A new study adds weight to the safety and effectiveness of a gene therapy for hypophosphatasia, a rare inherited disorder that causes abnormal bone development. The treatment, AAV8-TNAP-D10, has shown promising results in mice models, with female mice achieving improvements in bone and teeth at lower doses.
Leona M. Flores, a renowned researcher with expertise in biomedical sciences, has been appointed as the new vice president of research operations at Sanford Burnham Prebys. She will oversee the successful execution of research projects and provide administrative support to advance scientific discoveries.
Researchers at Sanford Burnham Prebys used two sequencing methods to reveal new mRNAs associated with Alzheimer's disease, dementia with Lewy bodies, and Parkinson's disease. The study found vast mRNA isoform diversity in genes related to neurodegenerative diseases.
David D. O’Keefe has been named vice president of research development at Sanford Burnham Prebys, bringing his expertise to nurture a culture of grant writing and collaboration. He will support new assistant professors and advance the institute’s mission by identifying research trends and developing funding strategies.
Three Sanford Burnham Prebys faculty members, David A. Brenner, Randal J. Kaufman, and Tariq M. Rana, are recognized for their significant scientific influence. Their publications have been cited top 1% globally by number of citations.
Researchers at Sanford Burnham Prebys found that pancreatic cancer cells rely on a specific nutrient, glutamine, to fuel their unchecked growth. The study identified two enzymes, aPKC zeta and iota, that play a regulatory role in the process of macropinocytosis, allowing cancer cells to scavenge alternative resources.
A team of scientists, led by Anne Bang, is working to establish clearer connections between genes and their effects on brain function and mental health. They will use high-throughput screening technology to study over 100 genes in brain cells.
Scientists at Sanford Burnham Prebys have developed a clearer picture of how crucial machinery in the human cell's recycling process for obsolete and misshapen proteins—known as proteasomes—are formed. The research team shed new light on how two protein chaperones bind on the top of the alpha subunit ring as it is constructed.
A new study reveals a link between senescent cells and the protein HIRA, which helps pack and unpack DNA. The research team discovered that HIRA is necessary for the cells to begin emitting inflammatory molecules, leading to chronic inflammation in the body.
Researchers at Sanford Burnham Prebys discovered that specific macrophage subpopulations, including TREM2+, are critical for resolving MASH and liver fibrosis. These cells help reduce inflammation, slow disease progression, and promote healing.
A new study reveals that the protein CRTC plays a crucial role in maintaining a healthy heart by ensuring it is neither too thick nor too thin. The research also shows that overexpression of CRTC causes cardiac hypertrophy, leading to adverse cardiovascular events.
Researchers have discovered that SARS-CoV-2 can infect more types of lung cells than previously thought, including those without known viral receptors. The study also found that the lung can independently muster an inflammatory antiviral response without immune system help when exposed to the virus.
A recent study has identified Nup358 as a critical regulator of myeloid cell development, revealing its role in the differentiation process of early progenitors. The findings provide insights into how alterations in Nup358 contribute to blood malignancies and may lead to novel therapies targeting transport machinery like NPCs.
Researchers found that tumor-associated macrophages respond to physical properties of fibrosis by synthesizing injury-associated collagens, resulting in metabolic changes that suppress CTL function. This provides an alternative explanation for why anti-tumor immunity is impaired in fibrotic solid tumors.
Scientists at Sanford Burnham Prebys and Vanderbilt University have identified phosphatidylinositol-5-phosphate 4-kinases (PI5P4Ks) as a key regulator of the hippo pathway, which is dysregulated in cancer. The study suggests that targeting PI5P4Ks may lead to new treatments for cancers with abnormal hippo signaling.
A new AI-based approach, PERCEPTION, uses single-cell transcriptomics to predict patient response to cancer drugs and monitor resistance. The approach was validated in three clinical trials for multiple myeloma, breast, and lung cancer, with promising results.
Scientists at Sanford Burnham Prebys have identified promising links between common HIV drugs and a reduced incidence of Alzheimer's disease. The study analyzed over 225,000 medical records and found that RT inhibitor exposure was associated with a statistically significant reduced incidence and prevalence of AD.
Scientists discovered that tiny brain bubbles called small extracellular vesicles carry more complete instructions for altering cellular function than previously thought. Researchers found nearly 80% of identified mRNAs were full-length, allowing them to be transcribed by recipient cells into viable proteins.
A new study published in npj Antimicrobials and Resistance found that pathogenic bacteria E. coli and A. baumannii employ shared and unique mechanisms to acquire resistance to antibiotics ciprofloxacin and GP6. The researchers developed a method to track the acquisition of drug resistance using whole genome sequencing, which revealed t...
A new 3D bioprinted liver tissue model has been developed to study nonalcoholic steatohepatitis (NASH), a serious complication of nonalcoholic fatty liver disease (NAFLD). The model, created using liver cells from healthy or NASH-diseased donors, displays all characteristics of the disease, including fibrosis.
Researchers at Sanford Burnham Prebys identified a molecular signaling pathway that could enhance current MS treatments by linking vitamin B12 deficiency to MS. The study found that supplementing with brain-targeted B12 formulations may improve drug efficacy for this class of medicines.
Researchers at Sanford Burnham Prebys have discovered Zika's shape-shifting machinery and identified a potential vulnerability. The virus's NS2B-NS3 enzyme complex performs multiple tasks, including breaking up proteins and dividing RNA into single strands, but a drug that blocks its conformational changes could be effective.
Researchers found that inhibiting an enzyme associated with aging accelerates nerve regeneration and restores strength in damaged nerves. By targeting the gerozyme 15-PGDH, they promote neuromuscular synapses and faster recovery from peripheral nerve injuries.
Researchers at Sanford Burnham Prebys have developed a new approach to treat pancreatic cancer by feeding tumors a copycat of the important nutrient glutamine. This method significantly slowed pancreatic tumor growth and prevented the spread of tumors in early clinical trials.
Liver cells become scarring when damaged by conditions like viral hepatitis or excessive drinking, leading to cirrhosis and liver failure. Reversing this process involves targeting the creation of scar tissue and addressing underlying causes, offering new hope for patients with liver fibrosis.
A new grant aims to decipher how diverse breast cancer tumors influence cancer cells' ability to resist treatment. By studying this phenomenon, researchers hope to uncover novel targets for more effective therapies.
Researchers found that mannose inhibits the growth of certain types of cancer cells, making them more vulnerable to chemotherapy. Mannose has been shown to trigger 'honeybee syndrome' in these cells, which impairs DNA synthesis and replication.
Researchers from Sanford Burnham Prebys have identified new genes that contribute to hypoplastic left heart syndrome (HLHS), a rare and life-threatening heart disease. The findings, published in eLife, bring scientists one step closer to unraveling the biology of this complex disease.
A new study from Sanford Burnham Prebys identified a protein called NR2F6 that helps melanoma cells evade the immune system, but also promotes anti-tumor immunity when targeted. The researchers hope to develop new drugs that can block this protein's activity, potentially doubling the effectiveness of immunotherapy for melanoma patients.
A study found that certain genetic mutations are associated with treatment-resistant breast cancer in young women, but not older women. The research proposes a new paradigm for classifying and studying all types of cancer.
Researchers at Sanford Burnham Prebys have discovered a new treatment for autoimmune diseases using LY3361237, an antibody that activates BTLA to inhibit the immune system. The study provides insight into the molecular biology of the immune system and could lead to the development of more effective therapies.
Researchers developed a computational approach to predict diabetic kidney disease using DNA methylation markers from a blood sample. The model can use this information to assess current and future kidney function, enabling early detection and prevention of kidney disease in people with type 2 diabetes.
Researchers at Sanford Burnham Prebys have found a way to revive exhausted T cells, which are crucial for destroying cancerous cells. The new approach targets PSGL-1 protein and slows down T cell exhaustion, significantly reducing tumor growth in mice with immunotherapy-resistant melanoma.
Researchers at Sanford Burnham Prebys have discovered that a 3D view of the genome can reveal genes in tumors that may be future targets for therapy. By visualizing how the genome is organized and arranged within tumor cells, they were able to identify new candidate targets for treatments.
Researchers at Sanford Burnham Prebys have identified a group of proteins called AJSZ that help solve a known problem in cellular reprogramming. By blocking the activity of these proteins, they were able to reduce scarring on the heart and induce a 50% improvement in overall heart function in mice that have undergone a heart attack.
Scientists at Sanford Burnham Prebys have discovered a new treatment approach that can kill prostate cancer cells by targeting the PI5P4Kα enzyme. This finding has significant implications for addressing treatment resistance in prostate cancer and may lead to improved treatments for other cancers.
A study by Sanford Burnham Prebys found that misfolded proteins in liver cells contribute to liver cancer development, potentially increasing the risk of certain patients receiving gene therapies for hemophilia. Improving FVIII folding could decrease this risk and develop safer treatments.
Researchers at Sanford Burnham Prebys have discovered the flexible structure of a key blood protein involved in macular degeneration and other age-related diseases. The study reveals how this protein adapts to changing pressure, leading to calcified plaque deposits characteristic of these conditions.
Researchers at Sanford Burnham Prebys have shown that inhibiting a key metabolic enzyme selectively kills melanoma cells and stops tumor growth. The study identifies GCDH as the crucial enzyme in metabolizing lysine and tryptophan, amino acids essential for human health.
Peter Adams and Bing Ren will map senescent cells in five tissues using state-of-the-art technologies to analyze gene expression and chromatin structures. The project seeks to reveal how and where aging cells accumulate, ultimately generating an atlas that can help develop strategies to prevent and treat age-related diseases.
Timothy Huang has been awarded $2.8 million by the National Institute on Aging to study the SORLA gene and its link to Alzheimer's disease. The project will use human stem cells transplanted into mice to determine how specific mutations impact microglia function.
Jianhua Zhao's five-year, $2.4 million grant will focus on proteolysis, the process cells use to break down and recycle unused proteins, with implications for cancer treatment. Cryogenic electron microscopy (Cryo-EM) will be used to study protein recycling and its structures, opening new avenues for cancer research.
Researchers found significant molecular differences in DNA repair genes and cellular signals controlling cell growth between healthy and tumor tissue of Black and white women. These differences may lead to improved treatment strategies for ER+ breast cancer, including earlier use of CDK inhibitors.
CERC-002, a cytokine neutralizer, has been shown to improve survival rates by 83.9% among hospitalized COVID patients compared to 64.5% with placebo. The treatment targets the immune response early on, preventing disease progression.
Researchers at Sanford Burnham Prebys have received a $3.6 million grant to study antibiotic resistance, employing an innovative approach known as the 'evolution machine.' This project aims to shed new light on how bacteria develop resistance and inform more precise antibiotic prescribing practices.
A study published by Sanford Burnham Prebys found unappreciated changes in brain cell types involving hundreds of thousands of never-before-seen RNAs in individuals with Down syndrome. This breakthrough provides new avenues for understanding both Down syndrome and Alzheimer's disease.
A recent study by Sanford Burnham Prebys and the National Cancer Institute has shown that CRISPR-Cas9 gene editing can favor cells with mutated forms of genes linked to cancer. This highlights the importance of monitoring patients undergoing CRISPR-based gene therapy for cancer-related mutations.
Researchers from Sanford Burnham Prebys have discovered a new source of stem cells that could help treat people living with Alagille syndrome. These 'outside the box' liver stem cells may offer hope for those with the rare genetic disorder, which causes severe liver damage and death.
A new study has identified RNF5 as a key player in acute myeloid leukemia, regulating gene expression through its interaction with protein RBBP4. Inhibiting RNF5 may improve treatment outcomes for AML patients by increasing sensitivity to existing targeted therapies.
Scientists have discovered a protein biomarker in blood that could help diagnose schizophrenia at an early age. The study found high levels of active CRMP2 protein, which is uniquely imbalanced in people diagnosed with the condition.