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Prime editing, a mightier version of CRISPR/Cas9 technology, has been part of rigorous research and development in recent years. Now, U.S. regulators have greenlit the first-ever clinical trial for this technology.

Massachusetts-based Prime Medicine received the go-ahead from the U.S. Food and Drug Administration (FDA) after preclinical data showed that its candidate was able to correct mutations in chronic granulomatous disease (CGD).

CGD is a rare condition and affects around one in 200,000 people worldwide. It is caused by mutations in any of the six genes that code for the molecule nicotinamide adenine dinucleotide phosphate (NADPH), which is responsible for carrying electrons within cells. White blood cells called phagocytes don’t function properly, and as a result, they fail to protect the body from bacterial and fungal infections.

The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.

The human genome consists of around 3 billion base pairs and humans are all 99.6% identical in their genetic makeup. That small 0.4% accounts for any difference between one person and another. Specific combinations of mutations in those base pairs hold important clues about the causes of complex health issues, including heart disease and neurodegenerative diseases like schizophrenia.

Although this is still an emerging area of research, a new study has announced a leap. Researchers from the Center for Neuromorphic Engineering at the Korea Institute of Science and Technology (KIST) have implemented an integrated hardware system consisting of artificial neurons and synaptic devices using hexagonal boron nitride (hBN) material.

They aimed to construct building blocks of neuron-synapse-neuron structures that can be stacked to develop large-scale artificial neural networks.

“Artificial neural network hardware systems can be used to efficiently process vast amounts of data generated in real-life applications such as smart cities, healthcare, next-generation communications, weather forecasting, and autonomous vehicles,” said KIST’s Dr. Joon Young Kwak, one of the study’s authors, in a press release.

Autonomous and AI-enabled systems increasingly rely on optical and radio frequency sensors and significant computer power. They face growing vulnerabilities from directed-energy laser and microwave weapons.

By David C. Stoudt

In May the U.S. secretary of the Air Force flew in an F-16 that engaged in a mock dogfight over the California desert while controlled by artificial intelligence. Carmakers from San Francisco to Boston are jousting to deliver driverless cars. In Norway a crewless cargo ship carries fertilizer from port to port. On the land, sea and in the air, we face the coming of such autonomous platforms—some envisioned to benefit humanity, and others meant for destruction—available to everyone, to governments, businesses and criminals.

In a stunning scientific feat in the field of cryonics, a team from Fudan University in Shanghai achieved a monumental breakthrough by successfully reviving a human brain that had been frozen for as long as 18 months. This record breaking achievement not only shatters previous records in cryogenic technology but has also been published in the esteemed academic journal Cell Reports Methods.

The team led by Shao Zhicheng created a revolutionary cryopreservation method, dubbed MEDY, which preserves the structural integrity and functionality of neural cells, allowing for the preservation of various brain tissues and human brain specimens. This advancement holds immense promise not only for research into neurological disorders but also opens up possibilities for the future of human cryopreservation technology.

Professor Joao Pedro Magalhaes from the University of Birmingham K expressed profound astonishment at the development, hailing the technology’s ability to prevent cell death and help preserve neural functionality as nothing short of miraculous. He speculated that in the future, terminally ill patients could be cryopreserved, awaiting cures that may emerge, while astronauts could be frozen for interstellar travel, awakening in distant galaxies.