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Category: biotech/medical

CRISPR has been heralded as one of the most important breakthroughs in modern science, but there could be a hidden and potentially dangerous side effect to the wonders of its genetic editing technology, a new study reveals.

A systematic investigation of CRISPR/Cas9 genome editing in mouse and human cells has discovered that the technique appears to frequently cause extensive mutations and genetic damage that the researchers say wouldn’t be detected by existing DNA tests.

“This is the first systematic assessment of unexpected events resulting from CRISPR/Cas9 editing in therapeutically relevant cells,” explains geneticist Allan Bradley from the Wellcome Sanger Institute in the UK.

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Exciting new research from Yale University has revealed a new method that could potentially objectively diagnose if a person is suffering through the early stages of Alzheimer’s disease using a non-invasive PET scan.

A major roadblock slowing down effective Alzheimer’s research is our inability to easily, or clearly, diagnose the disease at its early stages. Several blood tests are being explored that can identify biomarkers signaling the early presence of the disease, but nothing has proved conclusive enough to move into general clinical use.

The new Yale University innovation uses PET imaging technology to evaluate cognitive decline by effectively measuring how much synaptic loss or degradation has occurred in a patient’s brain. To quantify a person’s “synaptic density” the researchers homed in on a protein called SV2A. This protein is found in nearly all healthy synapses, but as those connections degrade, so does the presence of SV2A.

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Researchers at the Center for Nanoscale BioPhotonics (CNBP) have developed a new targeted treatment for cancer. Chemotherapy drugs are wrapped in “nano-bubbles” called liposomes, which are then injected into the desired part of the body and made to release their payload on demand, by applying X-ray radiation.

Liposomes are regularly used to protect drugs and carry them to where in the body they’re needed. Over the years, we’ve seen them used to protect insulin doses from the harsh environment of the gut long enough for it to enter the bloodstream, disarm bacteria without using antibiotics, and escort cancer-killers to tumors.

“Liposomes are already well established as an extremely effective drug-delivery system,” says Wei Deng, lead author of the study. “Made out of similar material as cell membranes, these ‘bubbles’ are relatively simple to prepare, can be filled with appropriate medications and then injected into specific parts of the body. The issue however, is in controlling the timely release of the drug from the liposome.”

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Groundbreaking research shows that neurological health depends as much on signals sent by the body’s large, leg muscles to the brain as it does on directives from the brain to the muscles. Published today in Frontiers in Neuroscience, the study fundamentally alters brain and nervous system medicine — giving doctors new clues as to why patients with motor neuron disease, multiple sclerosis, spinal muscular atrophy and other neurological diseases often rapidly decline when their movement becomes limited.

“Our study supports the notion that people who are unable to do load-bearing exercises — such as patients who are bed-ridden, or even astronauts on extended travel — not only lose muscle mass, but their body chemistry is altered at the cellular level and even their nervous system is adversely impacted,” says Dr. Raffaella Adami from the Università degli Studi di Milano, Italy.

The study involved restricting mice from using their hind legs, but not their front legs, over a period of 28 days. The mice continued to eat and groom normally and did not exhibit stress. At the end of the trial, the researchers examined an area of the brain called the sub-ventricular zone, which in many mammals has the role of maintaining nerve cell health. It is also the area where neural stem cells produce new neurons.

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Scientists have discovered a “Big Bang” of Alzheimer’s disease – the precise point at which a healthy protein becomes toxic but has not yet formed deadly tangles in the brain.

A study from UT Southwestern’s O’Donnell Brain Institute provides novel insight into the shape-shifting nature of a tau molecule just before it begins sticking to itself to form larger aggregates. The revelation offers a new strategy to detect the devastating disease before it takes hold and has spawned an effort to develop treatments that stabilize tau proteins before they shift shape.

“This is perhaps the biggest finding we have made to date, though it will likely be some time before any benefits materialize in the clinic. This changes much of how we think about the problem,” said Dr. Marc Diamond, Director for UT Southwestern’s Center for Alzheimer’s and Neurodegenerative Diseases and a leading dementia expert credited with determining that tau acts like a prion – an infectious that can self-replicate.

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“Every time you miss a protein crystal, because they are so rare, you risk missing on an important biomedical discovery.”

- Patrick Charbonneau, Duke University Dept. of Chemistry and Lead Researcher, MARCO initiative.

Protein crystallization is a key step to biomedical research concerned with discovering the structure of complex biomolecules. Because that structure determines the molecule’s function, it helps scientists design new drugs that are specifically targeted to that function. However, protein crystals are rare and difficult to find. Hundreds of experiments are typically run for each protein, and while the setup and imaging are mostly automated, finding individual protein crystals remains largely performed through visual inspection and thus prone to human error. Critically, missing these structures can result in lost opportunity for important biomedical discoveries for advancing the state of medicine.

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