Lifeboat Foundation

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CRISPR-Cas9 is a revolutionary gene-editing technology that offers the potential to treat diseases such as cancer, but the effects of CRISPR in patients are currently unknown. Stadtmauer et al. report a phase 1 clinical trial to assess the safety and feasibility of CRISPR-Cas9 gene editing in three patients with advanced cancer (see the Perspective by Hamilton and Doudna). They removed immune cells called T lymphocytes from patients and used CRISPR-Cas9 to disrupt three genes (TRAC, TRBC, and PDCD1) with the goal of improving antitumor immunity. A cancer-targeting transgene, NY-ESO-1, was also introduced to recognize tumors. The engineered cells were administered to patients and were well tolerated, with durable engraftment observed for the study duration. These encouraging observations pave the way for future trials to study CRISPR-engineered cancer immunotherapies.

Science, this issue p. eaba7365; see also p. 976.

Chimeric antigen receptor (CAR) T cells have transformed the treatment of refractory blood cancers. These genetically engineered immune cells seek out and destroy cancer cells with precision. Now, scientists at Memorial Sloan Kettering are deploying them against other diseases, including those caused by senescence, a chronic “alarm state” in tissues. The scope of such ailments is vast and includes debilitating conditions, such as fibrotic liver disease, atherosclerosis, and diabetes.

Key to the success of CAR T cell therapy has been finding a good target. The first US Food and Drug Administration-approved CAR T cells target a molecule on the surface of blood cancers called CD19. It is present on cancer cells but few other normal cells, so side effects are limited.

Taking their cue from this prior work, a team of investigators including Scott Lowe, Chair of the Cancer Biology and Genetics Program in the Sloan Kettering Institute, and Michel Sadelain, Director of the Center for Cell Engineering at MSK, along with their trainees Corina Amor, Judith Feucht, and Josef Leibold, sought to identify a target on senescent cells. These cells no longer divide, but they actively send “help me” signals to the immune system.

A human embryo editing experiment gone wrong has scientists warning against treading into the field altogether.

To understand the role of a single gene in early human development, a team of scientists at the London-based Francis Crick Institute removed it from a set of 18 donated embryos. Even though the embryos were destroyed after just 14 days, that was enough time for the single edit to transform into “major unintended edits,” OneZero reports.

Human gene editing is a taboo topic — the birth of two genetically modified babies in 2018 proved incredibly controversial, and editing embryos beyond experimentation is not allowed in the U.S. The scientists in London conducted short-term research on a set of 25 donated embryos, using the CRISPR technique to remove a gene from 18 of them. An analysis later revealed 10 of those edited embryos looked normal, but that the other eight revealed “abnormalities across a particular chromosome,” OneZero writes. Of them, “four contained inadvertent deletions or additions of DNA directly adjacent to the edited gene,” OneZero continues.

Chemical process called ELAST allows labeling probes to infuse more quickly, and makes samples tough enough for repeated handling.

When there’s a vexing problem to be solved, people sometimes offer metaphorical advice such as “stretching the mind” or engaging in “flexible” thinking, but in confronting a problem facing many biomedical research labs, a team of MIT researchers has engineered a solution that is much more literal. To make imaging cells and molecules in brain and other large tissues easier while also making samples tough enough for years of handling in the lab, they have come up with a chemical process that makes tissue stretchable, compressible, and pretty much indestructible.

“ELAST” technology, described in a new paper in Nature Methods, provides scientists a very fast way to fluorescently label cells, proteins, genetic material, and other molecules within brains, kidneys, lungs, hearts, and other organs. That’s because when such tissues can be stretched out or squished down thin, labeling probes can infuse them far more rapidly. Several demonstrations in the paper show that even after repeated expansions or compressions to speed up labeling, tissues snap back to their original form unaltered except for the new labels.

Genetic engineering and other advanced technologies may need to come into play if people want to live in Mars.

Last month’s NASA and SpaceX successful launch of astronauts from US soil for the first time in almost a decade, has reignited discussion about space travel to Mars and beyond. SpaceX is fronted by the billionaire Elon Musk.

Continue reading “Elon Musk makes getting humans to Mars his top priority” »

Although many animals have evolved intrinsic transparency for the purpose of concealment, the development of dynamic, that is, controllable and reversible, transparency for living human cells and tissues has remained elusive to date. Here, by drawing inspiration from the structures and functionalities of adaptive cephalopod skin cells, we design and engineer human cells that contain reconfigurable protein-based photonic architectures and, as a result, possess tunable transparency-changing and light-scattering capabilities. Our findings may lead to the development of unique biophotonic tools for applications in materials science and bioengineering and may also facilitate an improved understanding of a wide range of biological systems.

Here’s What You Need To Remember: Chinese so-called “carrier-killer” missiles could, quite possibly, push a carrier back to a point where its fighters no longer have range to strike inland enemy targets from the air. The new drone is being engineered, at least in large measure, as a specific way to address this problem. If the attack distance of an F-18, which might have a combat radius of 500 miles or so, can double — then carrier-based fighters can strike targets as far as 1000 miles away if they are refueled from the air.

The Navy will choose a new carrier-launched drone at the end of this year as part of a plan to massively expand fighter jet attack range and power projection ability of aircraft carriers.

The emerging Navy MQ-25 Stingray program, to enter service in the mid-2020s, will bring a new generation of technology by engineering a first-of-its-kind unmanned re-fueler for the carrier air wing.

Experts wonder how genetic engineering will eventually reshape the natural world.