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Is prime editing ready for prime time?

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.

Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells

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.

A new gene-editing system tackles complex diseases

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.

Chinese researchers successfully revive human brain frozen for 18 months

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.

Science has an AI problem: Research group says they can fix it

I found this on NewsBreak:#Publichealth #Computerscience #AI


AI holds the potential to help doctors find early markers of disease and policymakers to avoid decisions that lead to war. But a growing body of evidence has revealed deep flaws in how machine learning is used in science, a problem that has swept through dozens of fields and implicated thousands of erroneous papers.

Startup claims they have created AI head transplant system, plans to perform first procedure within decade

Scientists put their heads together for an insane medical breakthrough.

Neuroscience and biomedical engineering startup BrainBridge announced that it has created an AI-mechanized system for performing head transplants.

The procedure would graft a head onto the body of a brain-dead donor, maintaining the memories, cognitive abilities and consciousness of the transplanted individual.

Clogged Arteries Worsened by Cells that Behave like Cancer Cells

Columbia University researchers have found cells inside clogged arteries share similarities with cancer and aggravate atherosclerosis, raising the possibility that anticancer drugs could be used to treat atherosclerosis and prevent heart attacks.

Their study found that smooth muscle cells that normally line the inside of our arteries migrate into atherosclerotic plaques, change their cell identity, activate cancer genes, and proliferate inside the plaques.

“Our study shows that these transformed muscle cells are driving atherosclerosis, opening the door to new ways to treat the disease, potentially with existing cancer drugs,” says Muredach Reilly, MD, the Florence and Herbert Irving Endowed Professor of Medicine at Columbia University Vagelos College of Physicians and Surgeons and director of Columbia’s Irving Institute for Clinical and Translational Research.