A consortium of researchers and clinicians aims to develop living cardiac tissues and components for infants and young children with congenital heart disease. The THUMPIN' initiative, supported by a $29.6 million contract, will create living heart tissue that can grow and adapt throughout childhood.
A new study published in Imaging Neuroscience identified a brain circuit consisting of the cerebellum, amygdala, and thalamus that is altered in youth with anxiety. The circuit's altered organization and lower connections were associated with higher anxiety and related disorders. Precision interventions targeting this circuit may impro...
Researchers developed a novel approach to reduce neuropathic pain by targeting specific nerve cell interactions. By disrupting the interaction between voltage-gated sodium channels and ankyrin-G, they reduced pain-like behaviors in mouse models and paved the way for new therapeutics.
A new driver of resistance in HR+ breast cancer has been identified through the discovery of FAH's nuclear role in stimulating CDK9 activity. This finding suggests a possible path to target resistant cancer cells and offers a new biomarker for resistance to CDK4/6 inhibitors.
Researchers at Boston Children's Hospital discovered a receptor-free means of signal transduction, identifying a potentially more effective therapeutic target for treating autoimmune diseases. This new method suggests alternative pathways for cell-to-cell communication, which may lead to the development of new therapeutic treatments.
Researchers at Boston Children's Hospital developed a slow-release injectable drug delivery system that achieved pain-numbing anesthetic effects lasting two to three weeks in rats. The system uses liposomes with multiple compartments, allowing for ultra-slow release of hydrophilic drugs.
Researchers have developed a novel genome engineering method called prime assembly, which enables long DNA fragments to be stitched into precise target positions within living cells. This approach may allow for the development of universal gene therapies that can apply to many patients.
Researchers at Boston Children's Hospital identified a new target responsible for bone marrow scarring in myelofibrosis, a type of blood cancer. Combining traditional therapy with drugs blocking scar formation may improve treatment outcomes. The study found that inhibiting freecycling activity with drugs like hydroxychloroquine can red...
Researchers from Boston Children's Hospital have developed a new gene therapy approach that collects enough stem cells in a single hospital admission, improving turnaround time for genetically altering and infusing cells. The approach has shown long-term stability and safety, with patients not experiencing any adverse effects.
Researchers at Boston Children's Hospital found that fecal transplants from healthy donors reduced peanut allergy symptoms in some patients. The study suggests that the right bacteria can help restore tolerance to food through bile acid metabolites. Increased levels of immune regulatory cells also protected against allergic reactions.
A recent study found that severe COVID-19 reactivates certain dormant viruses in hospitalized patients, which may contribute to autoimmune disease and chronic conditions. Notably, reactivation of Anelloviridae viruses was associated with long-term physical disability and long COVID.
A new strategy makes stem cell transplants safer by replacing chemotherapy-based treatment with targeted antibodies that recognize specific markers on blood-forming stem cells. This approach allows for less toxic treatment and could enable chemotherapy-free or chemotherapy-sparing transplantation approaches.
Boston Children's Hospital has been awarded up to $17.25 million to participate in the Innovative Medicine and Precision Approaches to Clinical Trials (IMPACT) Network, an international effort aimed at speeding up the development of urgently needed treatments for children with profound autism and related neurodevelopmental disabilities...
A new COVID-19 vaccine adjuvant has been developed that can extend vaccine protection for up to two years, reducing the need for repeated boosters. The adjuvant, known as mannadjuvant, was created from a fungal component and showed a more pronounced response against omicron viral components than the vaccine alone.
A study by Boston Children's Hospital researchers found that the US missed four out of seven measles elimination status indicators, with most at risk. The latest outbreak has spread continuously for over a year and affected 45 states since January 2025.
Researchers discover cancer-driving genes in microglia of Alzheimer's patients, suggesting a link between blood cancers and the neurodegenerative disease. The findings could lead to new diagnostic tools and therapeutic options for Alzheimer's.
Dr. Stuart Orkin has been awarded the Breakthrough Prize in Life Sciences for discovering genetic mechanisms underlying hemoglobin production and developing a therapy using CRISPR/Cas9 technology. The therapy, Casgevy, treats patients with sickle-cell anemia and beta-thalassemia, debilitating blood disorders affecting millions worldwide.
Researchers at Boston Children's Hospital have identified a mechanism for immune cells to recycle defective or autoreactive antibodies by editing their genes. The new process uses an enzyme called RAG to swap out light chains with nearby new ones, often making them non-defective and functional.
New research reveals that tumor cells in supratentorial ependymomas cluster into distinct neighborhoods, each with a specific role, such as proliferating or invading. Understanding these cell subtypes could help predict treatment response and inform targeted therapies for this aggressive childhood brain cancer.
Dr. Zon's pioneering research has illuminated how blood develops in embryos, leading to a deeper understanding of rare blood diseases, including pregnancy complications. His discoveries have also led to breakthrough treatments for genetic blood disorders affecting babies and children.
Researchers at Boston Children's Hospital have developed a novel mass spectrometry tool called FLEXITau to analyze brain tissue from 203 patients with various tauopathies. The study identified 145 post-translational modifications and 195 cleavage sites across tau, providing a precise molecular roadmap for diagnostics and drug development.
Glycosylated RNAs on the cell surface form clusters with RNA binding proteins, regulating interactions with surrounding growth factors and controlling angiogenesis. The study found that altering the amount of RNA on the cell surface can modify intracellular signaling cascades through selective interaction with growth factors.
The collaboration aims to deliver life-saving treatments at a fraction of their current market cost, using Caring Cross's decentralized manufacturing model. Clinical data demonstrated robust increases in fetal hemoglobin with sustained mitigation of SCD disease manifestations.
A new study maps the impact of stressors during pregnancy on the developing fetal brain, revealing a cell atlas and identifying key immune pathways. The research sheds light on how maternal gut-immune disruptions can shape neurodevelopmental disorders in children.
A new study finds that infants' brain activity is influenced by their caregivers' reported income sufficiency, with delayed maturation seen in households where income was never adequate. The research suggests that policies strengthening families' basic needs during infancy may have lasting developmental benefits.
Boston Children's Hospital will provide clinical research coordination for the IMPACT Network, a multisite clinical trial infrastructure enabling rapid and effective therapeutic development for autism. The grant, from ARIA, will support the development of study design protocols, recruitment strategies, and efficient workflows.
A study of over 3,500 adolescents found that social jet lag has widespread negative effects on the developing brain's structure and neural circuits. This includes weaker connections between key brain regions responsible for sensory processing, emotion regulation, and social function.
Researchers found that DNA loops facilitate homologous recombination, a key DNA repair process linked to cancer. These loops enable the repair machinery to scan for an intact copy of the damaged region more efficiently.
A study published in Nature Neuroscience found that the same dopamine mechanism responsible for drug addiction also controls natural behavioral fatigue. Researchers used male fruit flies to demonstrate that desensitization of D2R receptors leads to a decrease in motivation and interest.
A new study published in Science finds CTE shares genetic similarities with Alzheimer's disease, including DNA damage and tau protein buildup in the brain. Researchers used single-cell genome sequencing to identify somatic mutations in neurons from patients with CTE, revealing abnormal patterns of damage similar to those seen in AD.
Hereditary spastic paraplegias are a diverse group of over 80 neurodegenerative disorders with limited therapeutic options. Boston Children's Hospital is expanding its research efforts to promote clinical trial readiness for these disorders, thanks to an $8.1M NIH grant.
Researchers discovered craters on the surface of melanoma cells that serve as immune hubs and facilitate local tumor killing. These structures, termed CRATERs, may serve as a more accurate marker of immunotherapy's success in treating solid tumors.
The team achieved rapid genomic sequencing and interpretation within hours, setting a Guinness World Records milestone. This breakthrough has the potential to expedite precise treatments for critically ill babies in the Neonatal Intensive Care Unit (NICU), reducing waiting times from days to hours.
Boston Children's Hospital has received a $35 million donation from Hansjoerg Wyss to further its translational neuroscience research, focusing on rare brain disorders and intellectual disability. The gift will support clinical trials, fellowships, and pilot studies, aiming to develop new therapies for vulnerable children.
Adolescents who frequently prefer solitude show measurable differences in brain structure and function, with weaker connections and greater fragility in social circuits. Clinicians can help families recognize risks by spotting early patterns of persistent withdrawal, opening the door to strategies that may protect mental health.
Researchers at Boston Children's Hospital report an alternative local anesthetic, 2',6'-pipecolylxylidine (PPX), that selectively blocks pain while leaving motor function intact. PPX has been found to be less toxic than conventional anesthetics and may enable longer-lasting pain relief.
The IMPACT-CETR aims to develop preventive strategies against antibiotic-resistant pathogens Staphylococcus aureus, Pseudomonas aeruginosa, and Klebsiella pneumoniae. The project seeks to harness immunity in these bacteria to prevent infections and reduce the emergence of resistance.
A $8.6 million NIH grant has renewed funding for the Rare Disease Clinical Research Network project, focusing on cognition, communication, and neuropsychiatric symptoms in individuals with rare diseases. The initiative aims to develop biomarkers, support pilot projects, and foster skilled investigators.
A new study found that short informational videos on social media can effectively promote HIV prevention among teens and young adults. The study suggests that public health campaigns should combine social media distribution with in-clinic viewing, use positive framing, and involve young people in content development.
Researchers at Boston Children's Hospital have developed a new approach to lengthening telomeres using synthetic RNA. The technique, known as eTERC, has been shown to increase telomere length in human stem cells and leave normal cell mechanisms intact. Additionally, polygenic scores have been developed to estimate the combined effect o...
New data published in JAMA provides further evidence for fetal embolization as a potential treatment for Vein of Galen Malformation. Outcomes show a dramatic reduction in mortality to 43% and no neurodevelopmental delay among treated patients.
Researchers have discovered a natural mechanism by which RNA modifications prevent innate immune activation, raising questions about their potential link to autoimmune disorders. The study found that N-glycans on glycoRNAs block hypermodified RNA bases from inducing immune responses.
Researchers have developed a novel synthetic ligand using AI-based computational protein design, activating the Notch signaling pathway in T-cells. This breakthrough enables broadened clinical T-cell production and advances immunotherapy development.
A multicenter study found influenza-associated acute necrotizing encephalopathy (ANE) carries a high risk of morbidity and mortality in children, with a 27% mortality rate. Survivors often experience neurological difficulties, emphasizing the need for rapid treatment and vaccination to prevent ANE.
A new study published in PNAS found that prolonged and repeated exposure to GABA anesthetic agents in infants under two months leads to accelerated visual cortical development. This may suggest the use of non-GABA-active anesthetics for newborns, as a large multicenter clinical trial is currently underway.
Researchers discovered a key molecular sensor detecting changes in intracellular pH that regulates immune defense against bacterial infections and influences inflammatory diseases and cancer. The findings may lead to innovative strategies for disease prevention and treatment by altering how the internal environment shapes immune function.
Scientists have identified a potential treatment strategy for ADOA by targeting the SARM1 protein, which triggers axon degeneration in retinal ganglion cells. Deleting or disabling SARM1 preserves RGCs and maintains vision in a mouse model.
The Autism Inpatient Collection (AIC) has released a comprehensive dataset of over 1,500 children with autism, ages 4-20, providing valuable insights into their phenotypic and genetic profiles. The data includes measures of behavior, communication, emotion regulation, and more.
Researchers find inhibiting TMEM219 signaling restores mucosal healing in inflammatory bowel diseases and protects intestinal stem cells from death. The study suggests TMEM219 overactivation induces intestinal stem cell death, preventing mucosal renewal during inflammation.
The Boston Children's Therapeutic & Medical Device Accelerator has announced its inaugural awardees, selected for their groundbreaking innovations in pediatric healthcare. The program provides targeted funding, expert mentorship, and strategic collaborations to advance hospital discoveries towards clinical impact.