A recent study published by PLOS ONE sheds light on the impact of genetic mutations on cystic fibrosis. The research found that about 5% of children with varying clinical consequences and 11% of those with unknown liability developed CF, highlighting the need to identify specific mutations to provide targeted treatment.
Researchers at Children's Hospital Los Angeles found that targeting survivin enhances chemotherapy effectiveness in retinoblastoma cells and mouse models. The combination of a survivin inhibitor with chemotherapy agents increased Rb cell death, demonstrating a therapeutic advantage.
A CHLA researcher has been awarded a four-year grant to study the development of cone photoreceptors in the retina, which can lead to devastating vision loss due to diseases like retinoblastoma. The goal is to improve modeling of these diseases and develop novel therapies using human embryonic stem cell-derived retina models.
A study by Children's Hospital Los Angeles researchers found that epidermal growth factor (EGF) negatively regulates epithelial cell shedding in the intestine through a selective MAPK-dependent signaling pathway. This insight could identify potential targets for correcting pathological shedding in diseases like IBD.
A new study found that leptin inhibits the development of neuronal connections between the brain and pancreas during embryonic development. This breakdown in communication can lead to impaired glucose regulation and increase the risk of type 2 diabetes in children, particularly those born to obese mothers.
The Saban Research Institute at Children's Hospital Los Angeles has received a $7.1 million grant from the California Institute of Regenerative Medicine to develop an 'off-the-shelf' cellular therapy for enteric neuropathies, which affect the digestive system. The goal is to create nerve cells from human induced pluripotent cells to tr...
A recent study by Children's Hospital Los Angeles found that excess fat accumulation in the lower legs of children with myelomeningocele is primarily associated with muscle-associated adipose tissue. This type of fat has been linked to increased risk for metabolic disorders, including diabetes.
Researchers found that HIV-infected adolescent males had increased levels of sCD14, a marker of macrophage activation, leading to lower bone mass and density. This is due to the protective effects of estrogen in females, which represses macrophage function.
Researchers developed a mouse model to study the effects of abnormal vascular endothelial growth factor (VEGF) levels on lung development. The study found that reduced VEGF signaling can 'turn on' lung disease, even in full-term animals, and replicate key features of neonatal respiratory distress syndrome.
A new study from Children's Hospital Los Angeles found that leukemia therapy leads to a significant drop in bone mineral density during the first month of treatment, affecting the lower spine and legs. This earlier onset than previously thought highlights the need for new approaches to mitigate bone toxicity in cancer patients.
A recent study at Children's Hospital Los Angeles found that standard Body Mass Index (BMI) is insufficient for measuring changes in body composition in children with leukemia. The researchers discovered a correlation between increases in body fat and losses of lean muscle mass during treatment.
The first FDA-approved immunotherapy for pediatric cancer, dinutuximab, has been shown to be equivalent to the commercially produced version and can be made available to all patients. Clinical trials have demonstrated improved event-free survival and overall survival in children treated with the treatment.
A study found a correlation between serotonin gene alleles and brain thinning in individuals at high familial risk of depression. Those with the 'S' allele had lower brain plasticity and were more likely to develop depression when exposed to stressors.
Research shows that inadequate prenatal iron intake can exert subtle effects on infant brain development. Higher dietary iron intake is associated with greater complexity and maturity of cortical gray matter, while lower intake is linked to lesser complexity and more immaturity.
A study using fMRI found that individuals with ASD exhibit different neural activity patterns when viewing emotional faces compared to typically developing participants. This suggests that ASD is not a uniform condition and may be linked to differences in how the brain processes emotional stimuli.
A new study reveals that chemotherapy-induced hearing loss in pediatric brain tumor survivors can lead to significant neurocognitive deficits, including intelligence, executive function, and verbal reasoning skills. Children who received radiation therapy and developed hearing loss are at particularly high-risk for these deficits.
A new study at Children's Hospital Los Angeles has confirmed the neuroprotective effects of hypothermia on newborn brain metabolism, using MRS imaging. The study found that therapeutic hypothermia can almost double the chance of a healthy outcome for infants with HIE.
Researchers at Children's Hospital Los Angeles have successfully engineered a colon from human cells that develops various types of neurons. The study has shown that these specialized neurons can be supplied to tissue-engineered colon, mimicking the enteric nervous system found in native colon.
A pilot study found nearly half of pediatric ED patients exhibit mental health symptoms, underscoring the need for early identification and treatment. The study aims to reduce stigma and provide quality medical care by incorporating mental health screening into emergency department assessments.
Researchers at Children's Hospital Los Angeles found that an admission hematocrit can identify pediatric patients at high risk for bleeding after injury. The study showed a reliable screening tool with low false negative rate and high specificity.
Investigators found that inhibiting TNFR2 can alleviate intestinal inflammation in mice, suggesting a new therapeutic approach for patients with IBD who do not respond to anti-TNF medications. This discovery could lead to new treatment options for the 65% of individuals with IBD who do not respond or become resistant to current therapies.
A study published in Neuropsychopharmacology found lower conflict-related brain activity in adolescents at high risk for psychosis, indicating a potential predictor of psychotic disorders. The researchers used fMRI scans to measure cognitive flexibility and detected reduced activation in key brain regions.
The Healthy Young Men's Study aims to improve HIV care and prevention among Black, Latino, and multiracial gay and bisexual young men. The 5-year study will investigate the use of new technologies and biomedical interventions to prevent new HIV infections in highly impacted communities.
The National Institutes of Health has awarded a five-year, multicenter study to evaluate the long-term outcomes of medical treatment for transgender youth. The study will examine the physiological and psychosocial impact of hormone blockers and cross-sex hormones on this population.
Researchers at Children's Hospital Los Angeles have developed a non-invasive method to classify pediatric brain tumors, allowing for early intervention. The technique uses magnetic resonance spectroscopy (MRS) to identify distinct metabolic features in medulloblastoma subgroups, enabling subgroup-specific therapy.
Researchers at Children's Hospital Los Angeles have created a dynamic functional mouse model for lung injury repair. The novel model used targeted Type 2 Alveolar Epithelial cells (AEC2) to study the effects of cell depletion on lung function and disease.
Researchers at Children's Hospital Los Angeles found that vertebral cross-sectional dimensions in newborn females were 10.6% smaller than males, indicating a predisposition to osteoporosis and scoliosis later in life. This difference may be mitigated with exercise and nutrition.
Researchers at Children's Hospital Los Angeles have shown improved outcomes in using stem cell transplantation to treat juvenile myelomonocytic leukemia (JMML), a rare form of chronic blood cancer. The study found that half of patients can be cured from their disease, and a potential standard conditioning regimen has been identified.
A study published in Journal of Adolescent Health found that transgender youth have sex hormone levels consistent with their assigned gender at birth. The research also highlighted the mental health impacts of living with a gender atypical experience, including depression and suicidal ideation.
A study published in Journal of Telemedicine and Telecare found that robot-assisted telemedicine improved the accessibility of neonatal intensive care specialists. The remote specialist required more time to care for patients due to internet connectivity issues, but overall, there were no significant differences in patient outcomes.
Researchers at Children's Hospital Los Angeles have developed a novel zebrafish model to study short bowel syndrome, which may lead to better care for patients. The model enables the study of adaptation in a cost-effective and efficient manner.
Researchers at Children's Hospital Los Angeles discovered two new locations in HIV's genetic code that affect the virus's ability to infect cells, influencing disease progression. The study found that slight genetic changes to the C2 region and bridging sheet of gp120 molecule can alter receptor usage.
Researchers found that teenagers and young adults with congenital adrenal hyperplasia have significantly more abdominal fat tissue, which increases their risk for harmful conditions such as cardiovascular disease. The study suggests a need for targeted prevention and therapeutics to avoid these adverse effects.
Researchers at Children's Hospital Los Angeles identified a new source of cells that can develop into coronary vessels and found the signaling protein CXCL12 guiding this process. The study may have clinical relevance, helping diagnose abnormal coronary vessels and developing interventional strategies to prevent heart attacks.
Researchers at Children's Hospital Los Angeles found that minimal residual disease alone is not predictive of risk or outcome in children with T-cell leukemia. Despite having few remaining leukemia cells, patients achieved complete remission and had excellent outcomes without intensifying therapy.
Researchers at Children's Hospital Los Angeles found that exosomic miRNAs released within the tumor environment affect resistance to chemotherapy in neuroblastoma. The exchange of specific microRNA called MiR155 between tumor cells and tumor-associated macrophages leads to increased telomerase activity and resistance to chemotherapy.
Researchers at Children's Hospital Los Angeles found that a new recombinant factor VIII fusion protein prolongs the duration of anti-hemophilia factor in children. This allows for reduced frequency of treatment, reducing the burden on patients with severe hemophilia A.
A study published in Nature Neuroscience found correlations between family income and brain structure in children and adolescents, with the poorest families showing the largest differences. The research suggests that increasing access to resources may improve brain development and cognition.
A team of investigators has developed the first fully implantable micropacemaker for fetal use, which has received FDA approval as a Humanitarian Use Device. The device is designed to treat fetuses with complete heart block, a condition that significantly slows the heart rate and impairs its ability to pump blood.
A study of 40 minority youth found a powerful relationship between prenatal PAH exposure and disturbances in brain structure, leading to cognitive and behavioral impairments. The findings suggest that air pollutants contribute to ADHD-like symptoms and other behavioral problems.
Researchers at Children's Hospital Los Angeles have found that liver MRI is more accurate than biopsy in determining total body iron balance. The study suggests that liver MRI can be a safer and less painful alternative to liver biopsy, which has high sampling variability and can cause bleeding.
Researchers have identified a promising new target for treating pulmonary fibrosis, a severe and chronic lung disease. By inhibiting the enzyme prolyl 4-hydroxylase, which promotes collagen production, researchers aim to reduce excessive fibrous tissue growth.
Researchers at Children's Hospital Los Angeles have identified a new role for VEGFA in the metastasis of neuroblastoma. The study found that an increase in bone deposition occurs when NB cells metastasize to the bone, driven by VEGFA, leading to increased bone resorption and severe bone loss.
A new protein-based therapy has been developed to target drug-resistant leukemia cells, with promising results in mouse models and potentially amplifying the potency of standard treatment options. The fusion protein CD19L-sTRAIL selectively binds and delivers a 'death signal' to leukemia cells.
Researchers at Children's Hospital Los Angeles found that neonatal mouse hearts can fully recover normal function after mild injury but fail to regenerate after severe injury. The study suggests that cardiac regeneration strategies should be tailored to the type and severity of heart injury.
A new study reveals that ghrelin, a key regulator of appetite, plays an unexpected role in early brain development and influences long-term appetite regulation. The research suggests a link between gut-brain axis maturation and later susceptibility to obesity, diabetes, and cardiovascular disease.
A new study by researchers at Children's Hospital Los Angeles has shown that tissue-engineered small intestine grown from human cells replicates key aspects of a functioning human intestine. The tissue-engineered small intestine offers a promising treatment for short bowel syndrome, a major cause of intestinal failure.
Researchers identified significant alterations in the thalamus and putamen of premature babies, which may lead to designing effective prevention strategies and early interventional treatments for cognitive disabilities. These findings suggest that brain structure and circuitry changes related to prematurity can affect important functio...
A recent study published in the British Journal of Surgery found that extending surgical resection after two cycles of chemotherapy may be beneficial for some pediatric patients with hepatoblastoma. The research, conducted at Children's Hospital Los Angeles, suggests a potential reduction in complications from chemotherapy.
Research at Children's Hospital Los Angeles found that obese patients with acute lymphoblastic leukemia are more than twice as likely to have minimal residual disease, a strong predictor of long-term survival and disease recurrence. The study suggests modifying chemotherapy regimens for obese patients may improve outcomes.