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UCLA team developing inhalable gene-editing treatment for CF

Researchers at the University of California, Los Angeles (UCLA) are developing a gene-editing therapy — designed to be delivered as a one-time inhalable treatment — that aims to correct the underlying mutations that cause cystic fibrosis (CF).

The team is using tiny fat-based particles to deliver the gene-editing machinery to lung stem cells, where, they believe, gene correction could be permanent. The treatment, according to a university news story, could offer hope to people with the genetic disease who do not benefit from current therapies.

CF is caused by mutations in the CFTR gene, which normally produces a protein of the same name. This CFTR protein helps regulate the flow of water and salt molecules in and out of certain cells, which is essential for the production of mucus. In CF, missing or dysfunctional CFTR instead results in the accumulation of thick and sticky mucus in several organs, particularly the lungs. This, in turn, leads to symptoms like shortness of breath, cough, and frequent lung infections.

Protective gene found to preserve mitochondria during kidney disease progression

Research led by Children’s Hospital of Fudan University in China has found that a gene called pancreatic progenitor cell differentiation and proliferation factor (PPDPF) helps protect kidney cells by supporting enzymes involved in maintaining cellular energy levels during chronic kidney disease.

Chronic kidney disease affects approximately 15% of the global population and is currently the ninth leading cause of death worldwide. Treatments that can slow the progression of this condition remain limited.

Genome-wide association studies have identified nearly 800 genetic loci associated with kidney function, yet more than 90% of these variants are located in noncoding regions. Specific genes and involved in early-stage remain incompletely understood.

Lilly’s lepodisiran reduced levels of genetically inherited heart disease risk factor, lipoprotein(a), by nearly 94% from baseline at the highest tested dose in adults with elevated levels

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Scientists Reveal the Hidden Chemistry of Air Pollution

The interactions between light and nitroaromatic hydrocarbon molecules have important implications for chemical processes in our atmosphere that can lead to smog and pollution. However, changes in molecular geometry due to interactions with light can be very difficult to measure because they occur at sub-Angstrom length scales (less than a tenth of a billionth of a meter) and femtosecond time scales (one millionth of a billionth of a second).

The relativistic ultrafast electron diffraction (UED) instrument at the Linac Coherent Light Source (LCLS) at SLAC National Accelerator Laboratory provides the necessary spatial and time resolution to observe these ultrasmall and ultrafast motions. The LCLS is a Department of Energy (DOE) Office of Science light source user facility.

In this research, scientists used UED to observe the relaxation of photoexcited o–nitrophenol. Then, they used a genetic structure fitting algorithm to extract new information about small changes in the molecular shape from the UED data that were imperceptible in previous studies. Specifically, the experiment resolved the key processes in the relaxation of o-nitrophenol: proton transfer and deplanarization (i.e., a rotation of part of the molecule out of the molecular plane). Ab-initio multiple spawning simulations confirmed the experimental findings. The results provide new insights into proton transfer-mediated relaxation and pave the way for studies of proton transfer in more complex systems.

Israeli startup grows world’s first real dairy protein in potatoes—no cows needed

Israeli food-tech startup Finally Foods has developed the world’s first genetically engineered potatoes containing cow-milk protein, a breakthrough that could revolutionize dairy production.

The company, part of Strauss Group’s The Kitchen food-tech incubator, is set to launch its first field trial next month in southern Israel, where the modified potatoes will be cultivated.

Once harvested, the potatoes will be processed to extract casein protein powder, a key component in dairy production. Casein, which makes up 80% of milk proteins, is essential for cheese-making and provides melting, stretching and foaming properties in dairy products.

Horses, donkeys and zebras have adaptations that break normal genomic rules

A genetic mutation in horses that would typically halt protein production has become a molecular asset. Researchers at Johns Hopkins University and Vanderbilt University have identified a rare instance of genetic recoding that enhances oxygen metabolism and energy production in horses, donkeys, and zebras.

The findings, published in Science, provide insight into the genetic foundation of exceptional equine athletic ability, and hint at an entirely new way of dealing with stop codons.

Few mammals match horses in aerobic performance. Muscle tissue in thoroughbreds consumes oxygen at rates exceeding 360 liters per minute. Oxygen uptake per unit of body mass is more than twice that of elite human athletes. While many genes involved in muscle structure and locomotion have been studied, the genetic basis for this level of metabolic output has remained unclear.

Scientists CRACKED Aging… But Is Living to 150 Worth It?!

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A Circuit Linking DNA Repair, Inflammation & Aging is Found

As our bodies grow, cells proliferate to form tissues, and cells frequently have to be repaired or replaced throughout life. But the genome can also become less stable over time, or may pick up mutations that can lead to disease; these and other processes can cause cells to enter a state in which they stop dividing, known as senescence. Senescent cells become more common as we age. There also tends to be more inflammation as we age, but the link between increasing instability in the genome and inflammation is not well understood. Now scientists have reported a direct connection between DNA instability and inflammation in senescent cells. The findings have been reported in Nature Communications.

“In addition to no longer growing and proliferating, the other hallmark of senescent cells is that they have this inflammatory program causing them to secrete inflammatory molecules,” noted senior study author Peter Adams, Ph.D., director and professor of the Cancer Genome and Epigenetics Program at Sanford Burnham Prebys.