Lifeboat Foundation

He Jiankui, the Chinese scientist who faced international condemnation in 2018 for creating the first gene-edited children, has presented a new research proposal involving modifying human embryos to address the challenges of the ageing population. He, who was sentenced to three years in prison in 2019 for “illegal medical practices,” resurfaced last year and announced the opening of a research lab in Beijing.

One of the oldest known molecules, the ribosome, creates proteins based on a copy of the genetic code found in the genome, known as mRNA. Scientists have believed that the ribosome performed the same type of work with all mRNA, like a standardized assembly line that it did not regulate on its own. However, researchers from the University of Copenhagen have discovered this is not the case.

Their findings are published in the journal Developmental Cell in an article entitled “Ribosomal RNA 2′-O-methylation dynamics impact cell fate decisions.”

“It has long been known that there are different types of ribosomes. But it has been assumed that no matter what mRNA you give the ribosome, it will produce a protein. But our results suggest that different types of ribosomes produce specific proteins,” says Anders H. Lund, professor at the Biotech Research and Innovation Center at the University of Copenhagen.

Recombinant adeno-associated virus (rAAV) vectors are a cornerstone of genetic medicine. These hollow virus particles are used to deliver commercial gene therapies like Glybera and Luxturna, as well as many other candidates still in development.

Despite this, the biopharmaceutical industry has yet to find the most effective way of making rAAV vectors at scale. McKinsey analysts recently described vector production as one of the major challenges faced by gene therapy developers. And the situation is only going to get worse as the gene therapy market evolves and begins developing products for larger patient populations.

“With a shift beyond ultra-rare indications, viral-vector manufacturing requires rapid expansion to be able to address various diseases in the commercial space,” the analysts wrote, adding, “The broader application of viral vector-based gene therapies (for example, to more common diseases) requires higher yields and lower cost of goods (COGs).”

Scientists adding a human intelligence gene into monkeys — it’s the kind of thing you’d see in a movie like Rise of the Planet of the Apes. But Chinese researchers have done just that, improving the short-term memories of the monkeys in a study published in March 2019 in the Chinese journal National Science Review. While some experts downplayed the effects as minor, concerns linger over where the research may lead.

The goal of the work, led by geneticist Bing Su of Kunming Institute of Zoology, was to investigate how a gene linked to brain size, MCPH1, might contribute to the evolution of the organ in humans. All primates have some variation of this gene. However, compared with other primates, our brains are larger, more advanced and slower to develop; the researchers wondered whether differences that evolved in the human version of MCPH1 might explain our more complex brains.

Article from 2019

Continue reading “What Happens When Scientists Put a Human Intelligence Gene Into a Monkey?” »

The weaponization of the scientific and technological breakthroughs stemming from human genome research presents a serious global security challenge. Gene-editing pioneer and Nobel Laureate Jennifer Doudna often tells a story of a nightmare she once had. A colleague asked her to teach someone how her technology works. She went to meet the student and “was shocked to see Adolf Hitler, in the flesh.”

Doudna is not alone in being haunted by the power of science. Famously, having just returned home from Los Alamos in early 1945, John von Neumann awakened in panic. “What we are creating now is a monster whose influence is going to change history, provided there is any history left,” he stammered while straining to speak to his wife. He surmised, however, that “it would be impossible not to see it through, not only for military reasons, but it would also be unethical from the point of view of the scientists not to do what they knew is feasible, no matter what terrible consequences it may have.”

According to biographer Ananyo Bhattacharya, von Neumann saw what was happening in Nazi Germany and the USSR and believed that “the best he could do is allow politicians to make those [ethical and security] decisions: to put his brain in their hands.” Living through a devastating world war, the Manhattan Project polymath “had no trust left in human nature.”

Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.

Antonio, who’s been legally blind for much of his 14 years, can see again.

The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?

Integrated Biosciences, a biotechnology company combining synthetic biology and machine learning to target aging, in collaboration with researchers at the University of California Santa Barbara, today announced a drug discovery platform that enables precise control of the integrated stress response (ISR), a biological pathway that is activated by cells in response to a wide variety of pathological and aging-associated conditions.

A new publication, “Optogenetic control of the integrated stress response reveals proportional encoding and the stress memory landscape,” authored by company founders and featured on the cover of Cell Systems describes a technique that triggers the ISR virtually using light and demonstrates how the accumulation of stress over time shifts a cell’s reaction from adaptation to apoptosis (programmed cell death).

“In a very real way, our platform puts cells into a virtual reality, making them experience stress in the absence of physical stressors,” said Maxwell Wilson, Ph.D., a co-founder of Integrated Biosciences and Assistant Professor of Molecular, Cellular, and Developmental Biology at the University of California Santa Barbara.

Summary: This week’s neuroscience revelations encompass intriguing findings from the enigmatic genetics of mind-controlling hairworms to the groundbreaking link between alcohol use disorders and certain neuronal plasticity genes.

A novel theory proposes that the location of memory storage in the brain depends on its generalizability rather than age, adding a new dimension to our understanding of memory management. A new AI system demonstrates an impressive ability to identify violations of social norms, promising advancements in AI capabilities.

Continue reading “Brain Bites: Top 5 Neuroscience News Articles of the Week” »

We know a lot about cancer, and yet, there is plenty we do not yet know. We do know that some cancers are genetic in nature and a series of changes in key genes can lead to identifiable malignancies down the line. We would certainly want to know what causes cancer in the first place.

Scientists have been trying to replicate the path a cell takes from being normal to becoming pre-cancerous (one of the earliest stages of cancer in which cells become abnormally shaped and sized) for quite some time now. It is a feat that requires human-derived cells to model how cancer comes to be.

Recently, researchers at The Stanford School of Medicine have been able to emulate some of the earliest stages of gastric cancer by starting with gastric organoids (a rudimentary version of the real stomach made from stem-cell-derived gastric cells) that have a single mutation. The study which was published in Nature outlines how the earliest changes in cells could be seen even before the precancerous stage.