Lifeboat Foundation

A radical new method of producing drug molecules, which uses downloadable blueprints to easily and reliably synthesise organic chemicals via a programmable ‘chemputer’, could be set to democratise the pharmaceutical industry, scientists say.

He Jiankui has now presented his controversial work at a gene editing summit in Hong Kong. CRISPR expert Helen O’Neill of University College London was there.

The introduction of the gene editing tool CRISPR in 2007 was a revolution in medical science and cell biology. But even though the potential is great, the launch of CRISPR has been followed by debate about ethical issues and the technology’s degree of accuracy and side effects.

However, in a new study published in Cell, researchers from the Novo Nordisk Foundation Center for Protein Research have described how Cas12a, one of the CRISPR technologies, works at the molecular level. This makes it possible to fine-tune the gene-editing process to achieve specific desired effects.

“If we compare CRISPR to a car engine, what we have done is make a complete 3D map of the engine and thus gained an understanding of how it works. This knowledge will enable us to fine-tune the CRISPR engine and make it work in various ways—as a Formula 1 racer as well as an off-road truck,” says Professor Guillermo Montoya from the Novo Nordisk Foundation Center for Protein Research.

Scientists at the annual meeting of the American Society of Hematology will hear about new blood cancer drugs this weekend.

New research examines the risk of heart attack and stroke after an infection, concluding that infections may trigger coronary events.

A couple of days ago, news about the first designer babies has shaken the world. A Chinese researcher, Jiankui He, and his team at the Southern University of Science and Technology of China have been recruiting couples in order to create the world’s first babies with artificially increased resilience to HIV. The embryos were modified using the CRISPR/Cas9 gene editing tool before being implanted in their mother.

According to the research lead, the twins were born healthy a few weeks ago, and genetic testing performed after they were born confirmed that the editing had actually taken place. While the academic community discusses whether it is acceptable or not to modify human genes, taking into account that the changes made will be inherited by these babies’ offspring and are now added to mankind’s genetic pool, I want to share my own views on designer babies.

A Phase 2 trial into the efficacy of a male contraceptive gel is about to get underway bringing modern medicine closer than it ever has before to finally developing a male birth control drug, decades after the female contraceptive pill hit the market.

Flying at 50,000 feet, diving deep in the ocean, or hiking for miles with gear through extreme climates, military service members face conditions that place unique burdens on their individual physiology. The potential exists to develop pharmacological interventions to help service members complete their toughest missions more safely and efficiently, and then recover more quickly and without adverse effects, but those interventions must work on complex physiological systems in the human body. They will not be realized under the prevailing system of drug discovery and development with its focus on engaging single molecular targets. DARPA created the Panacea program to pursue the means of rapidly discovering, designing, and validating new, multi-target drugs that work with the body’s complexity to better support the physiological resilience and recovery of military service members.

The premise of Panacea is that the physiological systems of the human body work in complex and highly integrated ways. Drugs exert effects on our bodies by physically interacting with and changing the functional state of biomolecules that govern the functions of cells and tissues. Most drugs target proteins, which are the principle cellular workhorses. Ideally, drugs would target multiple proteins simultaneously to exert precise, network-level effects.

One major problem facing the drug development community is that the functional proteome — the complete collection of proteins and their roles in signaling networks — is largely dark to science. Despite being able to identify many of the proteins within a cell, researchers do not have a firm grasp on everything those proteins do and how they interact to affect physiology.

Rosalinda Roberts had gotten used to seeing weird shapes in the brain. Over three decades of looking at brain tissue under an electron microscope, she’d regularly come across “unknown objects”—specks and blobs in her images that weren’t supposed to be there, and didn’t seem to relate to the synapses and structure that she was studying. “I’d just say, ‘well I’m not going to pay attention to that’” she explains. That’s all changed now.

Finding bacteria in the brain is usually very bad news. The brain is protected from the bacterial menagerie of the body by the blood-brain barrier, and is considered a sterile organ. When its borders are breached, things like encephalitis and meningitis can result. Which made it all the more surprising when Roberts, along with Charlene Farmer and Courtney Walker, realized that the unknown objects in their slides were bacteria.

Many of them were caught mid-stride, entering neurons or penetrating axons. Others were in the process of dividing. They were picky, too, strongly preferring some regions of the brain over others. The surrounding brain tissue showed no signs of inflammation. If the bacteria were in the brain while the individual was alive, they were not pathogenic.