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Category: genetics – Page 5

Lentiviral In Vivo CD19 CAR TCell Therapy in Neurologic Autoimmune Disorders

An international team of scientists has successfully tested an innovative in vivo CAR-T therapy, JY231, capable of treating severe autoimmune diseases with a single intravenous infusion. Published in the New England Journal of Medicine, the experimental treatment uses a harmless genetically modified viral vector to deliver genetic instructions directly into a patient’s T-lymphocytes, reprogramming them to target and destroy defective B-cells that produce autoantibodies. In a clinical trial involving 16 patients with treatment-resistant conditions such as multiple sclerosis, myasthenia gravis, and myopathy, the therapy demonstrated over 99% accuracy. Within two months, patients’ bone marrow began producing healthy immune cells, and after six months of observation, participants exhibited significant clinical improvements, including the elimination of chronic fatigue and the partial restoration of cognitive and muscular functions. Researchers are now preparing for large-scale randomized trials to definitively confirm the method’s long-term safety and efficacy.

Among 16 patients with refractory neurologic autoimmune disorders, lentiviral CD19 CAR T-cell therapy was associated with manageable side effects, complete B-cell depletion, and preliminary clinical improvement across disease groups.

Researchers traced Leonardo da Vinci’s family through 21 generations and found matching Ychromosome markers in six living men from a Renaissance family tomb could connect that genetic trail directly to his time

A genetic thread stretches across five centuries, connecting living descendants to the Renaissance master through his prolific father—and newly excavated remains could prove it.

Super-resolution imaging reveals that cohesin prevents local mixing of compact, active genome domains

The human genome is about two meters (6.6 feet) long, yet it is folded inside a cell nucleus only about 10 micrometers in diameter. To fit into this tiny space, DNA is wrapped around histone proteins to form nucleosomes, which are further organized into chromatin. For decades, chromatin has often been described in two simple forms: euchromatin, which is active, open and accessible, and heterochromatin, which is more compact and repressed.

However, a new study from an international team led by Kazuhiro Maeshima, a professor at the National Institute of Genetics, ROIS (Research Organization of Information and Systems) and SOKENDAI, has challenged this simple textbook view. The researchers demonstrated that euchromatin in living human cells is not merely open and loose but forms dynamic condensed domains. This domain organization helps prevent the mixing of neighboring domains.

The team further found that cohesin, a ring-shaped protein complex best known for organizing genome architecture, prevents local mixing between these condensed euchromatic domains for proper gene regulation in living human cells. The study was published in Nature Genetics on Sept. 8, 2026.

Your Mother’s Age May Leave a Biological Mark That Lasts for Generations

Maternal age effects may arise from reversible epigenetic changes whose impact depends partly on genetics and evolutionary pressures.

A mother’s age can leave a biological imprint on her offspring, influencing physical and behavioral traits across many animal species, including humans. These so-called maternal age effects are widespread, but scientists still do not fully understand the biological mechanisms behind them or why evolution has allowed them to persist.

“Maternal age effects are incredibly common, from invertebrates up through humans, elephants, other primates and other mammals,” said Kristin Gribble, an associate scientist in the Bay Paul Center at the Marine Biological Laboratory. “Nearly all forms of life show some level of maternal age effect, and most are negative effects caused by advanced maternal age.”

Gene circuits of human immunity

Scientists have unveiled a massive, high-resolution functional map of human immune cells that promises to transform our understanding of how genetics control health and disease.

Published in the journal Cell, the study represents a landmark achievement in immunology and genomics. By systematically stress-testing genes across the genome in 22 million human immune cells, scientists moved beyond mere DNA sequencing to decode the dynamic circuits that govern how these genes actually work in the context of health and disease. This leap from observation to intervention offers a powerful new framework for designing cancer immunotherapies and treating autoimmune conditions, among other things.

“To understand the significance of this study, you have to look at the last three decades of biology,” says a senior author of the study. “First came the Human Genome Project, which gave us the blueprint of our genes. Then, projects like the Human Cell Atlas showed us how different cells read that blueprint. Now, we’re in a grand third wave: discovering what happens to cells when you make targeted changes within the genome. We finally have a way to decode the link between genetic sequence and cell state.”

Type 1 Diabetes May Actually Be Two Different Diseases, Massive International Study Finds

Identifying distinctive types of conditions like diabetes is helpful in two ways.

It means treatments can be personalized more precisely to individuals, and gives researchers a better idea of how to make those treatments more effective going forward.

When it comes to type 1 diabetes, there are two genetic patterns called HLA-DR3 and HLA-DR4 that are associated with a higher risk of the disease. However, while the end result is the same (type 1 diabetes), the early signs differ between the two patterns.

Rewiring of protein interaction networks by autism mutations

For more than two decades, researchers have identified hundreds of genes that increase the risk of autism spectrum disorder (ASD). Yet multiple fundamental questions have remained unanswered: among them, how do mutations in these genes lead directly to changes in brain development and how can that knowledge be translated into more effective therapies?

In a landmark study published in Science, scientists have taken a major step toward answering both questions. The findings are the result of more than a decade of work. By building the largest-ever molecular interaction map of autism, the team revealed how hundreds of genes and dozens of mutations converge within a surprisingly small number of shared protein networks, hurdling a major roadblock to the development of new precision medicines.

Rather than focusing only on the genes linked to autism, the researchers mapped the proteins encoded by those genes and discovered exactly how individual disease-causing mutations can rewire the molecular machinery of the developing brain. The work uncovers an entirely new layer of disease biology that can be targeted therapeutically and provides a framework for designing medicines that directly address a wide range of underlying molecular causes of autism.

Resistant Hypertension Variants Link to Hyperaldosteronism and Potassium Levels

BACKGROUND: We aimed to characterize the genetic architecture of resistant hypertension (rHTN), which affects up to 18% of hypertensive individuals and increases cardiovascular disease risk. METHODS: We conducted a genome-wide association study on rHTN, defined as use of 3 or more concomitant antihypertensive drugs for at least 6 months without reaching blood pressure target (in the 3-drug case), comparing it to controlled hypertension (cHTN), in which persons on 1 or 2 antihypertensives for at least 6 months reach target BP after 30 days of therapy initiation. The study included 23 508 rHTN cases and 24 393 cHTN controls, identified through drug prescription and blood pressure data from Iceland (deCODE), the UK (UK Biobank), and the US (eMERGE).

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