The Cancer Dependency Map now includes nearly 150 3D cancer models, expanding its resource to include organoids and spheroids across 10 cancer types. These next-generation models reveal new genetic dependencies and disease-driving mechanisms, particularly in brain and gastrointestinal cancers.
A phase 1 clinical trial has started enrolling patients with symptoms of prion disease, evaluating the safety and tolerability of a small interfering RNA targeting the prion protein. The trial aims to slow disease progression and is supported by NeuroNEXT, a program of the National Institute of Neurological Disorders and Stroke.
The Cancer Dependency Map Consortium is launching Phase 3 to expand its research beyond cancer vulnerabilities to investigate resistance and surface targets. The consortium aims to develop novel oncology targets and biomarkers for the next generation of cancer therapies.
A study in mice reveals that chronic intestinal inflammation can lead to epigenetic 'memories' that promote colon cancer. Researchers found that even seemingly healed gut tissues retain molecular scars from earlier inflammation, making it easier for cancer to take hold.
The Stanley Family Foundation has renewed its $1 billion commitment to psychiatric research at Broad Institute, transforming the field of psychiatric genetics and schizophrenia/bipolar disorder research. Human genetics data from the Stanley Center has provided unprecedented clues to the brain biology underlying these conditions.
Researchers found that low-oxygen environments can protect the brain and restore movement in mice with Parkinson's-like disease by reducing excess oxygen molecules. The study suggests that hypoxia could be a new approach to treating Parkinson's, potentially targeting cellular dysfunction rather than protein clumps.
Researchers used prime editing to correct five different AHC-causing genetic mutations in mice, resulting in far fewer and less severe symptoms. The treatment successfully repaired up to 90% of treated cells, demonstrating its potential for treating people with this rare neurological disorder.
Researchers at the Broad Institute developed a gene editing approach that interrupts and stabilizes trinucleotide repeat expansions, which cause Huntington's disease and Friedreich's ataxia. The method, using base editing, prevents the repeats from growing in length, halting or slowing down disease progression.
Four researchers — Carl June, Bruce Levine, Isabelle Rivière, and Michel Sadelain — are awarded the Merkin Prize for developing CAR T-cell therapy, a groundbreaking form of personalized cancer immunotherapy. The technology has led to durable remissions in tens of thousands of patients with previously incurable blood cancers.
Researchers at Broad Institute of MIT and Harvard have identified four coordinated gene expression programs in immune cells from glioma tumors that can lead to immunotherapy resistance. The study found two programs that could be targeted to improve patient response to immunotherapies, including one that may reduce the effectiveness of ...
A new study reveals that the inherited genetic mutation in Huntington’s disease doesn't harm cells immediately, but slowly morphs into a highly toxic form that kills the cell. The findings suggest potential ways to delay or even prevent the disease by stopping or slowing CAG-repeat expansion in the HTT gene.
Researchers developed a gene-editing treatment that reduces prion protein levels in the brain by up to 60% and extends mouse lifespan by about 50%. The base-editing approach could lead to a one-time treatment for prion disease patients.
A comprehensive map of gene expression in the mouse intestine reveals unique regions tightly regulated by the immune system, including a region controlled by immune signals. The findings suggest resilience and adaptability of the intestine to perturbations.
Researchers have identified changes in immune and stromal cells that underlie myocarditis, a rare but deadly complication of cancer immunotherapy. The study suggests that targeted treatments might be able to address myocarditis while allowing patients to continue receiving life-saving anti-tumor immunotherapy.
Researchers have identified 18 beneficial bacterial strains that suppress the growth of harmful bacteria and alleviate inflammation in mouse intestines. The strains compete with harmful bacteria for nutrients, promoting a healthier intestinal microbiome and potentially treating antibiotic-resistant gut infections.
Researchers have discovered that oleic acid, a naturally occurring oil in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV. Oleic acid inhibits growth of detrimental bacteria and promotes healthier species associated with a stable microbiome.
Researchers at the Broad Institute of MIT and Harvard developed a machine-learning approach to design better AAVs for gene therapy. The tool helps engineer capsids with multiple desirable traits, such as targeting specific organs or working in multiple species. About 90% of predicted capsids successfully delivered cargo to human liver ...
Researchers at Broad Institute of MIT and Harvard have developed a gene-editing approach that efficiently corrects the most common mutation causing cystic fibrosis, found in 85% of patients. The new method precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to Trikafta.
A new CRISPR-based paper strip test could allow more patients to get the right treatment for the flu, researchers say. The test distinguishes between different influenza types and can be reprogrammed to recognize other viruses.
New research reveals that CRISPR/Cas9 gene editing tools have biases against cells from people of African ancestry, leading to false negative results. The study's findings highlight the importance of increasing genetic diversity in large-scale cell line libraries to mitigate this bias.
Scientists have improved a gene-editing technology that can insert or substitute entire genes in the genome, potentially treating multiple genetic diseases with a single therapy. The new approach, eePASSIGE, uses prime editors and recombinase enzymes to make gene-sized edits several times more efficiently than previous methods.
Researchers have engineered an AAV that efficiently crosses the blood-brain barrier and delivers genes to the brain in humanized mice. This could lead to new treatments for severe genetic brain disorders with no current cures or few treatment options.
Scientists have found that prolonged physical activity causes extensive cellular and molecular changes in all 19 organs studied in rats. The study provides potential clues into many human health conditions, including non-alcoholic fatty liver disease and inflammatory liver disease.
A massive genetic study of over 1.2 million people has pinpointed 95 loci associated with PTSD risk, including 80 previously unknown locations. The study also identified 43 genes that appear to contribute to the development of PTSD, providing new targets for prevention and treatment strategies.
Scientists have identified specific species of cholesterol-metabolizing bacteria in people with lower cholesterol levels, potentially leading to new treatments for cardiovascular disease. The study found that certain bacteria in the Oscillibacter genus take up and metabolize cholesterol, resulting in lower cholesterol levels.
Researchers have engineered a new mRNA structure by adding multiple “tails” to boost mRNA activity levels and prolong its presence in the body. The multi-tailed mRNAs increased therapeutic protein production in cells and animals, and showed improved efficiency in gene editing when incorporated into a CRISPR system.
Researchers found similar gene activity changes in brain tissue from people with schizophrenia and older adults, suggesting a shared biological basis for cognitive decline. The study identified the Synaptic Neuron and Astrocyte Program (SNAP), which is tightly coordinated between neurons and astrocytes.
A team of researchers at the Broad Institute has developed 10 polygenic risk scores for use in clinical research, optimizing them for diverse populations. The tests calculate a person's lifetime odds of developing diseases such as heart disease and type 2 diabetes, and can help guide clinical decision-making.
A team of scientists has identified two key human genetic factors that contribute to severe Lassa fever. The study, which involved 16 years of collaborative effort, found a set of LARGE1 variants linked to reduced Lassa fever risk and could lay the foundation for better treatments.
Scientists have developed two injectable priming agents to improve liquid biopsy performance by slowing down the clearance of circulating tumor DNA from the body. In mouse studies, these agents increased circulating tumor DNA levels by more than 10-fold, improving the sensitivity of detecting cancer in mice with low tumor burden.
Researchers develop a delivery system for prime editing, enabling the correction of disease-causing genetic mutations in animal models. By adapting virus-like particles to carry the machinery for prime editing, scientists have achieved partial restoration of vision in mice with two genetic disorders.
Researchers map thousands of cell populations across the mouse brain, revealing diverse cellular patterns in understudied areas. The study provides crucial resources for neuroscience and offers clues about cellular function and disease potential.
A new small-molecule drug candidate has been shown to increase the activity of immune cells and make tumors more vulnerable to immune attack. The molecule, ABBV-CLS-484, works by blocking proteins that normally shut down immune signals, resulting in a powerful dual response against cancer and immune cells.
Researchers have uncovered a new RNA-guided DNA-cutting enzyme called Fanzor in eukaryotes, which has the potential to edit the human genome with precision. The system was found to be more easily delivered to cells and tissues than CRISPR/Cas systems, making it a valuable new technology for human genome editing.
The study highlights 10% of human genome highly conserved across species, linked to embryonic development and environmental adaptation. It also pinpointed genetic variants associated with exceptional traits like brain size and hibernation, and at-risk species for extinction.
Researchers developed a programmable bacterial injection system that can deliver a range of proteins, including those for gene editing, to different cell types. The system has shown promising results in targeting cancer cells and delivering proteins to the brain in live mice without provoking an immune response.
Researchers have engineered a family of adeno-associated viral vectors that can deliver cargo to the primate brain, offering a safer and more efficient way to treat genetic diseases. The PAL family of AAVs has been shown to be three times better at delivering their cargo into the brain than current leading AAV delivery vehicle AAV9.
A new technology called RADARS allows scientists to detect and target specific cell types and states, opening up potential applications in diagnostics and therapeutics. The platform detects a particular RNA sequence in live cells and produces a protein of interest in response.
A machine learning model predicts which SARS-CoV-2 variants will dominate and cause surges in COVID-19 cases, offering a potential early warning system for public health officials. The model, PyR0, can analyze millions of viral genomes in an hour, estimating the effect of genetic mutations on the virus's fitness.
Researchers have created a massive single-cell atlas across human tissues, pinpointing specific cell types involved in multiple diseases. The study uses machine learning methods to query the resulting map, enabling comparisons across tissues and cells from frozen tissue.
A landmark genetic study of over 121,000 people has identified 10 protein-disrupting mutations in genes strongly increasing schizophrenia risk by up to 20-fold. A second study in a larger group of 320,400 people brings the total number of genome regions associated with schizophrenia risk to 287, including genes previously identified.
Researchers have developed a new family of adeno-associated viruses (AAVs) that target muscle tissue more efficiently, reducing the risk of liver damage and allowing for lower doses. This improved delivery method has shown promise in treating genetic muscle diseases, including Duchenne muscular dystrophy and X-linked myotubular myopathy.
A comprehensive molecular map of lung squamous cell carcinoma has identified potential new drug targets, including the gene NSD3, and highlighted immune regulation pathways that could help cancer evade immunotherapies. The study's findings have also revealed metabolic dysregulation and crosstalk between different cellular processes.
Researchers found that patients who developed severe COVID-19 had a muted antiviral response in cells collected from nasal swabs at the time of diagnosis. This early response may determine the course of disease, opening up new avenues for interventions to prevent severe disease.
A comprehensive genome-wide association study identified 13 loci strongly associated with infection or severe COVID-19, including causal factors like smoking and high body mass index. The findings could provide targets for repurposed drugs and illustrate the power of genetic studies in infectious disease.
Researchers used base editing to convert a pathogenic hemoglobin gene to a benign variant, rescuing disease symptoms in animal models and enabling long-lasting production of healthy blood cells. The treatment successfully edited up to 80% of the mutated gene in human blood stem cells and maintained its effects in mice for 16 weeks.
A team of scientists created a cell atlas using autopsy samples from COVID-19 patients, revealing how infected cells and lungs responded to the virus. The study found repeated attempts by the lungs to repair themselves failed due to viral-induced changes in cellular programs.
The Zoonomia Project has released a vast dataset of over 80% of all mammalian families, spanning 110 million years of evolution. This dataset captures mammalian diversity and includes genomes from endangered species.
The GTEx project has mapped genetic variations that affect gene expression across over four dozen tissues, revealing the importance of cell type in understanding gene regulation. The study cataloged QTLs governing the expression of over 23,000 genes, highlighting the complexity underlying genetic control of gene expression.
Researchers discovered that a person's genetic background can alter the risk of heart disease in individuals with familial hypercholesterolemia gene variants. A low polygenic score was found to lower the risk of breast cancer and colorectal cancer in those with high-risk single-gene variants, bringing it closer to the population average.