Scientists at The Children's Hospital of Philadelphia reprogrammed gene expression to produce fetal hemoglobin, which is unaffected by the genetic defect in sickle cell disease. This approach may open the door to a new treatment option for patients with this debilitating blood disorder.
A personalized cell therapy reprograms a patient's immune system to eliminate tumors in blood, showing complete responses in 89% of patients with high-risk ALL. The treatment has also been shown to persist in circulation and prevent cancer recurrence.
A novel gene variant associated with severe childhood asthma has been discovered through a genome-wide association study. The study found that the CDHR3 gene is particularly active in epithelial cells lining airways and may disrupt normal functioning, leading to increased risk of asthma.
Biomedical researchers can now explore complex data sets with ease, thanks to CHOP's open-source platform, Harvest. The toolkit provides a user-friendly interface to navigate different types and levels of data, allowing users to construct queries to slice and dice data.
A new national study found that children receiving solid-organ transplants have a high risk of developing advanced kidney disease. The study suggests the need for continued screening and preventive measures to slow or prevent disease progression.
Researchers studying chromosome 22q11.2 deletion syndrome aim to uncover genetic variation and identify biological pathways leading to schizophrenia in the broader population. The project may inform novel, more effective treatments for psychiatric disorders.
A study of over 2,200 children's hospital visits reveals racial and ethnic disparities in pain management and length of stay for abdominal pain. Black and Hispanic children were less likely to receive analgesics, while black patients had shorter hospital stays compared to white children.
Researchers at the Children's Hospital of Philadelphia developed a new approach to cancer immunotherapy by adjusting the function of immune cells. They showed that inhibiting the enzyme p300 can reduce Treg cell function and limit tumor growth in mice, offering potential for anti-tumor therapy.
A large new study found that current non-invasive techniques for respiratory support are less effective in reducing severe lung injury in very premature infants. The study refutes the common assumption that these therapies are reducing severe lung injury and BPD in tiny babies.
A new study from Children's Hospital of Philadelphia reports proton therapy as an effective, organ-sparing treatment option for children with high-risk neuroblastoma. Protons precisely target tumors while minimizing radiation exposure to healthy tissue.
Researchers have successfully reprogrammed skin cells from patients with rare blood disorders, such as Diamond Blackfan anemia and juvenile myelomonocytic leukemia, into induced pluripotent stem cells (iPSCs). The iPSCs can be used to understand the underlying mechanisms of these diseases and test innovative treatments.
New research from The Children's Hospital of Philadelphia estimates a potential surge in medically-attended injuries, particularly among currently uninsured young adults who will now be able to access affordable care. The study predicts an increase in demand for treatment among younger patients with mild and moderate injuries.
A study from The Children's Hospital of Philadelphia found a central signaling response in mitochondrial energy diseases, which may offer a common avenue for targeted treatments. Nicotinic acid partially restored normal functioning in cells taken from patients with mitochondrial disease.
Researchers have developed a bioengineered decoy that fools the immune system and prevents it from neutralizing the benefits delivered by a corrective gene. The approach could potentially increase the number of patients who can be treated with gene therapy, offering new hope for genetic diseases like hemophilia.
A study by The Children's Hospital of Philadelphia found that educating pediatricians about appropriate antibiotics for common respiratory infections led to a significant decrease in broad-spectrum prescribing. This intervention improvedRx guideline compliance, resulting in better antibiotic use and potentially reduced resistance.
A 8% reduction in BMI improves insulin sensitivity and glucose tolerance in obese teenagers, a threshold found consistent with adult results. The study suggests that modest weight loss can lead to significant improvements in metabolic health.
Children who underwent surgery to remove their adenoids and tonsils showed significant improvements in behavior, quality of life, and other symptoms compared to those treated with watchful waiting. However, there was no difference between the groups in attention and executive functioning.
Scientists have created a model cell system that allows them to investigate normal brain development and identify specific disruptions in biological signals contributing to neurodevelopmental disorders. The new model uses human embryonic stem cells to produce cortical interneurons, which control electrical firing in brain circuits.
Researchers from CHOP and Penn found that efavirenz is more effective than nevirapine in suppressing HIV in children aged 3-16 in low-resource settings. The study, published in JAMA, suggests a shift in pediatric HIV treatment guidelines could be warranted.
The US can reduce serious injury and death by optimizing rear seat design with technologies like load limiters and pretensioners, as well as modifying the geometry of the rear seat. The report also recommends policies and programs to increase rear seat restraint use.
A new teaching technique using scripted debriefings improved medical knowledge and instructor ratings among novice pediatric CPR trainers. The 'advocacy inquiry' method helped participants reflect on their decisions, leading to better performance in emergency situations.
A physician-researcher from The Children's Hospital of Philadelphia will lead the first-ever pediatric Dream Team to combine genomics and immunotherapy to increase cure rates in difficult childhood cancers. The team aims to develop more precise and effective treatments based on unique genetic characteristics of each child's tumor.
A new report from The Children's Hospital of Philadelphia and State Farm shows encouraging trends among teen passengers. In 2011, over half of teen passengers reported always buckling up, and risky behaviors declined by 23-47% between 2008-2011.
Children's Hospital of Philadelphia collaborates with Pfizer's Centers for Therapeutic Innovation (CTI) to advance pediatric research and development. The partnership aims to identify preclinical research with potential applications for innovative treatments, with a focus on translating scientific insights into bedside therapies.
Researchers found that different mutations in the same gene may require different treatments for child brain tumors. A new study identified a second-generation BRAF inhibitor that targets specific mutations, offering hope for personalized medicine.
Researchers at Children's Hospital of Philadelphia report complete remission in two pediatric ALL patients treated with novel cell therapy. The treatment, known as CTL019, uses engineered T cells that target a specific protein on the surface of leukemia cells, achieving a potent anticancer effect.
Infants with autism show subtle, measurable differences in attention as early as 7 months old. Researchers found a specific neural circuit causes the slower response, pointing to a problem with 'sticky attention'.
Pediatric oncologist Garrett Brodeur will receive the Pediatric Oncology Award and deliver a lecture highlighting his work on predicting disease course in neuroblastoma. His research has led to better understanding of the cancer's biology and development of more effective treatments.
Paul M. Weinberg, a pediatric cardiologist at The Children's Hospital of Philadelphia, received the 2013 Distinguished Teacher Award for innovative and compassionate teaching. He has made significant contributions to cardiovascular medicine through his extensive research and educational efforts.
A study by Children's Hospital of Philadelphia researchers found that children with congenital heart disease exhibit impaired growth, measured in weight, length, and head circumference. The study suggests that abnormalities in overall growth regulation play a role in poor growth, potentially related to factors beyond nutrition.
Researchers identified seven gene variants associated with human longevity, including deletions and duplications that impact alternative splicing. These variations may provide protection against diseases, allowing individuals to live longer.
A research team developed a one-step gene test that analyzes both nuclear and mitochondrial DNA to help evaluate the genetic cause of suspected mitochondrial disease. The new tool may shorten the diagnostic odyssey experienced by patients and families seeking the cause of debilitating symptoms.
A new software tool called ParseCNV detects copy number variation (CNV) associations with higher accuracy than existing software, enabling better identification of gene variants linked to genetic diseases. The algorithm automatically corrects for variations in DNA sequences, producing high-quality results.
A recent study published in Circulation found that longer CPR durations can extend survival in children and adults after hospital cardiac arrests. The research team analyzed data from thousands of patients and discovered that more children than expected survived prolonged CPR, with over 60% having good neurologic outcomes.
A recent study published in Nature Genetics found that neuroblastoma tumors undergo rapid evolution, rendering traditional targeting strategies less effective. Researchers refocused on understanding the dynamic nature of these tumors and their response to medicine.
The Children's Hospital of Philadelphia has been awarded a $2 million grant from the Patient-Centered Outcomes Research Institute to study the effectiveness of different antibiotic treatment options for children returning home after hospitalization. The research aims to inform healthcare decisions and improve patient outcomes.
Researchers identified 25 additional copy number variations associated with autism, which could serve as predictive markers. The study found that these variants have a strong effect in raising an individual's risk for autism and may be incorporated into clinical tests.
Research from PolicyLab at The Children's Hospital of Philadelphia shows that a home visiting program may not be effective in reducing early childhood injuries. Children served by the Nurse-Family Partnership had no fewer injuries than comparable families not enrolled in the program, with some even experiencing higher injury rates.
Researchers at Children's Hospital of Philadelphia use bioengineered T cells to destroy leukemia cells, achieving a complete response in a 7-year-old girl. The treatment, known as CTL019, also stimulates an immune response and can be combined with immunomodulating drugs to reduce side effects.
Researchers identified four new gene regions linked to low birth weight, influencing adult height, type 2 diabetes risk, and blood pressure. The study's findings suggest a strong genetic influence on fetal growth and potential lifelong health benefits from prenatal interventions.
Researchers have discovered two related genes, ARID1A and ARID1B, involved in the most aggressive form of childhood cancer neuroblastoma. These findings identify a novel pathway that scientists can now study to develop potential new therapies for children with tumors having these mutations.
A study of ER docs and pediatricians found a need for concussion-specific training and infrastructure to support optimal patient care. The Children's Hospital of Philadelphia created a new 'medical home' model for managing pediatric concussions, incorporating standardized clinical decision support tools and patient education.
A large-scale international study has identified three new gene variants associated with body mass index (BMI) levels in adults, providing fundamental insights into the biology of adult obesity. The study used a versatile gene discovery chip to detect gene variants involved in lipid-related diseases and other disorders.
Pediatricians at CHOP developed a three-part survey to help families and doctors make informed decisions about ADHD treatment. The results show that shared decision-making improves healthcare outcomes, but is not widely used in pediatric settings.
A new study from The Children's Hospital of Philadelphia found that New Jersey's Graduated Driver Licensing (GDL) decal requirement lowers crash rates among intermediate teen drivers. The study showed a 9% decrease in police-reported crashes and an 8% decrease in multiple-vehicle crashes after the decal's implementation.
A new study by pediatric researchers found that parents with higher stress levels are more likely to have obese children who eat fast food frequently and engage in less physical activity. The study suggests that interventions targeting parental stress and teaching coping skills may help address childhood obesity.
A large international study has identified 21 new gene variants associated with risks of heart disease and metabolic disorders. The findings expand the list of potential targets for drugs and other treatments for lipid-related cardiovascular disease, a leading global cause of death and disability.
The study found a significant increase in newborn mortality rates in Philadelphia after the closures, with rates rising from 5 deaths per 1,000 to 8 per 1,000. After 2000, the rates returned to pre-closure levels, but the outcomes did not improve over baseline.
A study found that prenatal diagnosis of congenital heart defect can cause significant stress, but healthy relationships and positive coping mechanisms can reduce this stress. Lower partner satisfaction and income were associated with higher levels of depression and anxiety.
The Center for Autism Research at Children's Hospital of Philadelphia has received a $2.2 million NIH grant to study brain development in infants as early as three months old. Researchers aim to identify biomarkers for autism and develop more effective treatments.