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Largest ever molecular map of autism opens new paths to precision therapies
For more than two decades, researchers have identified hundreds of genes that increase the risk of autism spectrum disorder (ASD). Yet multiple fundamental questions have remained unanswered: Among them, how do mutations in these genes lead directly to changes in brain development, and how can that knowledge be translated into more effective therapies?
In a new study published in Science, scientists at the Quantitative Biosciences Institute (QBI) and the Department of Psychiatry and Behavioral Sciences at the University of California, San Francisco (UCSF) have taken a major step toward answering both questions. The findings are the result of more than a decade of work. By building the largest molecular interaction map of autism, the team revealed how hundreds of genes and dozens of mutations converge within a surprisingly small number of shared protein networks, overcoming a major roadblock to the development of new precision medicines.
Rather than focusing only on the genes linked to autism, the researchers mapped the proteins encoded by those genes and discovered how individual disease-causing mutations can rewire the molecular machinery of the developing brain. The work uncovers an entirely new layer of disease biology, offering therapeutic targets and providing a framework for designing medicines that directly address a wide range of underlying molecular causes of autism.
Cell therapy substantially reduces severe rheumatoid arthritis in first clinical trial
Immunotherapies such as CAR T-cell therapy are used primarily to treat cancer. In the future, these patient-specific therapies, manufactured from patients’ own immune cells, could also help cure autoimmune diseases. Six patients with particularly severe rheumatoid arthritis have now received this treatment at Charité—Universitätsmedizin Berlin. In the journal Nature Medicine, the researchers report the results from the world’s first clinical trial of its kind: Disease activity decreased substantially in all participants. By the end of the observation period, three of the patients no longer required any medication for rheumatoid arthritis.
Rheumatoid arthritis is a chronic disease in which the immune system mistakenly attacks the body’s own joints. This causes recurrent inflammation and joint swelling and, as the disease progresses, can lead to joint damage. Currently available treatments can usually keep the inflammation under control but do not cure the disease. Patients therefore require lifelong medication, including anti-inflammatory drugs and medications that suppress the immune system, with all the associated side effects.
In some patients, even several of the newer treatments fail to produce an adequate response. Doctors then call the disease treatment-refractory. For those affected, this means persistent pain, restricted mobility and a substantial impact on quality of life.
How old are you really? Study explains what makes ‘epigenetic clocks’ tick, debuts new prediction tools
What do “epigenetic clocks” actually measure? These lab tests have become popular tools for studying biological aging, or how the body’s cellular function changes at a faster or slower rate than expected. However, the underlying biology behind each of these measurements has remained largely unknown until now.
Adam Becker on More Everything Forever and Tech’s Future Myths
Last summer I sat down with Adam Becker and asked him to name the most confident story in tech.
He picked the one nobody in Silicon Valley is allowed to question: that godlike #AI, digital immortality, and space empires are simply where history is headed. Not a hope. A destination.
Becker has a PhD in astrophysics and fifteen years as a science journalist. In his book More Everything Forever, he takes that story apart and shows where it actually came from: misread science fiction, fringe mailing lists, and a very old colonial logic about who deserves the future. From there it walked straight into university labs, congressional hearings, and your feed.
His line from our conversation stayed with me: Silicon Valley has confused science fiction with science, and science with branding.
The stakes are not academic. While we debate the welfare of trillions of hypothetical posthuman minds, the actual world runs on war, climate collapse, widening inequality, and a shared reality coming apart. Becker calls the grand visions a distraction. I asked him whether a civilization can function without a myth of the future at all.
I do not agree with everything Adam argues, and that is exactly why this one is worth your time. Watch it and tell me who you think is right about the #Singularity.