Researchers at St. Jude Children's Research Hospital and Rockefeller University have gained a better understanding of the cystic fibrosis transmembrane conductance regulator (CFTR). The new findings reveal how CFTR functions mechanistically and how disease mutations affect its function, paving the way for more effective therapies.
Scientists at St. Jude Children's Research Hospital demonstrate a framework to develop solutions to evade detoxification networks in drug development, potentially reducing side effects. By altering the structure of a small molecule, they found a way to stretch out binding regions, making it energetically unfavorable for drugs to bind a...
The study reveals new details about the intraflagellar transport (IFT) complexes, including previously unknown zinc-binding sites in IFT-A. The high-resolution structures of IFT-A and Tubby-related protein 3 (TULP3) can now be used to investigate developmental diseases involving cilia.
A recent study by St. Jude Children's Research Hospital found that socioeconomic deprivation and frailty are independently associated with increased late mortality in childhood cancer survivors. The research highlights the importance of policies to improve local environments and access to interventions that may improve health conditions.
Researchers found associations between cancer treatments and accumulated mutations in blood cells, a sign of accelerated aging called clonal hematopoiesis. This accumulation increases the risk of blood cancer and death in long-term childhood cancer survivors.
Scientists at St. Jude Children's Research Hospital developed a new design for chimeric antigen receptors (CARs) by adding a molecular anchor, increasing the anti-cancer activity of cellular immunotherapies in cancer models. The anchored CARs improved cancer killing and survival rates in animal models of multiple tumor types.
Researchers at St. Jude Children's Research Hospital used a next-generation protein degradation technology to study CTCF, revealing its functional insights into transcription regulation. The AID2 system overcame limitations of previous approaches, identifying specific zinc finger domains responsible for CTCF-dependent transcription.
Researchers at St. Jude Children's Research Hospital used cryo-electron microscopy to capture the first 3D structure of SPOP, a protein mutated in prostate and endometrial cancers. The study revealed previously unknown interfaces that harbor cancer-causing mutations, shedding light on how SPOP drives cancer.
A comprehensive study of childhood leukemia's genetic diversity provides insights into personalized therapy. Researchers identified distinct groups based on drug sensitivity profiles, which significantly impact prognosis.
Researchers created a mouse model that demonstrates how RNA splicing defects contribute to neurodegeneration in Alzheimer's disease. The findings show that dysfunctional U1 snRNP leads to excitatory toxicity and accelerates cognitive decline, opening new avenues for treatment.
Researchers found a direct connection between low oxygen conditions and increased fetal hemoglobin production in adults. A drug that activates the cellular response to low oxygen could potentially treat sickle cell disease by promoting HbF production, which has therapeutic benefits.
A study from St. Jude Children's Research Hospital found that nearly half of childhood cancer survivors experience multiple simultaneous symptoms of moderate to severe intensity. Health inequity issues, such as lack of education or insurance, were associated with a higher risk of experiencing severe symptom burden.
Scientists at St. Jude Children's Research Hospital identified the mechanism behind superior auditory abilities in Williams-Beuren Syndrome patients as hyperexcitable interneurons in the auditory cortex. The study suggests that reducing neuronal hyperexcitability might be a general mechanism for treating WBS through targeting VIPR1.
Scientists at St. Jude Children's Research Hospital have created a tool to identify 'safe harbor' sites for gene therapies, balancing safety and therapeutic expression. The tool uses genomic and epigenetic information to find regions with high variability among healthy individuals, reducing the risk of cancer or other problems.
Researchers have discovered that group 3 and group 4 medulloblastoma arise from the rhombic lip, a structure present in early cerebellum development. This finding helps better understand the biology of the disease and develops new research models to guide therapeutic targets.
Scientists at St. Jude Children's Research Hospital studied 36 families affected by Hodgkin lymphoma and identified 44 novel genetic variants linked to cancer predisposition, including PAX5, GATA3, IRF7, EEF2KMT, and POLR1E. The study provides new insights into the disease and may help identify potential targets for new treatments.
Scientists identified a key event in persister cell survival, finding that cytochrome c release kickstarts the integrated stress response pathway, promoting gene expression that prolongs survival. This process may lead to cancer recurrence and resistance to other treatments.
Researchers sequenced 2,574 pediatric ALL patient samples, identifying 376 significantly mutated genes that potentially drive cancer development. The study provides a foundational guide for physicians and scientists to understand disease development and improve treatment outcomes.
SAFER Ukraine successfully evacuated over 900 patients and families to reestablish medical care abroad. The effort demonstrates the importance of collaborative networks in global health and provides a model for future international emergency responses.
Researchers discovered that targeting the protein complex cBAF can increase memory T-cell generation, leading to improved CAR-T cell efficacy against solid tumors. By understanding the early decision points in T-cell activation, scientists can create more effective cancer therapies.
Scientists at St. Jude Children's Research Hospital developed an algorithm to identify temperature-sensitive conformations in proteins, revealing the importance of water networks in ligand binding sites. The findings challenge the assumption that well-resolved cryogenic water positions are both precise and accurate.
St. Jude researchers will present novel treatments for pediatric brain tumors, including a response rate of 86.8% for high-risk neuroblastoma patients. The hospital will also share findings on disparities in survival rates and improve childhood cancer outcomes worldwide through the World Health Organization Global Initiative.
Researchers found that ZBP1, a gene involved in innate immunity, contributes to negative effects of interferon treatment and high interferon levels in some COVID-19 patients. Deleting the gene Zbp1 in mice infected with coronavirus prevented cell death and mortality during interferon therapy.
Scientists used cryogenic electron microscopy to capture multiple states of the Kv4 ion channel, revealing a previously unknown mechanism of inactivation that differs from other VGICs. This finding may help researchers identify therapeutic strategies for neurological and cardiac conditions linked to mutations in Kv4.
Researchers at St. Jude Children's Research Hospital have discovered a DNA-independent mechanism that allows CD8+ T cells to produce both effector and memory cells, enabling the immune system to mount an immediate and long-term response to infections. The study reveals how a specific protein complex guides translation of an important i...
Scientists have identified a novel drug target that may help overcome resistance to EZH2 inhibitors in cancers with SMARCB1 mutations. By inhibiting the KDM2A gene, cancer cells can regain sensitivity to EZH2 inhibition, offering new hope for effective treatment.
Researchers at St. Jude Children's Research Hospital found that EGFR inhibitor treatment can target and kill persistent cancer cells in rhabdomyosarcoma, a type of soft tissue cancer common in children. The study's results support a new clinical trial strategy for treating the disease.
Researchers used single-molecule imaging to study GPCR activation, gaining insight into cellular signal relay and potential drug targets for various disorders. The findings show that the receptor tail plays an autoinhibitory role, controlled by agonist binding, which affects signaling intensity and duration.
Scientists at St. Jude Children's Research Hospital found that COVID-19 vaccination still expands T-cell memory and immune activation in individuals who have already experienced the virus, even if they were infected first. Vaccination also helps protect against new strains of the virus.
Scientists at St. Jude Children's Research Hospital identified two genes associated with accelerated aging in childhood cancer survivors. The study used genome-wide association studies to find variants that correlate with epigenetic age acceleration, a measure of the difference between biological and chronological age.
Scientists have discovered a new subtype of relapsed pediatric AML characterized by a specific gene mutation called UBTF exon 13 tandem duplication (UBTF-TD), which is associated with poor outcomes and an increased incidence of minimal residual disease. This mutation can be used to identify high-risk patients and guide treatment.
Scientists at St. Jude Children's Research Hospital and the Cancer Research Center in Spain have discovered a potential strategy to prevent B-ALL, the most common childhood cancer, using the drug ruxolitinib. By blocking IL-7 signaling, researchers were able to significantly reduce the risk of leukemia development in genetically predis...
Scientists at St. Jude Children's Research Hospital identified a 'modular' super-enhancer that controls gene expression during retina formation, revealing four distinct regions with different functions. This discovery provides a way to study gene expression during development and offers a blueprint for studying brain development.
A phase II clinical trial at St. Jude Children's Research Hospital found significantly higher survival rates for children with high-risk neuroblastoma treated with chemoimmunotherapy and the humanized monoclonal antibody hu14.18K322A, resulting in a three-year disease-free survival rate of 73.7% and an overall survival rate of 86%.
St. Jude researchers have identified a possible risk for late effects of treatment with the experimental cancer drug CX-5461, which targets topoisomerase II beta (TOP2β) instead of RNA polymerase 1. The study highlights the need for close monitoring of patients enrolled in ongoing clinical trials.
Researchers at St. Jude Children's Research Hospital have developed a test to detect measurable residual disease (MRD) in children treated for medulloblastoma, identifying those at risk of relapse earlier than traditional imaging scans. The study found that cell-free DNA in cerebrospinal fluid can be used to predict relapse and potenti...
Researchers at St. Jude Children's Research Hospital found that the tumor suppressor gene PTEN controls rhabdomyosarcoma cell identity and that enhancing PAX7 expression can maintain tumor cell existence, providing a potential treatment target for rhabdomyosarcoma.
A recent study published in Genome Biology found direct evidence that CTCF governs chromatin accessibility, a critical process for gene expression. By using cutting-edge technologies and a multidisciplinary approach, researchers discovered CTCF's role in rewiring genome-wide chromatin assembly.
Researchers at St. Jude Children's Research Hospital have identified the PANoptosome, a complex that integrates multiple cell death pathways to protect against infections. This discovery offers new insights into the functional role of inflammasome sensors in innate immune responses.
A recent study found that 20% of children with cancer infected with SARS-CoV-2 developed severe infections, compared to 1-6% in general pediatric patients. The Global Registry of COVID-19 and Childhood Cancer also showed that cancer care was disrupted, with 56% of patients having modified treatment and 45% having chemotherapy withheld.
Researchers at St. Jude Children's Research Hospital have developed a more accurate laboratory model for studying retinoblastoma, a rare pediatric eye cancer. The models closely mimic the biology of patient tumors and provide an important resource for studying the earliest stages of the disease as well as screening new therapies.
A large phase 3 noninferiority clinical trial showed that omitting pulse therapy in patients with low-risk disease improves quality of life without affecting survival. This finding is significant as it reduces neuropsychological side effects and late effects associated with long-term treatment.
A study by St. Jude Children's Research Hospital demonstrates the power of comprehensive whole genome, whole exome and RNA sequencing to better understand and treat each patient's unique cancer. The research found that 86% of patients had at least one clinically significant variation in tumor or germline DNA.
Researchers found thiopurine-induced mutations in leukemia cells, inactivating a DNA repair process and making the cancer resistant to multiple chemotherapy drugs. This study provides direct genomic evidence of chemotherapy-induced drug resistance mutations in pediatric cancer.
Scientists at St. Jude Children's Research Hospital have found a link between post-cancer treatment frailty and neurocognitive decline in young adult childhood cancer survivors. Frailty contributes to early onset of neurocognitive decline, which can influence daily activities.
Researchers at St. Jude Children's Research Hospital identified a metabolic control pathway that regulates T follicular helper cells, offering targets for drugs to stimulate the adaptive immune response and treat autoimmune diseases such as lupus. The finding holds promise for developing new vaccines and treatments.
Researchers analyzed molecular analysis data from a pediatric melanoma registry, identifying diverse subgroups and their characteristics. The study found that spitzoid melanomas often have gene fusions and benign clinical courses, while conventional melanoma patients with advanced disease had poorer outcomes.
Scientists at St. Jude Children's Research Hospital studied enhancers that regulate Foxp3 gene expression to control effector and killer T cells. They found that these enhancers work together to curb the immune cells' activity, ensuring they only attack cancer cells.
Researchers found that retinoblastoma survivors who received treatment began to recover their early learning and life skills by age 10. The study highlights the importance of early intervention to promote growth and development in young children with retinoblastoma.
A new study by St. Jude Children's Research Hospital found that COVID-19 vaccination significantly reduced asymptomatic SARS-CoV-2 infections in employees, with a 79% risk reduction compared to unvaccinated peers.