Researchers developed a machine learning model that predicts which intrinsically disordered protein regions form condensates in cells, finding they are linked to RNA biology and occur in only about 12% of human proteins. The model, IDR-Puncta ML, provides a valuable resource for studying condensate formation in cancer and RNA biology.
The Spatial Touchstone project has developed a standardized framework for spatial transcriptomics analysis, providing critical metrics and quality control measures. The repository includes curated datasets from six tissue types, paired with an open-source software tool to compare samples across institutions.
A multi-institute team led by St. Jude Children's Research Hospital has discovered how certain drugs used to treat epilepsy affect their target, a membrane protein found in nearly all neurons. The study provides guidance for therapeutic optimization and sheds light on a poorly understood but therapeutically important protein.
A new technique from St. Jude Children's Research Hospital provides the most complete picture of a person's entire T-cell repertoire at 10% of the cost of pre-existing approaches, enabling more scientists to study immune responses.
19 St. Jude scientists were named to the 2025 list of Highly Cited Researchers, demonstrating significant influence in their investigative fields. The recognition highlights the institution's commitment to fostering big science and advancing breakthroughs in childhood cancer research.
The AlphaSync database provides updated predicted protein structures on a regular basis, ensuring scientists can work with the most current information. The database contains 2.6 million predicted protein structures across hundreds of species and is updated using the latest data from UniProt.
The study identified two major factors that improve survival for children with IHG: achieving a complete surgical resection and cancer-directed adjuvant treatment. Early surgery in infants was fraught with risks of bleeding and early oncologic death, highlighting the need for strategies to minimize surgical morbidity.
Researchers at St. Jude Children's Research Hospital identified 117 kinases that can phosphorylate multiple locations within the RNA polymerase II protein tail, greatly expanding upon previous knowledge. This discovery links enzyme activity to multiple diseases, including cancer, and reveals a more integrated role for cell signaling.
Scientists have identified an alternative binding site on nuclear receptors, allowing for the design of more selective PROTACs. This discovery offers new therapeutic options for treating diseases linked to nuclear receptors, such as cancer and drug resistance.
Scientists at St. Jude Children's Research Hospital developed a new approach to safely reduce therapy in children with medulloblastoma, a pediatric brain tumor. The analysis of almost 900 patients identified four actionable treatment recommendation groups from low to high intensity.
Scientists at St. Jude Children's Research Hospital identified a mechanism behind pro-cancer gene expression in a high-risk AML subtype. Inhibiting Exportin-1 showed promise as a novel strategy to treat UBTF-TD AML, which is characterized by treatment resistance and relapse.
Researchers found that a structurally modified version of the drug florfenicol exploits drug resistance mechanisms in Mycobacterium abscessus to amplify its effect, perpetually. This approach presents a potentially safer and more effective way to treat certain antibiotic-resistant infections, minimizing host mitochondrial toxicity and ...
Researchers found significant overlap in ultrarare genetic variants between ALS and HSP, including newly identified genes contributing to disease risk for both conditions. This study highlights the shared origins of these disorders and may lead to more personalized care for patients.
Researchers at St. Jude Children's Research Hospital found that metabolism guides the activation states of regulatory T cells, which dampen inflammation. The study reveals a link between mitochondrial function and lysosome activity in controlling these immune cells.
The study reveals how heat shock chaperone proteins Hsp40 and Hsp70 bind each other and misfolded peptides, enabling the cellular machinery to work. The findings also identify a specific region of Hsp40 that handles protein handoffs, which could lead to therapeutic interventions for diseases.
Scientists at St. Jude Children's Research Hospital discovered that protein ASPL regulates stress granule disassembly by facilitating VCP phosphorylation and stabilizes interactions among core stress granule proteins. Additionally, ASPL facilitates stress granule assembly independent of VCP by stabilizing G3BP interactions.
St. Jude recognized as one of the top 10 pediatric cancer hospitals in US, consistently earning a place among the nation's best for pediatric oncology. The hospital maintains top survival rates for childhood cancers and drives progress in treatment and survivorship.
Researchers found that pasteurization kills the H5N1 virus and prevents oral tolerance, reducing health risks; unpasteurized milk poses a significant threat, highlighting the importance of proper food safety methods.
A new high-throughput screening approach has enhanced CRISPR genome-editing efficiency by identifying promising CAST variants. The method allows for rapid optimization of these candidates, uncovering mechanistic insights that can inform further engineering and potential clinical use.
Researchers at St. Jude Children's Research Hospital have implicated lipids in the initiation of LC3-associated phagocytosis (LAP), a specialized process for degrading dead cells and microbes. This finding opens new avenues for exploring therapeutic strategies to modulate LAP in diseases such as cancer and autoimmune disorders.
Researchers from St. Jude Children's Research Hospital have made a breakthrough in treating neuroblastoma by combining immunotherapy with the drug indisulam, achieving complete therapeutic responses in laboratory models regardless of cell state.
A new multi-disciplinary approach reveals that some mitochondrial DNA mutations occur before a cell turns cancerous and can influence both leukemia onset and progression. The study also suggests that these mutations may contribute to therapy resistance, making leukemia cells more likely to resist treatment.
Researchers discovered that disordered regions of the ZFTA-RELA fusion protein cause the formation of droplets within cells called condensates, which are essential for ependymoma development. The study provides a new therapeutic frontier by targeting the interacting partners within these condensates.
Researchers at St. Jude Children's Research Hospital found that midkine protein prevents Alzheimer's disease by blocking amyloid beta assembly growth. Midkine inhibits amyloid beta elongation and secondary nucleation, two specific phases during assembly formation.
A clinical trial shows that giving risdiplam to newborns as early as 16 days old is safe and effective in improving motor function and increasing survival rates. The treatment can delay or prevent disease progression and preserve strength, function, and quality of life from birth.
St. Jude researchers developed a computational method to design tandem CARs that target two cancer-related proteins, overcoming previous challenges and function better in treating animal models of cancer. The approach demonstrated improved surface expression and anti-tumor function, clearing tumors in four out of five mice.
St. Jude receives its third consecutive Magnet designation and first Magnet Designation with Distinction, recognizing its commitment to exceptional nursing care and patient outcomes. The hospital's nursing programs, including the Nurse Residency Program and Center of Advanced Practice, have contributed to its success.
Researchers at St. Jude Children's Research Hospital used cryo-electron microscopy to study the full range of motion of sweet taste receptors, discovering a previously unknown mechanism of activation that complements unbound and bound structures of the receptor.
Researchers at St. Jude Children's Research Hospital identified virtual memory T cells, a specialized group of immune cells, which provide nonspecific immunity for infants early in life. These cells are a bridge between innate and adaptive immunity, allowing the adaptive immune system time to develop true memory.
Scientists found H5N1 viruses in dairy cows are more similar to bird flu strains than human influenza viruses, indicating a low risk of pandemic threat. However, direct contact with infected animals or consuming raw milk poses a risk of transmission.
The study reconciles two closely related mechanisms in transcription, revealing they are deeply intertwined with shared driving forces. The findings suggest that while transcription can occur via both mechanisms, phase separation does not necessarily offer increased activity, but rather dampens it.
The study found that the stage of normal cell development at which B cells transform into leukemic cells impacts treatment outcomes for pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL). Researchers identified a 'multipotency score' to predict clinical outcomes, providing valuable insights into drug resistance and str...
A new computational tool called ColdBrew predicts the likelihood of water molecule positions within experimental protein structures, aiding drug discovery efforts. The tool leverages data on extensive protein water networks, providing insights into how ligands bind to proteins and potentially improving binding sites.
Gene therapy has been shown to significantly improve blood flow in the brains of patients with sickle cell disease, decreasing the risk of stroke. The treatment has a more substantial and long-lasting protective effect than other treatments like hydroxyurea or blood transfusions.
Researchers found common variants in TTN and BAG3 associated with reduced late-onset cardiomyopathy in childhood cancer survivors, similar to the general population. However, rare variants linked to early-onset cardiomyopathy in adults showed no association with late-onset risk in children.
Researchers at St. Jude Children's Research Hospital have identified a novel combination therapy approach to treat pediatric acute myeloid leukemia (AML) fueled by NUP98 fusions. Targeting the complex alone or in combination with another anticancer drug significantly increased survival in AML model systems.
A new study found that childhood cancer survivors' genetics and therapy type play a significant role in determining their risk of developing a second cancer. Radiation exposure was the most significant contributor to secondary cancer risk, accounting for about 40% or more of the risk.
Researchers show that stress granules, previously implicated in neurodegenerative diseases, can actually suppress fibril formation and even reverse the effects of disease-causing mutations. This discovery offers new insights into the development of potential treatments for neurodegenerative diseases.
Charles G. Mullighan, a leading leukemia researcher at St. Jude Children's Research Hospital, has been elected Fellow of the Royal Society. His work on genomic research has advanced the understanding and treatment of acute leukemia in children.
Scientists at St. Jude Children's Research Hospital discovered that TEAD proteins promote differentiation by switching partners with INSM1 as neural progenitor cells mature. This finding highlights the complexity of brain development and the importance of accounting for context in studying neurodevelopment processes.
Researchers found that infants with severe COVID-19 have distinct immune responses, including high levels of interferon and inflammatory cytokines. This unique response may help protect or contribute to the severity of the disease, requiring further studies.
A new study reveals how a deficient ELP1 gene predisposes children to SHH-medulloblastoma, a subtype of malignant pediatric brain tumor. Researchers identified an MDM2-targeted therapy that reawakens the anticancer protein p53, potentially extending survival in model systems.
Richard Finkel, MD, is named to TIME100 Health for his pioneering work in treating spinal muscular atrophy prenatally with the orally administered drug risdiplam. This breakthrough has shown promising results, with no identifiable features of SMA observed two years after treatment.
Scientists at St. Jude Children's Research Hospital created a cortical organoid model for the rare disorder UBA5-associated encephalopathy, revealing stunted GABAergic interneuron growth and imbalance in neuronal excitation and inhibition. The model suggests boosting the expression of the existing partially functioning copy of UBA5 may...
Scientists developed a data science framework to understand how cells travel through the body by analyzing chemokines and G protein-coupled receptors. They found that specific positions in structured and disordered regions determine how these proteins bind, allowing for rational alteration of cell migration.
Scientists at St. Jude Children's Research Hospital discovered FOXR2 activation in multiple pediatric CNS tumor types, including high-grade gliomas and pineoblastomas, with significantly different clinical outcomes. The study highlights the importance of combining molecular findings with other diagnostic approaches to improve treatment...
Researchers at St. Jude Children's Research Hospital have designed a new drug framework that selectively targets the CYP3A4 enzyme, reducing off-target effects. The discovery provides a roadmap for future drug developers to better evaluate drug interactions and selectively target CYP proteins.
Researchers used cryo-electron microscopy to visualize the dynamic motion of a human chromatin remodeler in action, capturing 13 distinct structures that reveal the full picture of nucleosome sliding. This comprehensive view sheds light on how chromatin remodeling affects gene access and expression.
A new study finds that more than half of all pediatric cancer deaths occur in regions of armed conflict, with a significant impact on diagnosis and treatment. Conflict disrupts healthcare systems, leading to delays and increased mortality risk.
Researchers at St. Jude Children's Research Hospital found that removing the 'signal jammer' protein VDAC2 can improve how tumors respond to immunotherapy. This breakthrough could lead to new ways to enhance immunotherapies and make them more effective in treating resistant cancers.