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Category: genetics – Page 3

Fusion superkine and focused ultrasound could enable targeted, noninvasive therapy for glioblastoma

Researchers at VCU Massey Comprehensive Cancer Center and the VCU Institute of Molecular Medicine (VIMM) have discovered a new and potentially revolutionary way to treat glioblastoma (GBM), the most aggressive type of brain cancer, which currently has no curative treatment options.

In a study led by Paul B. Fisher, MPh, Ph.D., FNAI, and Swadesh K. Das, Ph.D., recently published in the Journal for ImmunoTherapy of Cancer, researchers created a that demonstrates the ability to introduce a combination of treatment outcomes—direct toxicity and immunotoxicity—to kill the tumor while exploiting immunotherapy to potentially prevent the recurrence of GBM. The new molecule, a fusion superkine (FSK), contains dual-acting therapeutic cytokines in a single molecule.

“This is the tip of the iceberg,” said Dr. Fisher, the Thelma Newmeyer Corman Endowed Chair in Cancer Research at Massey, director of the VIMM and professor in the Department of Cellular, Molecular and Genetic Medicine. “We’re optimistic that our first trial in , planned for 2026, will show that the IL-24 gene and these therapeutic viruses are effective and safe. And [the FSK] will be the one knocking it out of the ballpark.

‘ALS on a chip’ model reveals altered motor neuron signaling

Using stem cells from patients with ALS (amyotrophic lateral sclerosis), Cedars-Sinai has created a lifelike model of the mysterious and fatal disease that could help identify a cause of the illness as well as effective treatments.

In a study published in the journal Cell Stem Cell, investigators detail how they created “ALS on a chip” and the clues the specialized laboratory chip has already produced about nongenetic causes of the disease, also known as Lou Gehrig’s disease.

The work builds on previous studies where adult cells from ALS patients were reverted into . The cells were then pushed forward to produce motor neurons, which die in the disease, causing progressive loss of the ability to move, speak, eat and breathe.

Gene editing treats smooth muscle disease in preclinical model

Using gene editing in a preclinical model, researchers at UT Southwestern Medical Center blocked the symptoms of a rare smooth muscle disease before they developed. Their findings, published in Circulation, could eventually lead to gene therapies for this and other genetic diseases affecting smooth muscle cells.

“Gene editing has been used in other disease contexts, but its application to inherited vascular diseases, particularly targeting in vivo, is still emerging. Our approach advances the field by demonstrating functional correction in a cell type that’s notoriously difficult to target,” said Eric Olson, Ph.D., Chair and Professor of Molecular Biology and a member of the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern.

Dr. Olson co-led the study with Ning Liu, Ph.D., Professor of Molecular Biology, and first author Qianqian Ding, Ph.D., postdoctoral researcher, both members of the Olson Lab.

Rare Gene Mutation Delays Alzheimer’s by Damping Immune Cell Inflammatory Signaling

Researchers at Weill Cornell Medicine report that a rare gene mutation that delays Alzheimer’s disease does so by damping inflammatory signaling in brain-resident immune cells in a preclinical study. The finding adds to growing evidence that brain inflammation is a major driver of neurodegenerative disorders such as Alzheimer’s—and that it may be a key therapeutic target for these disorders.

In their study “The R136S mutation in the APOE3 gene confers resilience against tau pathology via inhibition of the cGAS-STING-IFN pathway,” in Immunity, the investigators examined the effects of the mutation APOE3-R136S—known as the “Christchurch mutation”—which was recently found to delay hereditary early-onset Alzheimer’s. The scientists showed that the mutation inhibits the cGAS-STING pathway, an innate immune signaling cascade that is abnormally activated in Alzheimer’s and other neurodegenerative diseases. The researchers found that pharmacologically blocking the cGAS-STING pathway with a drug-like inhibitor replicated key protective effects of the mutation in a preclinical model.

“This is an exciting study because it suggests that inhibiting this cGAS-STING pathway could make the brain more resistant to the Alzheimer’s process, even in the face of significant tau accumulation,” said study senior author Li Gan, PhD, the Burton P. and Judith B. Resnick Distinguished Professor in Neurodegenerative Diseases and director of the Helen and Robert Appel Alzheimer’s Disease Research Institute at Weill Cornell Medicine.

“Delete-To-Recruit” — Scientists Discover Simpler Approach to Gene Therapy

Repositioning genes awakens fetal hemoglobin to treat disease. CRISPR editing may change future gene therapy.

Researchers have discovered a promising new approach to gene therapy by reactivating genes that are normally inactive. They achieved this by moving the genes closer to regulatory elements on the DNA known as enhancers. To do so, they used CRISPR-Cas9 technology to cut out the piece of DNA separating the gene from its enhancer. This method could open up new ways to treat genetic diseases. The team demonstrated its potential in treating sickle cell disease and beta-thalassemia, two inherited blood disorders.

In these cases, a malfunctioning gene might be bypassed by reactivating an alternative gene that is usually turned off. This technique, called “delete-to-recruit,” works by altering the distance between genetic elements without introducing new genes or foreign material. The study was conducted by researchers from the Hubrecht Institute (De Laat group), Erasmus MC, and Sanquin, and published in the journal Blood.

Human Cyborgs Are No Longer Science Fiction! (Insane Breakthroughs)

Are human cyborgs the future? You won’t believe how close we are to merging humans with machines! This video uncovers groundbreaking advancements in cyborg technology, from bionic limbs and brain-computer interfaces to biological robots like anthrobots and exoskeletons. Discover how these innovations are reshaping healthcare, military, and even space exploration.

Learn about real-world examples, like Neil Harbisson, the colorblind cyborg artist, and the latest developments in brain-on-a-chip technology, combining human cells with artificial intelligence. Explore how cyborg soldiers could revolutionize the battlefield and how genetic engineering might complement robotic enhancements.

The future of human augmentation is here. Could we be on the verge of transforming humanity itself? Dive in to find out how science fiction is quickly becoming reality.

How do human cyborgs work? What are the latest AI breakthroughs in cyborg technology? How are cyborgs being used today? Could humans evolve into hybrid beings? This video answers all your questions. Don’t miss it!

#ai.
#cyborg.
#ainews.

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Two proteins that could lead to less toxic cancer treatments identified

Cells depend on the precise reading of DNA sequences to function correctly. This process, known as gene expression, determines which genetic instructions are activated. When this fails, the wrong parts of the genome can be activated, leading to cancers and neurodevelopmental disorders.

Scientists at the University of Geneva (UNIGE) have identified two proteins that play a key role in regulating this essential mechanism, paving the way for promising new treatments that could be more effective and less toxic than those currently available. Their findings are published in Nature Communications.

Human DNA contains over 20,000 genes and would stretch nearly two meters if fully uncoiled. To fit this enormous amount of information into a tiny space within a cell—just 10 to 100 micrometers in diameter—it must be tightly compacted. This is the job of , a complex of proteins that packages and condenses DNA within the .

Detailed imaging of key receptors suggests new avenue for repairing brain function

For the first time, scientists using cryo-electron microscopy have discovered the structure and shape of key receptors connecting neurons in the brain’s cerebellum, which is located behind the brainstem and plays a critical role in functions such as coordinating movement, balance and cognition.

The research, published in Nature, provides new insight that could lead to the development of therapies to repair these structures when they are disrupted either by injury or affecting —sitting, standing, walking, running, and jumping—learning and memory.

The study, by scientists at Oregon Health & Science University, reveals the organization of a specific type of glutamate receptor—a that conveys signals between neurons and is considered the primary excitatory neurotransmitter in the brain—bound together with proteins clustered on synapses, or junctions, between neurons in the cerebellum.

Synthetic ‘killswitch’ uncovers hidden world of cellular condensates

Researchers at the Max Planck Institute for Molecular Genetics have developed a novel synthetic micropeptide termed the “killswitch” to selectively immobilize proteins within cellular condensates, unveiling crucial connections between condensate microenvironments and their biological functions.

Biomolecular condensates are specialized regions inside cells, existing without membranes, where critical biochemical reactions occur. Their importance in health and disease is well established, including roles in cancer progression and viral infection.

Methods to precisely probe and manipulate condensates in living cells remain limited. Existing strategies lack specificity, either dissolving condensates indiscriminately or requiring artificial protein overexpression, which obscures the natural behavior of native cellular proteins.