A novel gene therapy to lower the risk of cancer!

The researchers expect that the therapy could be ready for clinical trials within five years.

 US scientists have developed a new gene therapy that can help reduce the development of cancer cells that are an infrequent but dangerous by-product of gene therapies. Gene therapy holds potential for treating genetic diseases by replacing defective genes with repaired ones. It has shown promise in clinical trials but has also been set back by difficulties in delivering genes, getting them to work for a long time and safety issues. The findings were converted into a stem cell gene therapy to target a life-threatening immune deficiency in newborns called Severe Combined Immunodeficiency (SCID-X1), also known as ‘Boy in the Bubble Syndrome’ — a genetic disorder which results in an extreme vulnerability to infectious diseases.

 Our goal is to develop a safe and effective therapy for SCID-X patients and their families,’ said Grant Trobridge, Associate Professor at Washington State University. The researchers developed a vector from a foamy retrovirus — a natural choice for gene therapy because they work by inserting their genes into a host’s genome. The team altered it to change how it interacts with a target stem cell so it would insert itself into safer parts of the genome. They found that it integrated less often near potential cancer-causing genes. Unlike other retroviruses, they don’t normally infect humans. They also are less prone to activate nearby genes, including genes that might cause cancer. The researchers expect that the therapy could be ready for clinical trials within five years. 

The developments are published in the journal of Science Reports.

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Pomegranates May Help Fight Ageing

Researchers have found that a molecule in pomegranates, transformed by microbes in the gut, may enable muscle cells to protect themselves against one of the major causes of ageing.

As we age, our cells increasingly struggle to recycle their powerhouses.

Called mitochondria, these inner compartments are no longer able to carry out their vital function, and thus accumulate in the cell, researchers said.

This degradation affects the health of many tissues, including muscles, which gradually weaken over the years.

A buildup of dysfunctional mitochondria is also suspected of playing a role in other diseases of ageing, such as Parkinson's disease, they said.

Scientists from Ecole Polytechnique Federale de Lausanne (EPFL) and Amazentis company in Switzerland identified a molecule that, all by itself, managed to re-establish the cell's ability to recycle the components of the defective mitochondria: urolithin A.

"It is the only known molecule that can relaunch the mitochondrial clean-up process, otherwise known as mitophagy. It is a completely natural substance, and its effect is powerful and measurable," said Patrick Aebischer from EPFL.

Researchers started out by testing their hypothesis on the usual suspect: the nematode C elegans.

It is a favourite test subject among ageing experts, because after just 8-10 days it is already considered elderly, they said.

The lifespan of worms exposed to urolithin A increased by more than 45 per cent compared with the control group, they said.

The results led researchers to test the molecule on animals that have more in common with humans.

In the rodent studies, like with C elegans, a significant reduction in the number of mitochondria was observed, indicating that a robust cellular recycling process was taking place.

Older mice, around two years of age, showed 42 per cent better endurance while running than equally old mice in the control group.

The fruit does not itself contain the 'miracle molecule', but rather its precursor.

That molecule is converted into urolithin A by the microbes that inhabit the intestine, researchers said.

Because of this, the amount of urolithin A produced can vary widely, depending on the species of animal and the flora present in the gut microbiome.

Some individuals do not produce any at all, they said.

Researchers are currently conducting first clinical trials to test the molecule in humans.

The findings were published in the journal Nature Medicine.

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Blood Pressure Could Be Linked To A Stiffening Of The Arteries That Occurs With Age

About 90 percent of all high blood pressure cases cannot be explained by medical experts, a Norwegian University of Science and Technology news release reported.

"Our results suggest that arterial stiffness represents a major therapeutic target. This is contrary to existing models, which typically explain high blood pressure in terms of defective kidney function," says Klas Pettersen, a researcher at the Norwegian University of Life Sciences and first author of the study, recently published in PLOS Computational Biology.

High blood pressure can raise the risk of "heart failure, stroke and kidney disease," the news release reported.

When blood pressure travels from the aorta to the heart, sensors called baroreceptors, sense the stretching of the aortic wall and send signals to the nervous system; the higher the blood pressure the higher these signals are.

If the aorta stiffens (which generally happens in old age) it starts to lose its ability to sense the amount of blood pressure imposing pressure.

"With the stiffening of the wall that follows ageing, these sensors become less able to send signals that reflect the actual blood pressure. Our mathematical model predicts the quantitative effects of this process on blood pressure," Klas Pettersen, a researcher at the Norwegian University of Life Sciences and first author of the study said in the news release.

"If our hypothesis is proven right, arterial stiffness and baroreceptor signaling will become hotspot targets for the treatment of high blood pressure and the development of new medicines and medical devices," Stig W. Omholt from the Norwegian University of Science and Technology, who was the senior investigator of the research project, said in the news release.

The team hopes to show that the stiffening of the aorta causes the baroreceptors to misinform the nervous system, evoking an inappropriate response.

"If we are to succeed in developing predictive, preventive and participatory medicine envisioned by so many, there is no substitute for building much stronger transdisciplinary ties between the life sciences, the mathematical sciences and engineering across the whole spectrum of basic, translational and applied research. And mathematical models of the human physiology will be at the core of this development," Omholt said.

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