Researchers at Boston Children's Hospital used stem cell technology to create Purkinje cells from patients with tuberous sclerosis complex, a genetic syndrome often linked to autism. The lab-grown cells showed structural abnormalities and impaired development of synapses, which may help explain how autism develops at the molecular level.
Scientists have isolated a new botulinum neurotoxin, BoNT/En, from a strain of Enterococcus, sparking concern over the potential for multi-drug resistance. The toxin, found in cow feces, was unexpectedly discovered to resemble the toxin that causes botulism, with experts warning of its potentially deadly consequences.
Cancer researchers have identified CDK12 inhibitors as a potential treatment option for Ewing sarcoma, a rare and aggressive childhood cancer. The inhibitors show promise in killing Ewing sarcoma cells and can be combined with PARP inhibitors to increase effectiveness. This breakthrough brings hope to the pediatric oncology field, whic...
Conventional cancer therapy can create an inflammatory cascade in the body, leading to aggressive tumor progression and recurrence. However, resolvins have been shown to counteract these effects, enhancing the body's clearance of cell debris and reducing tumor growth.
Researchers developed an implanted robotic system that applies traction forces to stimulate tissue growth in stunted hollow organs. The device was tested on pigs and showed a 77% increase in esophagus length, promoting cell proliferation without disrupting organ function.
A randomized trial showed that an alternative, potentially one-time operation called endoscopic third ventriculostomy with choroid plexus cauterization (ETV/CPC) is as good for infant brain development as placing a shunt. The study found no significant difference in cognitive scores between the two groups after 12 months.
A new study found that person-to-person genetic differences can impact the efficacy of gene editing technologies like CRISPR-Cas9. The researchers analyzed 7,444 whole-genome sequences and discovered that about 50% of guide RNAs could be affected by variants at their target sites.
A study by Boston Children's Hospital and Harvard Medical School found that brain mutations increase with age, especially in people with genetic premature aging disorders. The researchers detected 'clocklike' mutations that accumulated like clockwork in both brain areas, as well as those linked to oxidative damage and faulty DNA repair.
Scientists have made a breakthrough in treating neurodegenerative disorders and lysosomal storage diseases by transplanting hematopoietic stem cells directly into the brain, achieving therapeutic benefits faster than traditional methods. This innovative technique could pave the way for new treatments for Parkinson's, Alzheimer's, and o...
A new gene therapy transplantation technique could improve treatment of neurodegenerative diseases by generating genetically engineered microglia-like cells in the brain. This technique has been tested on an experimental model for a metabolic disease and may have future therapeutic applications for other neurodegenerative diseases.
Researchers at Boston Children's Hospital are developing new models to test HIV vaccines, enabling the rapid evolution of human antibodies in mice. This approach could lead to a protective version of broadly-neutralizing antibodies in uninfected individuals through preventative immunization.
Scientists at Boston Children's Hospital have identified the molecule responsible for releasing interleukin-1 (IL-1) from immune cells. The study found that gasdermin D enables IL-1 release without killing the cell, which could lead to more potent and long-lasting vaccines.
Researchers at Boston Children's Hospital have developed a soft robotic system that can provide isolated support to the right or left ventricle, addressing one-sided heart failure. The system combines rigid bracing with soft robotic actuators to help diseased heart chambers pump blood effectively.
A new study from Boston Children's Hospital suggests that eating allergenic foods during pregnancy can protect children from food allergies when breastfed. The research found that antibodies in breast milk transferred to offspring helped induce food tolerance, and the findings support recent guidelines rejecting advice to avoid highly ...
Researchers at Boston Children's Hospital have successfully reversed type 1 diabetes in a mouse model by infusing pre-treated blood stem cells. The treated cells curbed the autoimmune reaction and reversed hyperglycemia in diabetic mice, with almost all mice cured of diabetes in the short term and one third maintaining normal blood sug...
Researchers analyzed anonymous Google location data to understand human mobility patterns before and after Hurricane Maria. Travel out of Puerto Rico increased by 20% the day before the storm, with people flocking to cities like Orlando and Miami for shelter.
Killer cells use a methodical approach to destroy bacterial invaders, inflicting oxidative damage and targeting critical proteins with the deadly enzyme granzyme B. The discovery offers new insights into how immune systems combat bacteria, potentially leading to the development of new antimicrobial drugs.
A recent study found that genetic variants in epithelial sodium channels may help rehydrate the airways of cystic fibrosis patients, reducing bacterial build-up and promoting stable lung function. This discovery brings ENaCs into the spotlight as a potential new therapeutic target.
Researchers developed a growth-accommodating implant that can expand in tune with a child's natural growth, decreasing the number of heart surgeries a child must endure. The novel design, inspired by nature, could also be adapted for other growth-accommodating implants throughout the body.
Researchers report that a gene therapy to treat cerebral adrenoleukodystrophy has effectively stabilized the disease's progression in 88 percent of patients. Fifteen out of 17 patients had stable neurologic functioning more than two years after receiving the treatment.
Researchers at Boston Children's Hospital found that specific neurons in the retina specialize in detecting different ranges of light intensity, allowing the brain to infer overall illumination levels. This complex system involves 'non-image' vision cells that function even in people with blindness.
Researchers developed a device to assess mitochondrial oxygenation, predicting cardiac arrest in critically ill heart patients. The device uses resonance Raman spectroscopy to quantify oxygen levels and has been shown to accurately predict cardiac arrest with 97% specificity.
Researchers at Boston Children's Hospital have developed a new DNA nanoswitch platform that can detect biomarkers associated with different diseases, viral strains, and genetic variabilities. The NLISA system uses gel electrophoresis to screen synthesized DNA reagents that change shape in the presence of a specific biomarker.
Researchers discovered mesenchymal cells drive lung cell formation through molecular interactions, guiding the production of alveolar and airway cells. This finding offers new avenues for treating lung disorders like asthma and emphysema.
A genetic mutation in the CLPX gene has been identified as a potential cause of erythropoietic protoporphyria (EPP), a form of porphyria that may have inspired vampire folklore. The discovery highlights the complex genetic network underlying heme metabolism and holds promise for future therapies.
The current approach to quality measurement in healthcare is characterized by inefficiency and imbalance, with measures that are duplicative, overlapping, or overrepresent certain areas of care. A lack of consideration for the cost of each measure hinders the ability to assess its value.
A team of researchers has made a breakthrough discovery in understanding autoimmune diseases, revealing that rogue B cells can trigger an 'override' of the body's tolerance to its tissues. This finding sheds new light on epitope spreading, a hallmark aspect of autoimmune disease where the immune system attacks multiple organs and tissues.
The von Willebrand factor molecule undergoes a two-step transformation to activate blood clotting in response to changes in blood flow, which can help prevent hemorrhage or life-threatening blood clots. The findings could inspire smart drugs that target only diseased areas of the body, reducing the risk of excessive bleeding.
A team of scientists at Boston Children's Hospital has developed a therapeutic cocktail that promotes axon regrowth after spinal cord injury or stroke, resulting in improved fine motor skills. The treatment showed significant improvement in mice with either condition, with error rates dropping to 30% and 46% respectively.
Researchers discovered that targeting arginase 2, an enzyme involved in nitrogen metabolism, can curb the growth of pancreatic tumors, especially in obese individuals. The study suggests a novel approach to treating this deadly cancer, with potential for improved survival rates and therapeutic windows.
Researchers designed a therapeutic fusion protein that triggers increased S1P receptor activity, recovering blood vessel health in mice with various vascular diseases. The combination of ApoM-Fc and S1P reduced blood pressure and mitigated tissue damage.
Researchers at Boston Children's Hospital developed a novel system using ultrasound to trigger the release of nerve-blocking agents, providing on-demand pain relief. The system, using ultrasound-triggered liposomes, has potential for replacing addictive opioids and short-lasting local anesthetics.
A recent study using three genetic sequencing technologies found that late-breaking mutations occurring after conception are linked to autism spectrum disorder (ASD). These mutations, known as post-zygotic mutations, were discovered in a subset of cells and disproportionately affect the amygdala.
A recent study published in Scientific Reports found a correlation between slower placental oxygen transport and lower newborn birth weights, brain, and liver volumes. Researchers used MRI to map oxygen delivery across the placenta in real-time, revealing differences in regional function that can predict fetal growth.
Researchers at Boston Children's Hospital have discovered a mechanism that links chronic inflammation to mental illnesses like anxiety and depression. Their study suggests that anti-IFNAR drugs may be effective in reducing symptoms of CNS lupus and other related conditions.
A recent study led by Boston Children's Hospital suggests that repealing the individual mandate in AHCA could undermine dependent coverage for young adults. Without an individual mandate, more young adults may lose health insurance and struggle to maintain coverage as they transition into adulthood.
A new study in American Journal of Public Health predicts seasonal influenza outbreaks one month earlier than before using a Thermia online health educational tool and iThermonitor wearable device. Data collected from 45,000 users revealed real-time detection of 'influenza-like illnesses', improving disease surveillance.
Scientists have successfully generated blood-forming stem cells in the lab using pluripotent stem cells from patients' own cells. The advance opens up new opportunities to create immune-matched blood cells and potentially treat genetic blood disorders.
Researchers have discovered a small molecule called hinokitiol that can transport iron across cell membranes, potentially treating iron deficiency and overload. This breakthrough has significant therapeutic potential for addressing various iron-related disorders.
Researchers found that chronic sleep loss significantly exacerbates pain sensitivity in mice, but using caffeine or wakefulness-promoting agents like modafinil can block this effect. These findings suggest a new approach to chronic pain management by addressing both sleep and daytime alertness.
A new computational approach developed at Boston Children's Hospital uses Granger causality analysis to identify the brain regions responsible for seizures. This technology enables surgeons to plan surgeries more accurately and reduce the risk of complications.
Researchers at Boston Children's Hospital report achieving strong vaccine responses in newborn animals and monkeys using a new adjuvant formulation. The study demonstrates the potential for a single-dose vaccine to protect against life-threatening infections, promising to reduce infant mortality globally.
A survey of over 17,000 parents of hospitalized children found variability in quality of care across US children's hospitals. The study highlights opportunities for improvement in communication, family engagement, and patient safety.
A new study by researchers at Boston Children's Hospital has successfully used stem-cell-derived cells to identify potential treatments for the rare blood disorder Diamond Blackfan anemia. The team found a promising compound called SMER28, which was able to get live mice and zebrafish to produce red blood cells.
Researchers have successfully restored a high level of hearing in deaf mice using an improved gene therapy vector, which also improves balance function. The technology has the potential to benefit patients with Usher syndrome, who suffer from profound hearing loss and balance problems.
A massive international study of over 750,000 people has uncovered 83 rare genetic variants that significantly affect human height. The findings shed light on previously unknown biological pathways involved in skeletal growth, with some variants influencing height by more than 2 cm.
Researchers at Boston Children's Hospital have identified a potential therapeutic solution for age-related fibrosis by pinpointing the gene responsible for the condition. Deletion of the PAD4 gene has been shown to curb fibrosis in mice, reducing organ dysfunction and improving heart function.
A soft robotic sleeve has been developed to mimic the natural compression motion of healthy cardiac muscles, restoring acutely failing hearts to 97% of their original output. The device attaches to the outside of the heart without contact with blood, reducing the risk of complications and infection.
A large study from Boston Children's Hospital found that epigenetic modifications to DNA, specifically DNA methylation, are linked to an increased risk of obesity-related health problems. The researchers identified 83 sites in the genome where methylation changes were associated with differences in energy balance and lipid metabolism.
Chelating zinc has been shown to promote survival of neurons in the retina and stimulate repair of damaged nerve fibers. In a mouse model, researchers saw dramatic levels of axon regeneration after using zinc chelators.