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Single drug provides first evidence of ‘nearly universal’ pharmacological chaperone for rare disease

A study published in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilize nearly all mutated versions of a human protein, regardless of where the mutation is in the sequence.

The researchers engineered seven thousand versions of the vasopressin V2 receptor (V2R), which is critical for normal kidney function, creating all possible mutated variants in the lab.

Faulty mutations in V2R prevent from responding to the hormone vasopressin, leading to the inability to concentrate urine and resulting in excessive thirst and large volumes of dilute urine, causing nephrogenic diabetes insipidus (NDI), also known as arginine vasopressin resistance, a rare disease affecting roughly one in 25,000 people.

Stem Cells Repair Brain Damage Caused by Stroke in Mice

Brain damage caused by blocked blood vessels may be treatable using injections of stem cells, according to a new study by researchers from the University of Zurich and the University of Southern California.

The results could one day help patients who have experienced some forms of stroke recover lost functions.

Using mice with stroke-induced brain damage, the researchers found that injections of human stem cells could successfully develop into immature brain cells. The results were dramatic: most of the implanted cells remained in place, developing features of fully functioning neurons and communicating with surrounding cells.

A mobile robot scientist capable of carrying out experiments by itself

We live in a time when robots can clean our homes, drive our vehicles, deactivate bombs, offer prosthetic limbs, help healthcare workers, read the news, entertain, teach, and many more. And now, there is a robot scientist that can work on behalf of humans 24 hours a day, seven days a week.

Researchers at the University of Liverpool have built an intelligent “robot scientist” capable of moving around a laboratory and carrying out scientific experiments by itself. The first of its kind machine with humanoid dimensions are designed to work in a standard laboratory, using instruments much as a human researcher does. It can also make its own decisions about which chemistry experiments to perform next.

The robot scientist is 1.75-meter tall, weighs around 400 kg, and can roam around the laboratory, performing a wide range of different tasks. Unlike a human being, the robot has infinite patience, can think in 10 dimensions, and works for 21.5 hours each day, pausing only to recharge its battery for two hours. This will allow scientists to automate time-consuming and tedious research they wouldn’t otherwise tackle.

Should We Sleep Outside? Turns Out There Are Some Benefits

A 2018 review became the first meta-analysis to quantify the impact of what they termed “greenspace exposure”. It sifted through five online databases leading up to January 2017 to look at the health outcomes of exposure to the great outdoors and found that “green prescriptions” could have substantial benefits to human health.

Perks mentioned included decreased salivary cortisol (the “stress hormone”), lower heart rate, blood pressure, and cholesterol, among others.

“Incidence of stroke, hypertension, dyslipidaemia, asthma, and coronary heart disease were reduced,” wrote the authors. “For several non-pooled health outcomes, between 66.7% and 100% of studies showed health-denoting associations with increased greenspace exposure, including neurological and cancer-related outcomes, and respiratory mortality.”

The latest on nucleotide therapy development

Oligonucleotide therapies — engineered strands of DNA or RNA — are transforming modern medicine. These cutting-edge treatments bring a new level of precision in combating disease by targeting specific genes to be silenced, activated or edited. “Nucleotide therapeutics allow us to design predictable outcomes by modifying sequences to address almost any condition,” says Peter Guterstam, product manager at biotechnology company Cytiva.

Due to an influx of research in recent years, many nucleotide-based drug candidates, including genetic therapies and vaccines for cancer and viral infections, are now in advanced clinical trial stages. “The development timeline is much quicker than we are used to,” notes Guterstam.


Significant challenges arise during development of RNA and DNA based therapies. From mRNA vaccines to gene editing, scientists are refining delivery methods, optimizing synthesis, and tackling scaling hurdles.

GIST Research reveals a promising new target to thwart Alzheimer’s decades before symptoms start

A person will have Alzheimer’s years before ever knowing it. The disorienting erasure of memories, language, thoughts—in essence, all that makes up one’s unique sense of self—is the final act of this enigmatic disease that spends decades disrupting vital processes and dismantling the brain’s delicate structure.

Once symptoms surface and doctors make a diagnosis, though, it can often be too late. Damage is widespread, impossible to reverse. No cure exists.

Attempts to develop drugs that clear away toxic accumulations of amyloid-beta and tau proteins—hallmarks of the disease that cause neurons to die—have ended in hundreds of failed clinical trials. Today, some scientists are skeptical over whether removing amyloid plaques is even enough. Others have a hunch that the best line of attack won’t target just one aspect of the disease, but many of them, all at once.

Breakthrough 3D Bioprinted Mini Placentas May Help Solve “One Of Medicine’s Great Mysteries”

To address these shortcomings, the team behind the latest study turned to bioprinting – a type of 3D printing that uses living cells and cell-friendly materials to create 3D structures. They took trophoblast cells and mixed them with a synthetic gel before 3D-printing them in precise droplets.

The printed cells then grew into miniature placentas, and the researchers compared them to organoids made via traditional manual methods.

“The organoids we grew in the bioprinted gel developed differently to those grown in an animal-derived gel, and formed different numbers of trophoblast sub-types. This highlighted that the environment organoids are grown in can control how they mature,” first author Dr Claire Richards said.

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