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A single-letter edit in DNA reduces levels of the disease-causing prion protein in the brain and could lead to a preventative, one-time treatment for the deadly neurodegenerative disorder.

The new system is the first to use a DNA-mobilizing enzyme called a CRISPR-associated transposase to make targeted gene-sized edits at therapeutically useful levels in human cells.

The new genome-editing tools could lead to safer, more efficient gene therapies.

Ó³»­´«Ã½ scientists built a diverse library of compounds and found one that stabilizes a dysfunctional protein in Crohn’s disease, demonstrating their library’s potential to uncover new therapeutic strategies.

BRD-810 inhibits the MCL1 protein and reactivates apoptosis in tumor cells, displaying therapeutic potential in animal models.

Researchers have developed a machine learning model that connects images of DNA structure to gene regulation.

A suite of new machine learning models can estimate the safety of potential new drugs

New study shows how leveraging unsupervised learning can decode DCIS progression from chromatin images.

The method could accelerate the development of more effective adeno-associated viruses (AAVs).

A new method allows precise activation or repression of genes in specific cells and tissues.

Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance.

Targeting the PELO gene could be a way to kill tumor cells in cancers with one of two common genetic mutations.

Slow-growing glioma tumors become more aggressive when their cells shift identities and acquire new genetic mutations.

Myocarditis is driven by a different immune response than the anti-tumor one, suggesting that the serious complication could one day be managed without halting cancer therapy.

The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

Researchers double the number of genetic factors associated with this common arrhythmia, highlighting biological pathways that could be targeted by new medicines.

Ó³»­´«Ã½ and Denmark-based scientists are collaborating to scale up their efforts to turn genetic insights into the fundamental biological mechanisms underlying metabolic disease.

The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease is forging connections between Danish and Ó³»­´«Ã½ scientists. Here are two of their stories.

The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease brings together Danish and Ó³»­´«Ã½ scientists. Managing director Kasper Lage shares the center’s origin story, its achievements so far, and what the future holds.

By studying a diversity of participants, scientists find nearly 150 new locations in the genome linked to type 2 diabetes risk.

Researchers suggest diabetes exists in multiple forms on a spectrum of varying genetic factors and symptoms.

The clusters point to a range of biological processes that likely contribute to the development of type 2 diabetes.

Specific species and strains of bacteria linked to changes in the functioning of the gut microbiome and a person's risk of type 2 diabetes

Researchers studying brain cells from Huntington’s patients show that the mutation, which changes over decades, becomes toxic only later in life.

Scientists discover possible way to harness immune cells in the brain to remove plaques associated with Alzheimer's.

Scientists have uncovered new genetic links to different types of epilepsy, which could lead to more tailored treatments.

Ralda Nehme, director of Ó³»­´«Ã½â€™s stem cell program, talks about how stem cells can be a model for disease in a dish and what scientists can learn from these experiments.

Scientists find combinations of known antibiotics and other molecules that, together, have greater bacteria-killing power.

Researchers examined scat from hundreds of animals around the world and pinpointed genes that could fuel drug-resistant infections.

The microbes control the growth of harmful bacteria in mice, promoting a healthier intestinal microbiome.

A low-cost CRISPR-based paper strip test distinguishes between influenza types and can be reprogrammed to recognize different viruses including the H5N1 bird flu virus.

Scientists from the US and West Africa have teamed up to build a better public health network that can quickly detect and respond to emerging viral threats.

In a pilot study with patient samples, the technology performed as well as the current gold-standard methods, yielding accurate results within hours instead of days.