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Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform gene editing in non-liver tissues, including skeletal muscle, using a single adeno-associated virus (AAV) vector. Experiments in non-human primates (NHPs) resulted in editing efficiencies exceeding 30% in muscle tissues.

CRISPR gene editing has revolutionized genetics, but delivery challenges have restricted its clinical applications primarily to ex vivo and liver-directed therapies. Conventional CRISPR nucleases, including Cas9 and Cas12a, exceed the packaging limits of a single AAV vector, necessitating dual-AAV strategies that reduce efficiency.

Smaller CRISPR systems such as Cas12i and CasX have been identified, but they remain too large or exhibit low editing efficiency. Existing compact systems like Cas14 and IscB have not demonstrated robust efficacy in large animal models.

Northwestern Medicine scientists have discovered new details about how the human genome produces instructions for creating proteins and cells, the building blocks of life, according to a pioneering new study published in Science Advances.

While it’s understood that genes function as a set of instructions for creating RNA, and thus proteins and cells, the fundamental process by which this occurs has not been well-studied due to technological limitations, said Vadim Backman, Ph.D., the Sachs Family Professor of Biomedical Engineering and Medicine, who was senior author of the study.

“It is still not fully understood how, despite having the same set of genes, cells turn into neurons, bones, skin, heart, or roughly 200 other kinds of cells, and then exhibit stable cellular behavior over a human lifespan which can last for more than a century—or why aging degrades this process,” said Backman, who directs the Center for Physical Genomics and Engineering at Northwestern. “This has been a long-standing open question in biology.”

A team of Chinese scientists has used targeted gene editing to develop rice that produces coenzyme Q10 (CoQ10), a vital compound for human health.

Led by Prof. Chen Xiaoya from the CAS Center for Excellence in Molecular Plant Sciences/Shanghai Chenshan Research Center and Prof. Gao Caixia from the Institute of Genetics and Developmental Biology of the Chinese Academy of Sciences (CAS), the researchers used targeted gene editing to modify just five amino acids of the Coq1 rice enzyme, creating new rice varieties capable of synthesizing CoQ10.

The study is published in Cell.

Aging is a natural process, but for centuries, humans have been searching for ways to slow it down or even reverse it. Recent advancements in stem cell research and regenerative medicine have given scientists unprecedented insights into aging and potential interventions. With breakthroughs in cellular therapy, gene editing, and tissue engineering, we are closer than ever to finding ways to rejuvenate the human body. But how close are we to reversing aging, and what challenges remain?

Stem cells are the body’s raw materials from which all other specialized cells are generated. They have the unique ability to divide and create identical copies of themselves (self-renewal) or differentiate into specialized cell types. However, as we age, our stem cells decline in both number and efficiency, contributing to tissue degeneration, slower healing, and an increased risk of age-related diseases.

Researchers have been investigating how stem cells can be manipulated to repair damaged tissues, regenerate organs, and potentially reverse signs of aging. By harnessing stem cells, scientists aim to restore youthful function in various tissues and organs, offering promising anti-aging therapies.

When the immune system becomes unbalanced, it can lead to serious problems, such as type 1 diabetes, other autoimmune diseases, or organ rejection after a transplant. Current treatments often involve suppressing the entire immune system, which can cause severe side effects, including a higher risk of infections and other complications. A better approach would be to regulate the immune response in a precise and targeted way. That’s exactly what researchers have now achieved by engineering specialized immune cells designed to restore balance without compromising overall immunity.

Engineering Immune Cells to Protect Rather Than Attack

The immune system defends the body against viruses, bacteria, and other threats by identifying harmful invaders and mounting a response. It also distinguishes between the body’s own cells and foreign ones, adjusting its reaction as needed. However, when the immune system becomes dysregulated, it can mistakenly attack the body’s own tissues. This happens in conditions like type 1 diabetes, where the immune system destroys insulin-producing beta cells in the pancreas. It can also reject transplanted organs, treating them as foreign threats. While immunosuppressant drugs can prevent these harmful reactions, they come with serious risks, including increased vulnerability to infections and cancer.

Synthetically replicating transmembrane protein signal transduction is a gaol of synthetic biology. Here, the authors show how the dimerization of synthetic transmembrane DNA receptors can be used to engineer sensing and actuation cascades in response to external molecular signals.

Manu Prakash, an assistant professor of bioengineering at Stanford, and his students have developed a synchronous computer that operates using the unique physics of moving water droplets. Their goal is to design a new class of computers that can precisely control and manipulate physical matter. For more info: http://news.stanford.edu/news/2015/ju

Music: “Union Hall Melody” by Blue Dot Sessions.