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Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome to advance into clinical development.

In a new article, published today in Science Translational Medicine, Dr. Scott Dindot, an associate professor and EDGES Fellow in the Texas A&M School of Veterinary Medicine and Biomedical Sciences’ (VMBS) Department of Veterinary Pathobiology, and his team share the process through which they developed this novel therapeutic candidate, also known as 4.4.PS.L, or GTX-102. Dindot is also the executive director of molecular genetics at Ultragenyx, which is leading the development of GTX-102.

Angelman syndrome (AS) is a devastating, rare neurogenetic disorder that affects approximately 1 in 15,000 live births per year; the disorder is triggered by a loss of function of the maternal UBE3A gene in the brain, causing developmental delay, absent speech, movement or balance disorder, and seizures.

(CNN) — Startup Relativity Space sent what it’s calling the “world’s first 3D-printed rocket” toward space on Wednesday, vaulting it into the upper reaches of the atmosphere. Though, it suffered an engine issue after launch and failed to reach orbit.

Terran 1, a 110-foot-tall (33.5-meter) vehicle designed to haul lightweight satellites into orbital space, lifted off from Cape Canaveral Space Force Station on Florida’s eastern coast at just before 11:30 pm ET. The rocket, powered by super-chilled methane and oxygen, burned a bright blue-green against the night sky.

After the first stage of the rocket — the bottommost portion of the rocket that gives the initial thrust at liftoff — expended its fuel, it detached from the rocket’s upper stage. But the engine meant to propel that portion appeared to ignite only briefly, leaving the rocket without enough power to reach orbit.

More than 150 million women worldwide use oral contraceptives to avoid unwanted pregnancies. However, a bombshell study released by researchers at the University of Oxford has now laid bare the risks involved. The study has reportedly established the link between the usage of progestogen and the increased risk of breast cancer.

Scientists from German Cancer Research Center (DKFZ), together with colleagues from Germany, Israel, and the U.S., have found that the gut microbiome may modulate the efficacy of CAR-T cellular immunotherapy CAR-T cells in patients with B cell lymphomas. Individualized microbiome information retrieved from patients’ gut microbiomes prior to initiation of CAR T therapy could accurately predict their subsequent responsiveness to therapy, but only in the condition that these patients were not pre-treated with broad spectrum antibiotics.

Increasing evidence from human studies and preclinical experiments suggests that the gut microbiome may modulate the efficacy of T cell-driven cancer immunotherapies, such as immune checkpoint blockade. Immunotherapy with CD19 chimeric antigen receptor (CAR)-T cell has opened up new treatment options for patients with certain forms of refractory and relapsing B-cell leukemias or lymphomas. But the therapy is hampered by considerable heterogeneity in responses. Complete and long-term remission is only achieved in up to 40% of patients.

Researchers from multiple centers in Germany and the United States, led by Eran Elinav, director of the DKFZ-Weizmann Institute of Science Microbiome & Cancer Bridging division, have found that the gut microbiome may modulate the efficacy of CD19 CAR-T cell immunotherapy in patients with B cell leukemias and lymphomas.

In this exclusive excerpt from ‘Your Brain on Art,’ we learn how sounds and images are proving to measurably heal the brain.

Changes in the brain caused by Alzheimer’s disease are associated with shortening of the telomeres—the protective caps on the ends of chromosomes that shorten as cells age—according to a new study led by Anya Topiwala of Oxford Population Health, part of the University of Oxford, UK, published March 22 in the open-access journal PLOS ONE.

Telomeres on chromosomes protect DNA from degrading, but every time a cell divides, the telomeres lose some of their length. Short telomeres are a sign of stress and cellular aging, and are also associated with a higher risk of neurological and psychiatric disorders. Currently, little is known about the links between telomere length and changes that occur in the brains of people with neurological conditions. Understanding those relationships could offer insights into the biological mechanisms that cause neurodegenerative disorders.

In the new study, researchers compared telomere length in white blood cells to results from brain MRIs and electronic health records from more than 31,000 participants in the UK Biobank, a large-scale biomedical database and research resource containing anonymized genetic, lifestyle and health information from half a million UK participants.

Researchers have shown for the first time exactly how immature neutrophils—white blood cells that are an important part of the immune system—are hijacked by pancreatic cancers to drive immunosuppression and treatment resistance. The study, led by investigators at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, is published in Cancer Discovery, a journal of the American Association for Cancer Research.

The paper describes a previously unrecognized signaling circuit in pancreatic cancer that instigates immunosuppression and tumor-promoting inflammation in the pancreatic tumor microenvironment, ultimately creating treatment resistance. The central regulator of this treatment resistance is neutrophil-derived TNF signaling. TNF, or tumor necrosis factor, is a substance in the body that causes inflammation.

This is the first study to implicate immunosuppressive signaling from immature neutrophils—the earliest sentinels in developing pancreatic cancer—in this process, said Jashodeep Datta, M.D., associate director of Translational Research at the Sylvester Pancreatic Cancer Research Institute. Datta, whose laboratory at Sylvester led the research, is senior author of the multicenter study.

Argentinian-born mathematician Luis Caffarelli has won the 2023 Abel Prize — one of the most coveted awards in mathematics — for his work on equations that are important for describing physical phenomena, such as how ice melts and fluids flow. He is the first person born in South America to win the award.

Caffarelli’s results “are technically virtuous, covering many different areas of mathematics and its applications”, says a statement by Helge Holden, a mathematician at the Norwegian University of Science and Technology in Trondheim who chairs the Abel Committee.

The winner says receiving the news was an emotional moment, because “it shows that people have some appreciation for me and for my science”.

It’s no secret that OpenAI’s ChatGPT has some incredible capabilities—for instance, the chatbot can write poetry that resembles Shakespearean sonnets or debug code for a computer program. These abilities are made possible by the massive machine-learning model that ChatGPT is built upon. Researchers have found that when these types of models become large enough, extraordinary capabilities emerge.

But bigger models also require more time and money to train. The training process involves showing hundreds of billions of examples to a model. Gathering so much data is an involved process in itself. Then come the monetary and environmental costs of running many powerful computers for days or weeks to train a model that may have billions of parameters.

“It’s been estimated that training models at the scale of what ChatGPT is hypothesized to run on could take millions of dollars, just for a single training run. Can we improve the efficiency of these training methods, so we can still get good models in less time and for less money? We propose to do this by leveraging smaller language models that have previously been trained,” says Yoon Kim, an assistant professor in MIT’s Department of Electrical Engineering and Computer Science and a member of the Computer Science and Artificial Intelligence Laboratory (CSAIL).