Scientists develop modified CRISPR-Cas9 technique that alters gene activity without cutting DNA, reversing diseases in mice models. The technique uses adeno-associated viruses to introduce genetic manipulation machinery to cells, promoting expression of target genes without introducing mutations.
Researchers created an animal model that closely replicates the human form of Alzheimer's disease, including pathological tau protein and amyloid plaques. This breakthrough allows for testing of new therapies targeting both pathologies.
Researchers at Karolinska Institutet discovered that Alagille Syndrome is caused by malformations of the bile ducts, leading to serious liver and heart problems. The study provides new insights into the disease and opens up possibilities for targeted therapies.
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A single infusion of wildtype hematopoietic stem and progenitor cells into a mouse model of Friedreich's ataxia restored normal cellular functions, halted cellular damage, and improved mitochondrial function. This breakthrough suggests a potential therapeutic approach for the currently incurable disease.
Researchers used gene therapy to stop the immune response that causes multiple sclerosis in mouse models, producing near-complete remission. The treatment combined a brain-protein gene with an existing medication, showing significant potential for treating multiple sclerosis and other autoimmune disorders.
Researchers from Instituto de Medicina Molecular created a chimera virus that can test molecules to treat cancers caused by human herpes virus infection in mice models of disease. This finding preserves the functionality of LANA, a protein vital for Kaposi virus maintenance, allowing new cancer treatments to be developed.
Scientists identified a compound, FR, that provides long-lasting airway relaxation and prevents hyperreactivity in mouse models of asthma. The locally administered compound also blocks aspects of airway remodeling without causing cardiovascular side effects.
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Researchers at Vanderbilt University Medical Center have developed a small molecule compound that works like the dimmer switch in an electrical circuit, relieving symptoms of Rett syndrome in mice. The study provides further evidence that a drug may be possible to treat this rare neurodevelopmental disorder in females.
The Jackson Laboratory will investigate a new mouse model for amyotrophic lateral sclerosis (ALS) with a $3.2 million federal research grant. The study aims to link genetic mutations in the mouse models to human ALS or other neuromuscular diseases.
A new study suggests that the small molecule LM22A-4 can improve spatial memory and motor skill defects in Rett syndrome mice by enhancing synaptic plasticity in the hippocampus. The treatment also shows promise for improving breathing problems associated with the disease.
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Researchers found that increasing serotonergic activity in a mouse model of autism improved social behavior and reduced abnormal brain activity. The study suggests that serotonin may be potentially therapeutic for discrete ASD symptoms.
A new mouse model has been created to investigate kidney cancer, allowing researchers to develop better treatments. The model reveals that gene mutations in the primary cilium contribute to renal cell carcinoma's progression.
Researchers have created a promising mouse model for the devastating genetic disorder NGLY1 deficiency. The double-deletion mice survive and exhibit symptoms analogous to humans with the condition, making them useful for testing potential therapies.
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Researchers at JAX will study mouse models of inherited RPE-driven disease to identify potential molecular pathways for druggable targets. Their goal is to prevent, delay onset or decrease the severity of age-related macular degeneration and other heritable retinal diseases.
Scientists have developed a new mouse model with a healthy immune system to study the Zika virus. The model allows researchers to investigate the immune response to Zika, which could lead to advances in vaccine development and treatment strategies.
Researchers propose a new therapy for Gaucher disease by blocking the molecule C5aR1, which drives inflammation and organ damage. The treatment may offer fewer risks and lower costs than current therapies.
A team of researchers has discovered a novel approach to treating juvenile Batten disease by activating a protein called TFEB, which stimulates the cell to produce more lysosomes and degrade cellular waste. This breakthrough may lead to improved neurological symptoms in patients with the condition.
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Research from the University of British Columbia found that a vitamin A deficiency in the womb or early infancy can increase production of amyloid beta and lead to cognitive impairments. Providing supplements after birth may help slow the degenerative brain disease.
Researchers developed a new mouse model that faithfully reproduces the pathologies of COPD and CF, revealing two key pathways: oxidative stress and protease-antiprotease imbalance. The model shows promise for developing new medication therapies.
New research identifies physiological changes in the body that could explain why older mothers are more likely to experience complicated births. The study found that maternal age influences the structure of the uterus, leading to impaired muscle contraction properties, reduced sensitivity to oxytocin, and altered hormonal signals.
A new mouse model has revealed the role of CCN6 protein in developing metaplastic breast cancer, a rare and aggressive subtype of triple-negative breast cancer. The study identified potential genes to target with therapeutics, offering hope for better treatment options for patients.
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Researchers developed a mouse model to assess early tissue responses to biomaterials, including bioactive glass. The model's feasibility and reliability have been demonstrated using various biomaterials, enabling the design of novel biomaterials for regenerative medicine.
Researchers have discovered a potential treatment for Prader-Willi syndrome (PWS), a rare genetic disorder affecting children, by activating silenced genes. The NIH-funded study found that two drugs, UNC0638 and UNC0642, improved survival and growth outcomes in mice with PWS.
Scientists found that female mice are more susceptible to vaginal Zika virus infection during a specific stage of their reproductive cycle. The study suggests that sex hormones play a role in allowing the virus to establish itself in the reproductive tract and spread beyond it.
Researchers confirm live Zika virus infects reproductive tract, replicates and causes disease in mouse models. Hormonal injection timing affects vulnerability to infection, with diestrus-infected mice showing no signs of disease despite viral persistence.
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Researchers at the Forsyth Institute have defined the immune-regulatory mechanisms of Sjögren's syndrome, revealing how PD-L1 and PD-1 proteins interact to suppress protective immunity. The study found that inhibiting this pathway accelerates autoimmune responses and disease development.
A team of scientists has developed a mouse model that closely mimics fetal brain abnormalities caused by the Zika virus, revealing abnormal blood vessel formation and a leaky blood-brain barrier. This finding highlights the need to understand all the effects of Zika infection if successful therapies are to be developed.
Researchers have developed a new mouse model that can be used to study the Zika virus and its effects on the body. The model, which employs mice with functioning immune systems, has been shown to develop symptoms of neurological disease after infection, providing valuable insights into potential treatments.
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Researchers have developed two new mouse models of amyotrophic lateral sclerosis (ALS) that exhibit protein clumping and display clinical features seen in patients. The models may help scientists better understand the disease and develop new treatments.
A pediatric otolaryngologist is developing a mouse model to identify the most effective treatment for respiratory papillomatosis, a rare condition that can cause chronic hoarseness and breathing problems in children. The goal is to provide personalized medicine and reduce the need for frequent surgeries.
Researchers from Ben-Gurion University of the Negev discovered a novel molecular mechanism that could lead to new therapies for ALS. They found that endogenous multifunctional protein macrophage migration inhibitory factor (MIF) acts as a chaperone for misfolded SOD1 proteins, which accumulate and cause cell death in ALS patients.
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Researchers from University of Eastern Finland discovered that retinal changes can be detected earlier than brain changes in CNS diseases. Functional abnormalities were found in three genetically engineered mouse models of human CNS diseases, suggesting eye examinations could be used as a noninvasive screening tool.
The grant will accelerate the creation of high-priority mouse models for Charcot-Marie-Tooth disease and other peripheral neuropathies. Researchers aim to investigate disease mechanisms and develop treatments, which currently have no cures or effective treatments.
Researchers developed a mouse model that mimics human Alzheimer's disease, showing that beta-amyloid accumulation is insufficient to trigger tau clumping alone. The study suggests combination therapy targeting both processes may be effective in preventing the disease.
A study published in Cell Reports has found that inhibiting the enzyme cdk4 can prevent and reverse the initial stage of Non Alcoholic Fatty Liver Disease (NAFLD). Researchers at Cincinnati Children's Hospital Medical Center used two FDA-approved drugs to inhibit cdk4, significantly reducing hepatic steatosis in mouse models.
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Researchers at UC Santa Barbara have discovered a possible first therapy for Mucolipidosis IV by extending their findings from fruit flies to a mouse model. Bone marrow transplantation significantly delayed the onset of motor deficits in MLIV mice, preventing the amplification process that causes neurodegeneration and blindness.
Research from Baylor College of Medicine found that Notch activation promotes metastasis in prostate cancer by upregulating FoxC2, a molecule important for metastatic potential. The study used a mouse model with prostate-specific loss-of-function Pten to demonstrate the role of Notch in prostate cancer progression.
Scientists at Hong Kong University of Science and Technology discover that interleukin-33 (IL-33) rescues contextual memory deficits and reduces beta-amyloid peptide deposition in AD mouse models, suggesting a new therapeutic intervention.
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Researchers have discovered that caspase 12 does not act as a dominant-negative regulator of caspase-1 activation and inflammasomes. This finding challenges a stubborn dogma in the field and opens up new avenues for studying caspase 12's role in physiological processes.
Researchers found that endocannabinoids quiet neurons in the orbitofrontal cortex, leading to an over-reliance on habit. The study suggests a new therapeutic target for OCD and addictions: treating the brain's endocannabinoid system to restore goal-directed action.
Researchers have established mouse models of Zika virus transmission from a pregnant mouse to her fetus, demonstrating viral invasion and damage to the placenta. The studies reveal that Zika virus can cause congenital problems, including fetal death, by breaching the placental barrier.
A study published in PLOS Neglected Tropical Diseases describes a suitable small animal model for testing ZIKV interventions. The A129 mouse model accumulates virus in the brain and other tissues, exhibiting symptoms similar to those in humans.
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Researchers created a mouse model that closely replicates the symptoms and gene expression patterns of ALS and frontotemporal dementia. The model allows scientists to understand how the C9orf72 gene mutation causes paralysis and dementia, and potentially develop treatments.
A new mouse model developed by researchers at the University of Wisconsin-Madison School of Veterinary Medicine enables the study of Zika virus infection and its effects on the brain. The model, which lacks key immune system components, allows for the testing of vaccines and antivirals against the virus.
Scientists at the University of Wisconsin-Madison have created a mouse model to study Zika virus, allowing researchers to test vaccines and antivirals while understanding the virus's effects on human brains. The model, lacking key immune system defenses, shows the virus causes severe pathology in brain tissue.
Researchers have established a mouse model that mimics aspects of Zika virus infection in humans, allowing for the testing of vaccines and therapeutics. The model shows high levels of the virus in the brain, spinal cord, and testes of male mice, supporting clinical data on sexual transmission.
Human placental trophoblasts resistant to Zika virus proliferation and release an antiviral molecule called type III interferon to stop viral replication. Mouse models show high virus levels in brain, spinal cord, and testes after inoculation through skin.
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Researchers at UTMB have developed a new mouse model for Zika infection, removing a bottleneck in preclinical testing. The model is available for immediate screening of antivirals and vaccine candidates.
Researchers used a mouse model to explore the causes of preeclampsia and found that Cox2 inhibition can improve symptoms by treating embryos before implantation. This approach has potential for preventing preeclampsia-related complications.
Researchers discovered that changes in gut bacteria are strongly associated with PCOS-related obesity and signs of diabetes. Modifying the gut microbiome may be a potential treatment option for women with PCOS.
High-arched palate is under-researched, but researchers developed a reliable technique using a mouse model of Treacher Collins syndrome to study its genetic aspects. The study found that TCS mice exhibited high-arched palates and provided significant criteria for defining the condition.
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Researchers at Oregon State University have developed a therapy that halts the progression of ALS in mice for nearly two years, allowing them to approach their normal lifespan. The treatment uses copper-ATSM, which delivers copper specifically to cells with damaged mitochondria and has low toxicity.
Researchers discovered a distinctive odor signature in mouse models of Alzheimer's disease that appears before significant brain pathology development, suggesting a non-invasive tool for early diagnosis. The odor profile may be related to the presence of an underlying gene rather than actual brain changes.
Researchers at the University of Florida have discovered a protein that stimulates fat metabolism when stressed, leading to weight gain. The study found betatrophin, a previously touted diabetes treatment, has a new role in regulating body fat.
Researchers used genetically-modified mice to study hereditary deafness caused by mitochondrial dysfunction. Reducing enzyme activity in mice showed promise for preventing or delaying deafness, according to new research.
Researchers have developed a new mouse model for spinal muscular atrophy that responds to therapy, allowing for post-symptomatic treatment and potentially improving outcomes for patients. The treatment, an antisense oligonucleotide, restores motor unit function in the muscles even after symptoms have begun.
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Researchers develop novel mouse model for Leber hereditary optic neuropathy and demonstrate successful gene therapy in improving visual function. The approach could aid therapy development for various mitochondrial diseases, with a clinical trial currently underway.
Research identifies a key trouble spot in the brain that contributes to intellectual disability in Down syndrome, shedding light on disrupted brain networks. The study suggests therapies targeting these networks may be beneficial for future treatments.
Researchers develop new approach to treating Rett syndrome by extending lifespan and improving behavioral symptoms in mouse models. The treatment targets PTP1B, an enzyme with abnormal levels in the disorder, restoring BDNF signaling and promoting neural growth.
Researchers at Case Western Reserve University used stereomicroscopy to create detailed 3D images of mouse models of IBD, revealing distinct patterns related to health and disease. The findings suggest two mouse models most closely resembling human forms of the illness.
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