Researchers at UC San Francisco have identified CUL5, a protein that tags tau for elimination, as a key player in preventing the formation of toxic tau protein clumps that can lead to dementia. The study found that neurons with more CUL5 are less vulnerable to Alzheimer's disease.
Researchers at the University of British Columbia have developed a topical CRISPR-based therapy that can correct faulty genes in human skin, potentially treating genetic skin conditions like ARCI and eczema. The treatment, using lipid nanoparticle technology, restores up to 30% of normal skin function.
Researchers at Monash University have developed an AI-powered approach to create highly accurate and specific anti-CRISPR molecules, enabling faster development of gene editing tools for various applications. This breakthrough addresses the inconsistent performance and safety risks associated with CRISPR technology.
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Researchers discovered that CHD1 and MAP3K7 gene deletion improves tumor vulnerability to immunotherapy. This finding suggests new biomarkers for predicting patient response and opening up personalized cancer care. The study sheds light on why some patients are more or less likely to respond to certain types of cancer treatments.
A two-step genome editing method integrates large human genomic fragments into mice, mimicking human regulatory landscapes. This platform enables the creation of physiologically relevant humanized models for therapeutic targets and disease research.
A new diagnostic platform enables rapid and accurate detection of drug-resistant C. auris pathogens using CRISPR technology, allowing for more effective treatment and prevention of hospital outbreaks. The dSHERLOCK test can detect the presence of mutations causing antimicrobial resistance in just 40 minutes.
The CRISPR-Cas3 system has been shown to induce reliable and extensive deletions of the TTR gene in mouse models of ATTR, reducing serum TTR levels by up to 80%. This technology holds promise for treating not onlyATTR but also other incurable inherited diseases.
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Researchers discovered genes that regulate fibroblast growth, which builds the scaffolding between cells. Adjusting these factors reversed age-related changes and improved health outcomes in mice. The study offers new opportunities to understand and reverse aging-related diseases.
Researchers have identified a novel CRISPR mechanism, Cas12a3, that specifically targets transfer ribonucleic acids (tRNA) in bacteria. This discovery provides new insights into the immune response of bacteria and has potential applications for molecular diagnostics and other technologies.
A CRISPR screen identified 331 essential genes for brain cell generation, including PEDS1 linked to a severe developmental disorder. The study provides a new approach to identifying genes involved in neurodevelopmental disorders like autism and offers insights into gene inheritance patterns.
Researchers at Nara Institute of Science and Technology discovered that parasitic plants recognize
Researchers have identified DNA switches that control how brain cells called astrocytes work, which are known to play a role in Alzheimer's disease. The study used CRISPRi technology and single-cell RNA sequencing to test nearly 1000 potential switches, finding that about 150 of them controlled genes implicated in Alzheimer's disease.
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Scientists at Penn Vet have identified two genes, Ctnna1 and Bcl2l13, that suppress metastasis in preclinical models of colorectal cancer. These findings could lead to better treatments and therapies for patients with metastatic disease.
Researchers used CRISPR to increase fungal production efficiency and cut environmental impact by 61% without adding foreign DNA. The genetically tweaked fungus tastes like meat and is easier to digest than its naturally occurring counterpart.
Researchers have shown that disabling the NRF2 gene with CRISPR technology can restore drug sensitivity and slow tumor growth in lung cancer. The approach, which targets a master switch for resistance, has potential across multiple tumor types.
Researchers found that CRISPR-Cas9 gene editing persists longer and produces more predictable results in non-dividing neurons. They also discovered new DNA repair genes that can be used to control gene editing outcomes, which could lead to safer and more effective therapies for genetic diseases.
A recent study published in Nature Communications reveals that the mechanical properties of the developing brain play a significant role in synapse formation and electrical signal emergence. The researchers found that softer regions exhibit higher synapse densities, while stiffer regions show lower densities.
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Researchers at Gladstone Institutes and UCSF have identified the genetic switches that regulate FOXP3 levels in human and mouse cells. In humans, multiple enhancers work together to keep FOXP3 active, while a repressor keeps it off in conventional T cells. This discovery has important implications for developing immune therapies.
Researchers developed a scalable method to produce human kidney organoids, combining them with pig kidneys outside the body for transplantation. The transplanted organs functioned normally and showed no signs of damage or toxicity.
Researchers at the University of Texas at Austin have developed a novel gene-editing method that can correct multiple disease-causing mutations simultaneously. This approach uses bacterial retrons to protect the microbes from viral infection and has shown promising results in correcting scoliosis-causing mutations in zebrafish embryos.
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Researchers developed a new diagnostic test, CAARRD, to detect ribonucleic acid (RNA) at room temperature, increasing sensitivity and reducing complexity. The test uses special 'anti-tag' CRISPR sequences to block the activity of the Cas13a enzyme, allowing for faster and more affordable detection of viral RNAs such as HIV.
Researchers at CNIO have created a 'human repairome', a catalogue of 20,000 DNA 'scars' that reveal how genes affect DNA repair. This information can help determine the best treatment for each cancer type and overcome resistance to therapy.
The CityUHK team is developing two core therapeutic medicines using state-of-the-art DNA surgery technology to treat liver and cardiovascular genetic diseases. Their approach offers a durable and long-lasting solution, eliminating the need for repeated medications.
Researchers discovered that the functional splitting of transposon-derived RNAs drove the emergence of Type V CRISPR-Cas immunity. This innovation enabled the development of compact nucleases with flexible guide RNAs, offering design principles to create smaller and more versatile CRISPR tools.
Researchers identified genetic modifications that can improve the efficacy of chimeric antigen receptor (CAR)-T cell treatment for multiple myeloma and other cancers. The study used CRISPR screening to pinpoint genes that influenced T cell function and survival in culture and in a preclinical model of multiple myeloma.
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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 UC San Diego have created a new genetic editing approach that uses small nuclear RNA base editing, which can modify the genetic code with greater precision and safety than CRISPR. This method has the potential to treat various diseases, including neurodegenerative, cardiovascular, and immune disorders.
Scientists at UCSF successfully used CRISPRa to increase SCN2A levels in mice with the genetic disorder, resulting in reduced seizures and improved brain function. The therapy offers hope for treating neurodevelopmental issues related to SCN2A haploinsufficiency.
Researchers at MIT have developed a new approach to gene editing that reduces errors by up to 90%, making it a safer alternative for treating genetic diseases. The technique uses modified versions of the Cas9 enzyme to target specific DNA sequences, reducing off-target effects and increasing precision.
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Researchers at Stanford Medicine have created an AI-powered CRISPR tool called CRISPR-GPT, which helps scientists design and analyze gene-editing experiments. The technology aims to produce lifesaving drugs faster and expand access to gene editing for a wider range of scientists, including those with limited experience.
Researchers from Mass General Brigham developed a bespoke CRISPR-Cas9 gene-editing enzyme to correct the genetic error causing multisystemic smooth muscle dysfunction syndrome, a rare condition associated with stroke and death in childhood. The therapy extended survival four-fold in mouse models of MSMDS.
Researchers at Northwestern University have developed a new CRISPR delivery system that triples efficiency using DNA-wrapped nanoparticles, improving safety and effectiveness. The new system, called LNP-SNAs, targets specific cells and tissues, reducing toxicity and boosting gene-editing efficiency by threefold.
A key contributor is the lab of Prof. Ayal Hendel at Bar-Ilan University's Goodman Faculty of Life Sciences, focusing on evaluating CRISPR-based gene editing precision and safety. The project aims to decentralize CAR-T cell therapy production, making it available to more patients by reducing costs and increasing accessibility.
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Researchers have developed a new epigenetic editing method using CRISPR technology, which can switch genes back on by removing methyl groups attached to silenced or suppressed genes. This approach shows promise for treating people with Sickle Cell-related diseases, reducing the risk of unwanted changes and potential health problems.
Researchers at UC Berkeley developed a method to understand the isotope signatures of methane from different environments using CRISPR. This study can change how scientists calculate the contributions of various environmental sources to Earth's total methane budget.
A research team developed a new method to precisely edit DNA by combining genetic engineering with artificial intelligence. The technique enables accurate modeling of human diseases and lays the groundwork for next-generation gene therapies.
Researchers at the Salk Institute have identified dozens of microproteins that play a crucial role in regulating fat cell proliferation and lipid accumulation. This breakthrough discovery offers new potential drug targets for treating obesity and metabolic disorders, building on recent advances in CRISPR gene editing technologies.
A team of scientists proposes an integrated framework combining biotechnology and AI to revolutionize crop breeding, exploring multi-omics, genome editing, and high-throughput phenotyping. The authors present a forward-looking framework for AI-assisted crop germplasm design, offering a roadmap for sustainable agriculture.
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Researchers developed a CRISPR-based gene-editing system that changes a single molecule within mosquitoes, halting malaria-parasite transmission. The new system is designed to genetically spread the malaria resistance trait until entire populations of the insects no longer transfer the disease-causing parasites.
A team of scientists proposes using gene editing to restore lost genetic diversity in endangered species, enabling them to adapt to future environmental changes. The approach could complement traditional conservation methods and attract new investors and expertise.
Researchers successfully modulate the activity of key immune system genes using CRISPR-Cas9 technology, revealing a new approach to treating inflammatory diseases. The study demonstrates precise control over gene expression and its impact on tumour growth and inflammation.
Scientists have identified a set of genes in zebrafish that reactivate after damage to the heart and patch it up like new. The researchers hope to use CRISPR tools to reactivate similar genes in humans and jump-start repair of the heart and other tissues after injury.
The Center will develop personalized CRISPR on-demand treatments for children with severe inborn errors of immunity and metabolic disease. The initiative aims to make CRISPR cures more affordable and accessible, building on recent clinical success in treating ultra-rare genetic diseases.
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Researchers developed Variant-aware Cas-OFFinder, a web-based tool that improves CRISPR accuracy by identifying off-target effects across genetic variations. The tool offers a significant step forward in personalized genome editing by incorporating genetic diversity directly into off-target predictions.
Researchers have successfully edited harmful mitochondrial DNA mutations in liver and skin cells using a genetic tool called a base editor. The study, published in PLOS Biology, offers promising results for treating mitochondrial diseases and aging-related conditions.
Researchers developed a new laboratory method that helps diagnose patients with suspected immune system disorders like activated-PI3Kδ syndrome (APDS). The method identified dozens of additional genetic variations associated with the condition, enabling rapid diagnosis and treatment. This breakthrough may also help identify more patien...
Researchers have found a promising new method for gene therapy by bringing dormant genes closer to enhancer switches on the DNA. This 'delete-to-recruit' strategy has potential for treating genetic diseases such as sickle cell disease and beta-thalassemia, offering an alternative to expensive current treatments.
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Researchers at KAIST have developed a groundbreaking technology capable of selectively acetylating specific RNA molecules within the human body using the CRISPR-Cas13 system. This breakthrough enables precise, programmable control of RNA function and is expected to open new avenues in RNA-based therapeutic development.
Researchers leveraged CRISPR/CasRx to silence Ctnnb1 and Smo, two genes involved in osteoarthritis development. Simultaneous knockdown of both genes effectively mitigated joint structure degeneration, highlighting the potential of RNA-based gene therapies for OA management.
Researchers discovered that a ligase called Lig3 inhibits base editing, while the mismatch repair pathway helps cytosine base editing. The study sheds light on the complex mechanisms behind base editing and its potential applications in treating genetic diseases.
Researchers at Stanford University have developed a new CRISPR technology called CRISPR-TO that can transport RNA molecules to specific locations within neurons, enabling repair and regeneration. The technology has shown promising results in increasing neurite growth by up to 50% in mouse brain neurons.
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Researchers at MSK uncovered a key signaling molecule involved in the body's immune response against leptomeningeal metastasis. A new grading system to assess thrombocytopenia risk after CAR T cell therapy was also developed. Additionally, a statistical method called UnitedMet estimates metabolic characteristics from challenging clinic...
Scientists at Rockefeller University have identified a novel CARF effector called Cat1 that prevents viral replication by depleting NAD+ metabolites. The discovery sheds new light on the complex molecular mechanisms behind CRISPR-Cas9 defense systems.
A new generative AI technique allows for the design of RNA molecules with improved functions, opening up potential for novel therapeutics and diagnostics. The SANDSTORM and GARDN systems enable the prediction and generation of RNA sequences tailored for specific tasks in cells or diagnostic assays.
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Researchers at Karolinska Institutet have developed a technique to deliver gene editors and protein therapeutics to cells using engineered extracellular vesicles. The method shows promising results in animal studies, highlighting the potential for treating genetic diseases and neurological disorders.
Mass General Brigham researchers developed a machine learning algorithm, PAMmla, to predict properties of genome editing enzymes. The approach helps reduce off-target effects and improves editing safety and efficiency, enabling customized enzymes for new therapeutic targets.
Tulane University scientists developed a handheld device to deliver rapid and accurate tuberculosis diagnoses in under an hour. The device, called the lab-in-tube assay (LIT), can detect Mycobacterium tuberculosis DNA in saliva, blood, and sputum samples, offering a cost-effective tool for improving TB diagnoses in resource-limited areas.
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Researchers at KAIST discovered that DDX54 is the master regulator hindering immunotherapy's effectiveness in lung cancer. Supressing DDX54 enhances immune cell infiltration into tumors and improves immunotherapy efficacy.
Researchers have discovered a protective cloaking mechanism in jumbo phages that shield their genetic material from the host's immune system. This innovation could lead to new therapies for antibiotic-resistant infections.
Researchers developed a CRISPR-based diagnostic test that rapidly detects low levels of pathogen genetic material in blood without nucleic acid amplification. The test demonstrated unprecedented sensitivity and could be used to develop highly sensitive CRISPR-based diagnostic tests for detecting pathogens in minutes.