnati Children’s Heart Institute report Sept. 28 in Nature Communications that after simulating heart injury in laboratory mouse models, they stopped or slowed cardiac fibrosis, organ enlargement and preserved heart function by blocking a well-known molecular pathway.
When it comes to male reproductive fertility, timing is everything. Now scientists are finding new details on how disruption of this timing may contribute to male infertility or congenital illness.
Researchers report encouraging preclinical results as they pursue elusive therapies that can repair scarred and poorly functioning heart tissues after cardiac injury. Scientists from the Cincinnati Children’s Heart Institute inhibited a protein that helps regulate the heart’s response to adrenaline, alleviating the disease processes in mouse models and human cardiac cells. Their data publishes Aug. 22 in the Journal of the American College of Cardiology.
Scientists used human pluripotent stem cells to generate human embryonic colons in a laboratory that function much like natural human tissues when transplanted into mice, according to research published June 22 in Cell Stem Cell. The study is believed to be the first time human colon organoids have been successfully tissue engineered in this manner, according to researchers who led the project.
An international team of researchers bioengineering human liver tissues uncovered previously unknown networks of genetic-molecular crosstalk that control the organ’s developmental processes – greatly advancing efforts to generate healthy and usable human liver tissue from human pluripotent stem cells. The scientists report online in Nature on June 14 that their bioengineered human liver tissues still need additional rounds of molecular fine tuning before they can be tested in clinical trials.
A detour on the road to regenerative medicine for people with muscular disorders is figuring out how to coax muscle stem cells to fuse together and form functioning skeletal muscle tissues. A study published June 1 by Nature Communications reports scientists identify a new gene essential to this process, shedding new light on possible new therapeutic strategies.
Immune diseases like multiple sclerosis and hemophagocytic lymphohistiocytosis unleash destructive waves of inflammation on the body, causing death or a lifetime of illness and physical impairment. With safe and effective treatments in short supply, scientists report in PNAS Early Edition (Proceeding of the National Academy of Science) discovery of an experimental treatment that targets an Achilles heel of activated immune cells – killing them off and stopping autoimmune damage.
Vaccinating pregnant mothers year-round against flu in the resource-challenged region of subtropical Nepal reduced infant flu virus infection rates by an average of 30 percent, increased birth weights by 15 percent and resulted in babies having less influenza, according to a study published in The Lancet Infectious Diseases. An international research team says expanding year-round flu vaccinations during pregnancy would also benefit children in other tropical and subtropical parts of the world.
Scientists report in Neuron the lost function of two genes prevents infant laboratory mice from developing motor skills as they mature into adults. Researchers also suggest in their study that people with certain motor development disabilities be tested to see if they have mutant forms of the same genes. Their data show that neural circuits between the brain’s motor cortex region and the spinal cord did not properly reorganize in maturing mice lacking the genes.
Scientists report in Nature Communications it may be possible to therapeutically fine tune a constantly shifting balance of molecular signals to ensure the body’s peripheral nerves are insulated and functioning normally. In a study published April 26, they suggest this may be a way to treat neuropathies or prevent the development of peripheral nerve sheath tumors. They discovered genetic dysfunction in the HIPPO-TAZ/YAP and Gαs-protein feedback circuit disrupts the balanced formation of the protective myelin sheath.
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