Researchers at Weill Cornell Medicine have assembled a comprehensive atlas of messenger RNA variants in the mouse and human brain, revealing intricate patterns of gene expression and its relationship to major brain disorders. The study provides valuable insights into brain development, neuron specialization, and disease mechanisms.
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.
Researchers developed a prediction tool to classify proteins based on their potential to bind RNA G-quadruplexes, showing high protein disorder and hydrophilicity. This discovery provides insights into gene expression and phase-separation into membrane-less organelles.
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Researchers unveil innovative strategies to overcome metabolic constraints in CAR-T cell therapy, aiming to boost its efficacy in treating solid tumors. Metabolic interventions targeting immunosuppressive metabolites, metabolite uptake, and mitochondrial metabolism are proposed to enhance anti-tumor activity.
In nerve cells, insulin facilitates the elimination of defective mitochondria when energy is available. However, during energy scarcity or disrupted insulin signaling, mitochondrial recycling is reduced, allowing potentially damaged power plants to continue operating. This process affects ageing processes and neurological diseases.
Researchers have identified a subset of T-cells that acts like stem cells and continuously generates effector T-cells that attack transplanted organs. Targeting the transcription factor IRF4 may lead to innovative therapies for patients with chronic infections, cancers, autoimmune diseases and transplanted organs.
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Researchers analyzed molecular data from brain tissue and blood of individuals who committed suicide, identifying risk markers for novel therapeutic targets. The study found alterations in the prefrontal cortex and inhibitory neurotransmitters, highlighting potential new approaches for preventing suicidal behavior.
Researchers create a technique using prime editing to quickly and easily screen cancer genes, revealing new information on p53 mutations. The method allows for the analysis of over 1,000 different mutations in the tumor suppressor gene p53, which are seen in more than half of all cancer patients.
Researchers found that T cells can reshape their memory and maintain diversity against COVID-19 variants in response to successive mRNA vaccinations. The study revealed a shift among clonotypes, with a change from early responders to main responders after the second shot, suggesting a new dominant population of effector-memory T cells.
Scientists at St. Jude Children's Research Hospital found that ruxolitinib, a drug inhibiting both Janus Kinase 1 (JAK1) and JAK2, is a safe and potentially more effective therapy for Hemophagocytic lymphohistiocytosis (HLH), a rare and aggressive syndrome of hyperactive inflammation. The study showed that ruxolitinib increased surviva...
Research from UCSF found COVID antigens lingering in blood up to 14 months and tissue samples for over two years after infection. The persistence of these fragments may drive long COVID symptoms and associated risks.
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Researchers at John Innes Centre used cryo-EM to visualize the structural architecture of chloroplast RNA polymerase and build a detailed atomic model. The study reveals new insights into transcription, a fundamental step in making photosynthetic proteins, and how these proteins interact with DNA and mRNA.
UCSF scientists discover delivering therapeutic molecules to amniotic fluid can effectively treat Angelman syndrome and other neurological conditions. The treatment uses antisense oligonucleotides, which can alter gene expression, and has shown improved motor function and learning outcomes in mice.
Researchers at the University of Pennsylvania have developed a new method to efficiently determine which lipid nanoparticles are likely to bind to the lungs, rather than the liver. This breakthrough enables targeted delivery of mRNA therapeutics beyond the liver, offering new hope for treatments of cystic fibrosis and lung cancer.
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Researchers at Aarhus University discovered that RNA modification N4-acetylcytidine (ac4C) plays a key role in stress granule formation and function. Acetylated transcripts are localized to stress granules, regulating their assembly and dispersal.
Researchers created novel gene editing enzymes with improved precision, reducing off-target RNA edits by over 99%. The technology has potential applications in treating mitochondrial genetic diseases and may lead to transformative treatments within the next five years.
A new study aims to enhance and prolong vaccine effectiveness by delivering adjuvants to white blood cells using lipid nanoparticles. The research, led by WVU professor Sharan Bobbala, has the potential to provide broader protection against evolving viruses and multiple diseases.
Researchers developed a novel assay that integrates data from four fusion callers to identify disease-related gene fusions in pediatric tumors with high accuracy and efficiency. The new bioinformatics platform detected fusions, prioritized them, and custom curated downstream processes for consensus fusion calling.
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An experimental mRNA vaccine against CMV elicited promising immune responses, outperforming a previous vaccine candidate in preventing the virus from infecting epithelial cells. The new vaccine may protect adults against CMV, potentially preventing women from passing the infection to their babies during pregnancy.
Researchers developed a faster test for mpox using CRISPR and nanopore sensing technology, which can detect the virus in 32-55 minutes, compared to hours of current lab testing. The test is specific to mpox and was confirmed not to detect other viruses.
Researchers discovered two new genetic pathways associated with worse ICH outcomes, including a pathway affecting blood coagulation and cellular interactions. Population data showed a decrease in aneurysmal SAH strokes and a rise in non-aneurysmal subarachnoid hemorrhages, offering encouraging evidence for intervention success.
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Researchers from Tokyo University of Science identify Cpeb4 protein's crucial role in mRNA splicing and osteoclast differentiation, shedding light on bone disease mechanisms. The study's findings may lead to new diagnostic techniques and treatments for conditions like osteoporosis.
Researchers created a DNA-based vaccine that mimics the structure of a virus, inducing a strong antibody response against SARS-CoV-2. The vaccine uses a DNA scaffold carrying viral proteins, allowing the immune system to focus on the target antigen.
Researchers have identified regional biological signatures in invasive brain tumor margins of high-grade glioma, which could lead to improved diagnosis, prognosis, and treatment. Advanced MRI techniques may help distinguish between the genetic and molecular alterations, providing insights into resistance to treatment.
A new American Heart Association scientific statement emphasizes the need for increased awareness of cerebral venous thrombosis (CVT) to facilitate prompt medical treatment. The statement updates guidelines for diagnosing and managing CVT, focusing on advancements in treatment approaches.
A pilot study proposes a promising global genomic assay for diagnosing molecular subtypes in pediatric B-ALL, leading to more accurate diagnosis and targeted treatment options. RNA sequencing analysis accurately identified subtypes in all known cases and determined genetic subtype in 79% of previously unknown cases.
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A study in rats found that excessive adolescent stress alters genes associated with energy metabolism, leading to behavioral problems and psychiatric disorders in adulthood. The prefrontal cortex's response to stress was also affected, suggesting a link between mitochondrial function and emotional regulation.
A study reveals thyroid cancer's genetic changes contribute to resistance to BRAF inhibitors and can lead to tumor dedifferentiation. Researchers identify potential targets for new therapies, including dual-targeted treatments and immunotherapy combinations.
A new study using CRISPR technology enables researchers to activate genes in easily accessible cells, providing a potential breakthrough in the diagnosis and understanding of rare genetic diseases. This method could revolutionize the process by enabling faster results within weeks.
Researchers developed an mRNA therapeutic that combats ovarian cancer by producing functional p53 protein, shrinking and killing tumors. The treatment is effective against metastases and has shown promise in preclinical studies.
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Researchers found that high levels of FSP1 are associated with relapse after cisplatin treatment in head and neck squamous cell carcinoma. Targeting FSP1 can attenuate tumor stemness and downregulate invasion and metastatic rates, suggesting a potential new approach for treating drug-tolerant persister cancer cells.
Researchers analyzed LTBR expression levels in various cancers, finding it associated with low patient survival and immune cell infiltration. The study identified LTBR as a potential target for cancer immunotherapy and marker of poor prognosis.
A team of researchers identified a CTP-dependent transcription factor controlling Shigella virulence gene expression, providing new avenues for combating this and related bacterial pathogens. The discovery sheds light on the molecular mechanisms underlying bacterial pathogenesis.
A new study reveals that RNA binding motif protein X-linked (RBMX) plays a crucial role in predicting cancer prognosis and response to immunotherapy. Abnormal expression of RBMX is associated with immune regulation, tumor microenvironment, and therapeutic effects of immune checkpoint inhibitors.
A new computational tool, SNAF, has been developed to identify shared splicing neoantigens that can help a patient's immune system fight cancer. The tool has already uncovered promising targets for melanoma and other cancers.
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Yiliang Ding's pioneering work on RNA structure and function has led to breakthroughs in plant virus treatment, increasing structural understanding of this crucial molecule. Her award-winning research has the potential to drive scientific innovation in agriculture and human health.
Researchers from Tokyo University of Science discovered that manipulating polyamines enhances the functional profiles of monoclonal antibodies. The study found that controlling polyamine levels increases IgG galactosylation, leading to improved therapeutic efficacy.
Researchers identified RBM5 as a key regulator of HOXA9 expression in leukemia cells, revealing its dual function in DNA and RNA handling. Removing RBM5 from cells significantly reduced HOXA9 mRNA levels, suggesting its potential as a therapeutic target for acute myeloid leukemia treatment.
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Researchers have successfully used mRNA technology to correct a rare genetic disease in mice, demonstrating its potential therapeutic use. The treatment corrected the lethal consequences of the disease and restored glutathione metabolism.
Scientists have created a new generation of prime editors based on the Cas12a protein and circular RNAs, expanding the scope of precision genome editing. The new editors show high editing efficiencies and low off-target effects, paving the way for diverse applications in biological research, disease treatment, and crop breeding.
Mayo Clinic researchers used genetic sequencing to study the measles virus's spread in a human brain. The study found that the virus acquired distinct mutations that drove its spread from the frontal cortex outward. This knowledge may help develop effective antiviral drugs to combat SSPE, a rare and lethal brain disease caused by measles.
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Researchers identified key proteins involved in the process, including mTOR and TSC2, which can be targeted to slow or halt disease progression. The study's findings suggest new therapeutic strategies to save dopaminergic neurons and alleviate motor and cognitive symptoms.
Researchers at TTUHSC are studying a new approach to inhibit STAT3, a protein associated with 70% of human tumors. Disrupting STAT3 synthesis on ribosomes could lead to new cancer treatments.
Researchers at the University of Toronto have discovered a novel ionizable lipid nanoparticle that enables efficient muscle-focused mRNA delivery while minimizing off-target effects. The study demonstrates potent cellular immune responses and potential as a viable candidate for cancer vaccine development.
Researchers have identified a sequence within therapeutic mRNAs that causes unintended immune responses and found a way to correct these errors. By designing 'slip-resistant' mRNAs, the team aims to produce future mRNA vaccines with improved safety and efficacy.
Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting galectin 1 protein using gene therapy can shrink liver cancer in mice, improving the anti-cancer immune response and increasing killer T cells. This approach has potential as a treatment for HCC prevention.
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Researchers found that cancer cells are more vulnerable to radiotherapy when using the less common 'YC' first-base-cytosine site instead of the usual 'YR' adenine or guanine start sites. This discovery enables further understanding of gene regulation in cancers and potential targets for treatment.
Researchers at Goethe University Frankfurt have identified a specific gene locus, MYNRL15, that is critical to the survival and replication of leukemia cells. Inhibiting this gene has been shown to deactivate genes necessary for AML cell survival, offering a new possibility for fighting leukemia.
Researchers at Children's Hospital of Philadelphia found that gene splicing can reduce CD20 protein levels, making immunotherapies ineffective. However, CAR-T cell therapy may still be effective against patients with low CD20 levels, offering a new treatment option for these patients.
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A single dose of zilebesiran safely and effectively lowered systolic blood pressure in people with mild-to-moderate high blood pressure for up to six months. The study found significant reductions in daytime and nighttime systolic blood pressure, as well as low rates of adverse events.
Lepodisiran, a short interfering RNA, targets hepatic synthesis of apolipoprotein(a), reducing serum lipoprotein(a) concentrations. Phase 1 study results support further investigation of lepodisiran's efficacy in treating elevated lipoprotein(a) levels.
A new therapeutic, lepodisiran, has shown promising results in reducing lipoprotein(a) levels to undetectable levels for nearly one year. The study found that the medication is safe and effective in lowering Lp(a) levels by more than 94%.
Researchers at Weill Cornell Medicine have discovered a new mechanism that makes some cancers treatment-resistant, involving the shuttling of messenger RNAs from the nucleus to the cytoplasm. The approach targets this mechanism with a combination of approved chemotherapies, showing promise in treating persistent cases.
A new neighborhood-based care model has been shown to be effective in treating hepatitis C among injection drug users and those experiencing homelessness. The study found that 92% of participants had undetectable levels of the virus after treatment, with 84% achieving sustained virologic response.
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Researchers developed a novel method using extracellular vesicles to deliver mRNA for cancer therapy, overcoming challenges of accurate targeting and production. The approach shows promise for treating hard-to-treat tumor types by reversing immunosuppressive environments and making tumors detectable to the immune system.
Researchers developed a method using microRNA and messenger RNA to identify five common human body fluids at crime scenes. The combined mRNA and miRNA system showed significant advantages over previous methods, providing a scientific reference for RNA-based body fluid identifications.
Scientists designed an mRNA nanovaccine using machine learning to overcome delivery barriers, promoting strong immune responses and activating the STING pathway to kill tumor cells. The therapeutic strategy demonstrated stronger anti-tumor effects in melanoma and colorectal cancer models.
Researchers discovered that targeting TUG1 can control brain tumor growth in mice, suggesting a potential strategy to combat aggressive brain tumors. By inhibiting TUG1, the therapy significantly suppressed tumor growth and improved survival rates when combined with standard treatment.
Researchers at Weill Cornell Medicine have discovered that a tiny chemical modification on mRNAs, known as m6A, is key to the formation of stress granules during cell stress. Longer mRNAs dominate stress granules because they have high levels of m6A, which triggers their sequestration.
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Researchers at Mayo Clinic discovered that senescent macrophages in the lung promote tumor growth by blocking the immune system's response to abnormal cell growth. Eliminating these senescent cells delays tumor formation, suggesting a potential therapeutic target for cancer treatment.