SELECT SCIENTIFIC DISCOVERIES
Nature Structural and Molecular Biology
Structure of a eukaryotic CRISPR-Cas9 homolog, Fanzor2, shows its promise for gene editing
• Gene editing tools like CRISPR-Cas9 have revolutionized science, but adapting them for eukaryotic cells (like human cells) remains complex due to variations in molecular structures.
• St. Jude researchers analyzed the structure of the eukaryotic gene-editing protein Fanzor2 using advanced imaging and biochemical techniques to understand more about the evolution of RNAguided nucleases.
• The team uncovered unique features of this CRISPR-Cas homolog that could make gene editing in eukaryotic cells more precise and efficient.
• This breakthrough underscores the pioneering role St. Jude plays in advancing cutting-edge molecular biology tools to accelerate discoveries in human health.
Learn more: Structure of a eukaryotic CRISPR-Cas9 homolog, Fanzor2, shows its promise for gene editing
Science
Disrupting Asxl1 gene prevents T-cell exhaustion, improving immunotherapy
• T cells can become “exhausted” during prolonged immune responses, reducing their effectiveness in immunotherapy.
• Researchers targeted the Asxl1 gene, which influences T-cell exhaustion, using genetic tools to disrupt its function.
• Blocking Asxl1 prevented T-cell exhaustion, leading to more robust and sustained immune responses and opening new avenues for improving cancer immunotherapies.
• This groundbreaking work by St. Jude demonstrates expertise in epigenetics and cancer immunotherapy innovation.
Learn more: Disrupting Asxl1 gene prevents T-cell exhaustion, improving immunotherapy
Elizabeth Kellogg, PhD
Caitlin Zebley, MD, PhD | Ben Youngblood, PhD
Immunity
Nutrients and organelle signaling shape tissue immunity
• Understanding how immune cells adapt to local tissue environments is crucial for improving immunotherapies.
• Using advanced CRISPR screens, scientists explored how nutrients and organelle signaling regulate memory T cells in intestinal tissues.
• The research revealed critical nutrient-dependent cellular processes that enhance the functionality of tissue-resident memory T cells, key players in immunity.
• This multidisciplinary approach championed at St. Jude sets the standard for understanding immune cell function in complex environments.
Nature Communications
Tumor suppressor forms gel-like assemblies to sacrifice cancer cells
• Cancer cells evade death by disrupting tumor suppressor pathways.
• Scientists investigated the behavior of the tumor suppressor p14ARF, which interacts with other proteins to arrest cancer cell growth.
• The research revealed that p14ARF forms gellike structures (biomolecular condensates) that suppress ribosome production, effectively sacrificing cancer cells.
• The cutting-edge exploration into biomolecular condensates at St. Jude redefines approaches to cancer biology and therapeutic targeting.
Learn more: Nutrients and organelle signaling shape tissue immunity
Learn more: Tumor suppressor forms gel-like assemblies to sacrifice cancer cells
Hongbo Chi, PhD
Richard Kriwacki, PhD
Science
Glutamine metabolic switch is key to red blood cell development and disease
• Understanding metabolic shifts during red blood cell formation can help address diseases like sickle cell anemia and β-thalassemia.
• Researchers examined how cells transition from breaking down glutamine to producing it during red blood cell development.
• The study identified this metabolic switch as critical for healthy red blood cell formation and a potential target for treating related disorders.
• This fundamental discovery links cellular metabolism to disease, offering new avenues for therapeutic interventions.
Nature Communications
St. Jude researchers identify unexpected genetic link between skin disorder and inner ear
• Dyschromatosis universalis hereditaria (DUH) is a rare skin pigmentation disorder linked to the protein ABCB6. DUH is also associated with other symptoms ranging from learning difficulties to diabetes, as well as hearing impairment.
• Scientists at St. Jude Children’s Research Hospital investigated ABCB6 in two model organisms, uncovering a potential link between ABCB6 and the inner ear.
• Importantly, one of the models with hearing disruption demonstrated no pigmentation issues, implying the two symptoms appear independently of each other.
• The findings suggest a novel link between inner ear development and ABCB6, with disruption of this process potentially leading to hearing deficits.
Learn more: Glutamine metabolic switch is key to red blood cell development and disease
Learn more: St. Jude researchers identify unexpected genetic link between skin disorder and inner ear
Jian Xu, PhD | Min Ni, PhD
John Schuetz, PhD
JACC: CardioOncology
Predicting cardiac issues in cancer survivors using a serum protein panel test
• Childhood cancer survivors face an increased risk of cardiac issues later in life due to treatments that, while lifesaving, can cause long-term damage to the heart.
• St. Jude researchers developed a serum protein panel test that analyzes specific biomarkers in the blood. This test allows for early and precise identification of survivors at higher risk for cardiac dysfunction.
• This innovation could improve traditional methods by providing a targeted, non-invasive and more accurate tool to predict heart-related complications.
• This marks another significant advancement in survivorship care from St. Jude, supported by the St. Jude LIFE study, which tracks thousands of cancer survivors over decades.
Nature Communications
Research links a genetic risk for schizophrenia to a malformed skull
• Schizophrenia has long been associated with genetic and developmental factors, yet the biological pathways linking genetic risk to the disease remain poorly understood.
• Researchers at St. Jude leveraged advanced imaging and genetic analysis techniques to explore how genetic factors and skull malformations intersect during early brain development.
• The study identified specific gene variants that influenced brain development by affecting skull shape, suggesting a mechanical link between skull development and neurological traits.
• This work underscores a commitment to uncovering the roots of complex diseases like schizophrenia and translating those findings into therapeutic insights.
Learn more: Predicting cardiac issues in cancer survivors using a serum protein panel test
Learn more: Research links a genetic risk for schizophrenia to a malformed skull
Yadav Sapkota, PhD
Stanislav Zakharenko, MD, PhD
Daniel Blair, PhD
Nature
Universal barcodes unlock fast-paced small molecule synthesis
• Developing new medicines requires creating and testing many chemical compounds, which is often slow and expensive, making it harder to find new treatments quickly.
• St. Jude scientists developed an innovative chemical tagging method using “universal barcodes” that enable high-throughput synthesis and screening of small molecules.
• The new method has drastically reduced the time and complexity of creating diverse small molecules, fostering faster identification of promising therapeutic candidates with enhanced precision and scalability.
• By combining cutting-edge technology, interdisciplinary collaboration and robust infrastructure, St. Jude is pioneering advancements in small-molecule therapeutics.
Learn more: Universal barcodes unlock fastpaced small molecule synthesis
St. Jude names M. Madan Babu, PhD, senior vice president and chief data scientist
St. Jude Children’s Research Hospital announced M. Madan Babu, PhD, FRS, as the institution’s first Chief Data Scientist, Senior Vice President for Data Science and leader of the newly formed Office of Data Science. This $195 million research enterprise will have 115 new positions. In his new role, Babu will bring new, advanced computing technologies and data science approaches to biomedical research. His team will also facilitate the integration of biological and biomedical data, foster a data science research culture and build international collaborations.
St. Jude ranks in top 10 of U.S. News Best Children’s Hospitals for Cancer
St. Jude Children’s Research Hospital was recognized today by U.S. News & World Report as one of the top 10 pediatric cancer hospitals for the 17th straight year. Every year since 2008, the publication has evaluated about 100 hospitals with pediatric oncology programs and ranked the top 50 as part of its annual list of Best Children’s Hospitals. Hospitals are evaluated based on clinical data and an annual survey of pediatric specialists. The survey also evaluates compliance with best practices and patient outcomes.
Mitchell Weiss of St. Jude Children’s Research Hospital
elected to the National Academy of Medicine
Mitchell J. Weiss, MD, PhD, chair of the Department of Hematology and Arthur Nienhuis Endowed Chair, has been elected to the National Academy of Medicine. Membership in the Academy is considered one of the highest honors in the fields of health and medicine. In his nomination, Weiss was cited for his leading research in the biology of blood cell formation and how noncancerous blood diseases disrupt the process. His recent translational studies aimed at developing new treatments for blood disorders using novel genome editing and base editing tools.
Scientists from St. Jude Children’s Research Hospital listed among most cited globally
Eleven scientists from St. Jude Children’s Research Hospital are among those recognized on the 2024 list of Highly Cited Researchers. The annual list is compiled by the Web of Science™ together with qualitative analysis performed by experts at the Institute for Scientific Information (ISI)™ at Clarivate. A Highly Cited Researcher is considered one who has demonstrated significant and broad influence in their fields of investigation. The list represents a small fraction of scientists worldwide who have disproportionately contributed to extending the boundaries of knowledge through innovation and discovery.
St. Jude appoints leading scientist to create groundbreaking Center of Excellence for Structural Cell Biology
St. Jude Children’s Research Hospital announced the addition of Georgios Skiniotis, PhD, as a faculty member in the Department of Structural Biology. Skiniotis will also spearhead the newly created Center of Excellence for Structural Cell Biology. In his role as director of the Center of Excellence, Skiniotis will develop a world-class technology center that will advance our understanding of cell biology from the atomic scale to the micron scale, including the implementation of emerging capabilities in cryogenic electron tomography (cryo-ET) and volume electron microscopy (vEM) imaging. This will bridge the strengths of St. Jude in structural biology and cell biology and define the leading edge of innovation in this new field.
St. Jude names Maria Megdal as executive vice president and chief administration officer
St. Jude Children’s Research Hospital has appointed Maria Megdal as the hospital’s executive vice president and chief administrative officer. Medgal will oversee departments vital to St. Jude operations, including Human Resources; Information Services; Diversity, Equity and Inclusion; Environmental Services; Facilities Design and Construction; Facilities Operations and Maintenance; Food Services; Biomedical Engineering; the Gift Shop; and Security.
First Sickle Cell Gene Therapy Patient, 12, Leaves Hospital
After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a treatment, their difficult experiences hint at what others will be up against.
Doctors, nurses and friends of Kendric Cromer, center, cheered as he was discharged from Children’s National Hospital in Washington after receiving a novel gene therapy treatment to cure his sickle cell disease.
How a childhood cancer drug shortage makes the unimaginable worse
Doctors told Laura Bray that she was “lucky” because her 9-year-old daughter’s leukemia was curable.
Abby had a 90% chance of beating the blood cancer if she followed a three-year treatment. There was just one problem. Doctors told Bray the key drug that kicked off Abby’s chemotherapy in 2018 was hard to find. She had to figure out a way to tell her daughter.
The family eventually found that medication. Later, two others Abby needed were hard to find. The Brays, like thousands of families across the country, got a firsthand education in the widespread shortage of children’s cancer drugs.
Bird flu begins its human spread, as health officials scramble to safeguard people and livestock
Almost from the beginning of the spread of H5N1 bird flu among farms and ranches in the U.S. earlier this year, experts and researchers warned that a critical lag in the blood testing of exposed workers might lead to an underestimation of the virus’s potential transmission to humans.
Those warnings have proved prophetic. And the federal Centers for Disease Prevention and Control (CDC) now finds itself not only trying to blunt the spread of the virus, but also playing catch-up with testing methods that have been largely resisted among America’s farmers.
Bird flu risk to humans: Canadian case has unsettling viral mutations
The virus, which appears not to have spread to anyone else, underwent mutational changes virologists didn’t want to see.
The genetic sequence of the H5N1 bird flu virus that infected a teenager in British Columbia shows that the virus had undergone mutational changes that would make it easier for that version of H5N1 to infect people, scientists who have studied the data say. The source of the teen’s infection has not been determined, so it’s impossible to know for sure if the mutations were in the virus that infected him or her. But scientists think it is more likely that the mutations developed during the course of his or her infection.
CDC: Fewer parents vaccinating kindergartners, disease rising
Fewer parents in the U.S. vaccinated their kindergartners as more sought exemptions from routine shots over the past school year, new data shows.
The figures, released Wednesday by the Centers for Disease Control and Prevention, follow trends since the COVID-19 pandemic, with more parents foregoing children’s vaccines that have been instrumental in saving early lives. In turn, preventable diseases have resurfaced across the U.S.
Study cautions radiologists not to over-rely on AI tools for diagnosis
Researchers also found that physicians were more likely to trust an AI explanation if it pinpointed a specific area on an X-ray.
While artificial intelligence (AI) is a revolutionising tool in medicine, radiologists may over-rely on its advice when it highlights a specific part of an X-ray, according to a new study. A team of US researchers recruited 220 physicians across multiple sites in the country tasked with reviewing chest X-rays alongside AI-generated advice.
St. Jude Researchers Pioneer
New Liquid Biopsy Panel Targeting Pediatric Cancers
NEW YORK – Researchers at St. Jude Children’s Research Hospital have developed a non-invasive comprehensive genomic profiling assay designed specifically for pediatric cancers, publishing results of an initial validation study in Nature Leukemia earlier this month.
St. Jude names 1st chief data scientist, creates $195M research enterprise
M. Madan Babu, PhD, has been appointed the inaugural chief data scientist of St. Jude Children’s Research Hospital in Memphis, Tennessee.
Dr. Babu was also named senior vice president for data sciences and will lead the hospital’s newly formed Office of Data Science, according to an October 1 news release from St. Jude.
The new $195 million research enterprise will create 115 new positions, the release said. In his new role, Dr. Babu will bring data science approaches and advanced computing technologies to biomedical research, the release said.
