Researchers at UMass Chan Medical School and the Marine Biological Laboratory identified four core clock genes controlling circadian rhythms in a crustacean, which also regulate circatidal behavior. The genetic system is highly malleable, allowing the tiny shrimp to maintain both a 24-hour and a 12.4-hour clock.
Researchers have developed an mRNA immunotherapy that eliminates pancreatic tumors in mice, achieving complete tumor responses and long-term disease-free periods. The treatment uses a cocktail of immune cytokine and tumor-associated antigen mRNAs, showing promise as a potential transformative treatment for pancreatic cancer.
A new study has discovered a molecular navigation system that precisely directs neutrophils to sites of infection, allowing them to fight off disease-causing microbes without damaging healthy tissue. This finding represents a promising therapeutic strategy for treating chronic inflammatory diseases.
Researchers at UMass Chan Medical School have developed a microRNA-based gene therapy that suppresses mutant SOD1 production, delaying disease onset by 60 days and extending lifespan by 100 days in mice models of ALS. The therapy, delivered via adeno-associated virus (AAV) vector, preserves motor neurons and maintains neuromuscular con...
Philip Tai, PhD, received a $1.6 million grant to investigate AAV vector mechanisms using high-resolution DNA sequencing technology. His findings could lead to new vector designs that improve gene therapy treatments' safety. The goal is to remove mutations that cause cancer-causing integration into host cells.
The UMass Center for Clinical and Translational Science has received a seven-year, $50 million grant from the NIH. The funding supports collaboration among researchers at multiple UMass campuses and clinical partners.
Researchers have developed a new gene editing technology called 'prime assembly' that allows efficient insertion of large DNA segments into the human genome. This innovation enables treatment of genetic diseases by replacing entire genes, promising substantial progress in treating conditions with multiple mutations.
Toloo Taghian, a UMass Chan Medical School assistant professor, has received a five-year, $3.2 million grant to develop a gene therapy for UBA5 disorder, a rare genetic disease that affects protein balance and leads to neurological impacts. The goal is to monitor disease progression and determine the efficacy of future clinical trials.
Dr. Marcus Ruscetti receives the prize to study senescence and its impact on immune responses in cancer metastasis. The funding will help him develop novel treatments targeting senescent cells.
A Phase I clinical trial of a human monoclonal antibody for Lyme disease demonstrates its safety, tolerability, and pharmacokinetics. The treatment, TNX-4800, provides lasting serum concentrations and has the potential to offer passive immunity against the disease.
Research reveals inflammation in the GI tract changes how nerves are arranged, affecting intestinal muscle contractions. A protective stress response pathway helps neurons survive, preserving their structure and potentially offering a way to curtail persistent symptoms associated with IBD.
Researchers have created a comprehensive map of the DNA sequences that control gene expression in human cells, identifying 2.37 million potential regulatory elements. This registry reveals previously unrecognized classes of elements and illuminates how noncoding genetic variation contributes to cell type-specific traits.
The study created a critical framework for understanding the architecture of the genome and its association with gene function in cells. The 4DN Consortium integrated data from over a dozen techniques to compile an extensive catalogue of looping interactions between genes and regulatory elements.
A Phase I/II clinical trial found that gene therapy reduced seizures, improved oral feeding, and increased production of the HexA enzyme. Participants experienced fewer and more controllable seizures, and some remained on full oral feeds for up to 27 months.
Researchers have identified a key protein associated with Meckel-Gruber syndrome that is cut in half to perform two separate functions, both fundamental to healthy cilia development. The findings shed new light on how cilia formation and cell signaling are finetuned by external forces.
Researchers have identified a specific group of neurons in the brain responsible for suppressing binge drinking behavior. The discovery may lead to new therapeutic targets for treating alcohol dependency and related health challenges.
Researchers have discovered a new mechanism of how anticancer drugs attack and destroy BRCA mutant cancer cells, including drug-resistant breast cancer cells. The study found that small DNA nicks can expand into large single-stranded DNA gaps, leading to cell death.
A gene therapy treatment has demonstrated significant promise in treating maple syrup urine disease (MSUD), a rare genetic disorder. The therapy prevented death and normalized growth in animal models, including a calf with the condition, and is being explored for potential use in humans.
Researchers from Walhout lab published two papers in Nature describing a significant advance in understanding the complex functions of the metabolic network. The studies reveal how cells adjust their metabolism in response to changes in nutrient intake and identify key 'core' metabolic functions that are compensated for or repressed.
Researchers discovered immune system dysfunction as a potential biomarker for long COVID and myalgic encephalomyelitis/chronic fatigue syndrome. A novel treatment approach was also introduced, showing promise in reducing symptoms. The study suggests that targeting CD8 T-cell dysfunction may help alleviate symptoms of these conditions.
Researchers established a molecular link between specific B12-producing gut bacteria and acetylcholine production in C. elegans, a neurotransmitter important to memory and cognitive function. A diet rich in these bacteria reduced seizure-like behavior in mutant worms by restoring excitatory/inhibitory balance.
A novel antisense therapy has restored fragile X protein production in human cell samples, revealing aberrant alternative splicing of messenger RNA as a key factor in fragile X syndrome. This finding offers real hope for developing new treatments and improving the lives of individuals affected by the condition.
Researchers at UMass Chan Medical School identified the Bmal1 gene as a crucial regulator of circatidal behavior in P. hawaiensis, establishing a molecular link between circadian and circatidal clocks. The study provides new insights into the genetics underlying circatidal rhythms.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
A team of researchers developed a model system to study individual differences in metabolism using C. elegans worms. They identified a novel metabolic condition linked to variation in the hphd-1 gene, which has implications for personalized medicine and tailoring dietary advice and disease treatment to an individual's genome sequence.
Researchers discovered that an inorganic polyphosphate released by nerve cells contributes to the death of motor neurons in people with ALS and frontotemporal dementia. The study found that lowering levels of this toxin may be an innovative therapeutic strategy for diverse types of ALS/FTD.
A UMass Chan clinical trial demonstrates the safety and efficacy of an antisense oligonucleotide in suppressing mutant C9ORF72, a common cause of familial ALS. The treatment led to reduced levels of neurotoxins and stable or improved ALS functional scores.
A randomized clinical trial of 125 young adults with concussions found that restricting screen time for 48 hours significantly reduced symptom duration. The study, published in JAMA Pediatrics, suggests that limiting screen time may be a useful strategy to accelerate concussion recovery.
A UMass Medical School study finds that low levels of PAPPA, commonly screened for chromosomal disorders, are associated with adipose tissue remodeling and gestational diabetes. The protein is linked to insulin-like growth factor-1 (IGF-1), which promotes healthy adipose tissue development during pregnancy.
A new $2 million grant from NCI SeroNet will enhance representation of Black and Hispanic people in SARS-CoV-2 immunology research through narrative storytelling. Researchers aim to overcome barriers to participation by connecting with community members personally and motivating them to volunteer.
Researchers at MassBiologics discovered a cross-reactive human monoclonal antibody to SARS-CoV-2 spike proteins, blocking ACE2 receptor binding and potentially preventing or limiting COVID-19 disease. The antibodies, in the sIgA isotype, exhibited higher binding affinity and neutralization activity than usual circulating IgG antibodies.
Researchers identified nearly 1 million potential functional genomic elements in the human genome, which control gene expression and promote health or disease. The UMMS team's registry of these elements can be used to study links between regulatory switches and genetic diseases.
Researchers at UMass Chan Medical School have developed a gene therapy approach that protects synaptic connections in the brain from damage in multiple sclerosis models. The study suggests that therapies targeting specific circuits of the brain could be effective in preserving vision and preventing neurodegenerative symptoms.
Researchers at UMass Medical School and the University of Queensland identified a novel genetic defense system in koalas that controls retrovirus production through germline cells. This innate genome immune response recognizes viral RNAs via anti-sense piRNAs, suppressing assembly of viral particles.
Research from the Alkema lab reveals that the 'fight-or-flight' response triggers tyramine release, which stimulates insulin pathways to satisfy energy demands but down-regulates cellular protection against environmental stress. This dynamic regulation negatively impacts health and aging in nematode worms.
A new approach to gene editing has been developed by scientists at UMass Medical School, allowing for the correction of microduplications associated with 143 different diseases. The strategy uses CRISPR/Cas9 and harnesses the homology-directed repair pathway to remove duplicated sequences and restore functional genes.
Caterina Strambio De Castillia has been named a CZI Imaging Scientist and received over $1 million in grant funding to develop standardized tools for microscopic imaging. The grant aims to facilitate sharing of reproducible datasets, analytical procedures, and analysis results among biomedical researchers.
Researchers at UMass Medical School have developed a technology that allows mammals to see infrared light using nanoantennae injections. The temporary vision enhancement enables animals to perceive near-infrared patterns and shapes without interfering with their visible light ability.
Researchers at UMass Medical School developed an siRNA therapy that reduces circulating sFLT1 levels and normalizes blood pressure and proteinuria in pregnant mice. The therapy shows promise as a potential strategy for treating preeclampsia in humans, but further studies are needed to optimize safety and efficacy.
A new study by UMass Medical School researchers successfully delivers RNAi-based gene therapy to silence SOD1 protein mutations linked to ALS without adverse effects. The therapy, delivered via a viral vector, achieved silencing of over 90% in some motor neurons, suggesting a safe and potentially one-time treatment for humans.
Researchers discovered that cannabis's endocannabinoids help regulate gut inflammation by turning off an aggressive immune response. This mechanism provides a new drug target for treating patients with inflammatory bowel diseases.
Studies by UMass Medical School Professor Oliver J. Rando and colleagues found that small RNAs in sperm play a crucial role in normal pre-implantation development, with subtle differences between sperm from the epididymis impacting offspring health.
Researchers identify KIF5A as a new gene associated with ALS, implicating the role of cytoskeletal defects in axon communication. The discovery suggests the cytoskeleton as a potential target for new drug development and may lead to improved treatments for familial and sporadic ALS.
A study by UMMS researchers has identified a protein called GDF6 as a primary role in metastatic melanoma, found to be expressed in 80% of human melanomas and correlated with increased melanoma growth and spread. The findings offer new therapeutic potential for treatment-resistant skin cancer.
Researchers have made a breakthrough in treating alpha-1 antitrypsin deficiency by using a new gene editing technique. The approach combines RNA interference and gene augmentation to repopulate diseased livers with healthy cells, preventing damage and promoting regeneration.
A new study from UMass Medical School found that obese patients who underwent knee or hip replacement surgery reported similar pain relief and improved function as normal-weight patients. The study suggests that obesity is not a barrier to joint replacement surgery, which can provide long-term relief from joint pain.
A team led by Gang Han has designed a human protein-based, tumor-targeting Magnetic Resonance Imaging (MRI) contrast that can be easily cleared by the body. This discovery holds promise for clinical applications, including enhanced MRI contrast for early-stage tumor detection.
A UMass Medical School study found that the bacteria in the gut microbiome can affect how sensitive worms are to chemotherapy drugs. The researchers used Caenorhabditis elegans, a roundworm, and fed them different types of bacteria to see which ones made them more or less sensitive to the drug floxuridine, used to treat colorectal cancer.
A new study led by Jennifer Tjia found that training nursing home staff to recognize unmet needs of cognitively impaired residents significantly reduced antipsychotic medication use. The OASIS program shifted care planning from focusing on limitations to meeting resident needs, resulting in a 7% drop in antipsychotic prescriptions.
Researchers at UMass Medical School and the University of Toronto identify naturally-occurring proteins that inhibit Cas9 enzyme, providing greater control over genome edits. This discovery bolsters promise for therapeutic applications and better control over editing.