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Could This Biocomputer Revolutionize Neuroscience and Drug Discovery? Dive into the World of Human Brain Cells on a Chip!

Australian startup Cortical Labs unveils CL1, a groundbreaking biocomputer using human neurons on silicon chips. This fusion offers real-time learning and adaptation, revolutionizing neuroscience and biotech research. Could this be the dawn of bioengineered intelligence?

CRISPR gene editing in blood stem cells linked to premature aging effects: Study offers solutions

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, compromising their long-term ability to regenerate the blood system.

The study, published in Cell Reports Medicine, outlines new strategies to overcome this hurdle, improving both the safety and efficacy of -based therapies for inherited blood disorders.

The research was led by Dr. Raffaella Di Micco, group leader at SR-Tiget, New York Stem Cell Foundation Robertson Investigator and Associate Professor at the School for Advanced Studies (IUSS) of Pavia, in collaboration with Professor Luigi Naldini, Director of SR-Tiget, and several European research partners.

AI used to design immune-safe ‘zinc finger’ proteins for gene therapy

Machine learning models have seeped into the fabric of our lives, from curating playlists to explaining hard concepts in a few seconds. Beyond convenience, state-of-the-art algorithms are finding their way into modern-day medicine as a powerful potential tool. In one such advance, published in Cell Systems, Stanford researchers are using machine learning to improve the efficacy and safety of targeted cell and gene therapies by potentially using our own proteins.

Most human diseases occur due to the malfunctioning of proteins in our bodies, either systematically or locally. Naturally, introducing a new therapeutic protein to cure the one that is malfunctioning would be ideal.

Although nearly all therapeutic protein antibodies are either fully human or engineered to look human, a similar approach has yet to make its way to other therapeutic proteins, especially those that operate in cells, such as those involved in CAR-T and CRISPR-based therapies. The latter still runs the risk of triggering immune responses. To solve this problem, researchers at the Gao Lab have now turned to machine learning models.

Promising breakthroughs in amyotrophic lateral sclerosis treatment through nanotechnology’s unexplored frontier

This review explores the transformative potential of nanotechnology in the treatment and diagnosis of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder characterized by motor neuron degeneration, muscle weakness, and eventual paralysis. Nanotechnology offers innovative solutions across various domains, including targeted drug delivery, neuroprotection, gene therapy and editing, biomarker detection, advanced imaging techniques, and tissue engineering. By enhancing the precision and efficacy of therapeutic interventions, nanotechnology facilitates key advancements such as crossing the blood-brain barrier, targeting specific cell types, achieving sustained therapeutic release, and enabling combination therapies tailored to the complex pathophysiology of ALS.

Novel Treatment Based on Gene Editing Safely and Effectively Removes HIV-Like Virus from Genomes of Non-Human Primates

(Philadelphia, PA) – A single injection of a novel CRISPR gene-editing treatment safely and efficiently removes SIV – a virus related to the AIDS-causing agent HIV – from the genomes of non-human primates, scientists at the Lewis Katz School of Medicine at Temple University now report. The groundbreaking work complements previous experiments as the basis for the first-ever clinical trial of an HIV gene-editing technology in human patients, which was authorized by the Food and Drug Administration (FDA) in 2022.

The preclinical study, published online in the journal Gene Therapy, tested EBT-001, an SIV-specific CRISPR-Cas9 gene-editing therapy, in rhesus macaques. The study shows that EBT-001 effectively excises SIV from reservoirs – cells and tissues where viruses like SIV and HIV integrate into host DNA and hide for years – without any detectable off-target effects in animals. The work is a significant advance in the generation of a cure for HIV/AIDS in humans.

“Our study supports safety and demonstrates evidence of in vivo SIV editing of a CRISPR gene-editing technology aimed at the permanent inactivation of virus in a broad range of tissues in a large, preclinical animal model, using a one-time injection of the treatment,” said Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Microbiology, Immunology, and Inflammation, Director of the Center for Neurovirology and Gene Editing, Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine, and senior investigator on the new study.

Clinical trial on artificial blood cells to begin in Japan

A clinical study of artificial red blood cells that can be stored for transfusions in times of emergency will begin in Japan by next March, according to Nara Medical University.

The university aims to put the artificial cells into practical use by around 2030, it said in early July, in what would likely be a world first.

The development of the blood cells, designed for use in remote areas and disasters, comes as a blood shortage is expected at medical facilities due to a declining number of donors amid the country’s shrinking population.

Artificial cell-like structures mimic self-reproduction and release polymeric spores

Life on Earth possesses an exceptional ability to self-reproduce, which, even on a simple cellular level, is driven by complex biochemistry. But can self-reproduction exist in a biochemistry-free environment?

A study by researchers from Harvard University demonstrated that the answer is yes.

The researchers designed a non-biochemical system in which synthetic cell-like structures form and self-reproduce by ejecting polymeric spores.