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Category: biotech/medical – Page 23

Without the right tests, the best medicines make no difference

A new analysis from UC San Francisco argues that diagnostics—medical tests that match patients to the appropriate treatment—are being overlooked both in the United States and around the world. This is slowing progress against major diseases, despite rapid advances in targeted therapies and precision health.

The authors note that nearly half of the world’s population lacks adequate access to diagnostics. These tests receive less investment for research and development, as well as lower insurance reimbursement than drugs, and this is creating barriers to innovation.

“Most people can easily understand how a new drug or surgery might help a patient,” said Kathryn Phillips, Ph.D., a professor of Health Economics in the School of Pharmacy at UC San Francisco and the lead author of the study, which appears in Science. “But the tests that guide medical decisions are just as critical.”

Novel gene-based therapy helps nerves heal better after severe injury

Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite surgery and advances in understanding and treating nerve injuries, many patients don’t get all their movement or feeling back.

Researchers at The Ohio State University College of Medicine and College of Engineering developed a new way to improve healing after severe nerve injuries by helping the body grow new blood vessels where the nerve is repairing itself. The new approach combines nerve graft surgery with tissue nanotransfection (TNT), a novel non-viral gene therapy developed at The Ohio State University.

Scientists used TNT to deliver three specific genes (Etv2, Fli1 and Foxc2) that tell cells to help form new blood vessels. These genes were applied via a very quick electrical pulse to nerve grafts used during surgery in mice with severe nerve injuries.

Your DNA has a secret “second code” that decides which genes get silenced

However, research is increasingly showing that these so-called synonymous codons are not truly equal. Some codons make mRNA molecules more stable and easier for cells to translate into proteins, making them more efficient. Others, considered non-optimal, lead to weaker translation and are more likely to be broken down. Until now, scientists have not fully understood how human cells recognize and respond to these less efficient codons.

Scientists Search for the Cell’s “Quality Control” System

To investigate this question, a research team from Kyoto University and RIKEN, led by Osamu Takeuchi and Takuhiro Ito, carried out a series of experiments aimed at uncovering how cells handle codon efficiency.

AI can design and run thousands of lab experiments without human hands. Humanity isn’t ready for the new risks this brings to biology

Faster protein engineering could mean faster responses to emerging infections and cheaper drugs.

The dual-use problem

Researchers have raised concerns that these same AI tools could be misused, a challenge known as the dual-use problem: Technologies developed for beneficial purposes can also be repurposed to cause harm.

TRUE-MOGAD ScoreA Novel Scoring System to Identify MOGAD Among Positive MOG-IgG Test Results

This study shows that a TRUE-MOGAD score of 2 or more accurately predicts MOGAD in patients with MOG-IgG titers of 1:20 or higher using the described assay. Read more.

Myelin oligodendrocyte glycoprotein (MOG) antibodies (MOG-IgG) are a biomarker of MOG antibody-associated disease (MOGAD). However, false positives remain common. We aimed to develop a scoring tool to guide interpretation.

Lab-grown pineal gland organoids produce melatonin, offering a new sleep model

Organoids are miniature, simplified versions of an organ. Over the past two decades, scientists have developed them for the gut, lung, liver, mammary gland, brain, and more. Now, researchers at Yale School of Medicine (YSM) have organoid-ized the pineal gland, a small structure in the brain that regulates sleep patterns through its production of the hormone melatonin.

In a study published in Cell Stem Cell, the researchers demonstrate how pineal gland organoids can be used to study sleep dysfunction in conditions like Angelman syndrome, autism, and depression.

“In a number of neuropsychiatric conditions, severe sleep problems are a major symptom,” says In-Hyun Park, Ph.D., associate professor of genetics at YSM and senior author of the study. “With pineal gland organoids, we may be able to uncover the causes of those sleep disturbances and possibly identify treatments.”

Little-used cholesterol test could prevent more heart attacks and strokes

A routine blood test taken by millions in the U.S. each year to measure “bad” cholesterol is not the best measure to guide treatment and prevent heart attacks and strokes, suggests a new Northwestern Medicine study published in JAMA. The study found that another blood test called apolipoprotein B (apoB) outperformed LDL and non-HDL cholesterol in guiding cholesterol-lowering therapy, such as taking statins and other medications.

“We found that apoB testing to intensify cholesterol-lowering medication would prevent more heart attacks and strokes than current practice, and that these health benefits were achieved at a cost that represents good value for U.S. health care payers,” said study lead author Ciaran Kohli-Lynch, assistant professor of preventive medicine in the division of epidemiology at Northwestern University Feinberg School of Medicine.

According to Kohli-Lynch, this is the first comprehensive study to show that using apoB testing to guide cholesterol-lowering treatment is cost-effective.

Double‐Pronged NAD Preservation: Delaying Cellular Senescence and Initiating Musculoskeletal Regeneration

A novel synergistic drug combination (N + A) consisting of an NAD+ precursor (NMN) and an NAD+ consumption (CD38) inhibitor (API) promotes musculoskeletal regeneration in aging. Notably, increased NAD+ serves as a coenzyme for SIRT3, exerting a robust anti-senescence effect, thus promoting tri-lineage differentiation into chondrocytes, osteoblasts, and myocytes. Furthermore, oral administration of the N + A formulation modulated the intestinal microenvironment, promoting the gut microbiota-derived production of the metabolite PHS, thereby exerting indirect anti-aging effects in musculoskeletal disorders.

A ‘stemness checkpoint’ helps control stem cell identity

A study published in Cell Research advances a central idea in stem cell biology by identifying a checkpoint that controls the identity of many different types of stem cells across developmental stages. For nearly two decades, scientists have understood that stem cell self-renewal depends on blocking differentiation signals—a concept described in earlier work, including Qi-Long Ying and Austin Smith’s 2008 Nature paper titled “The ground state of embryonic stem cell self-renewal.”

Now, researchers from the labs of Ying at USC and Guang Hu at the National Institute of Environmental Health Sciences (NIEHS), one of the National Institutes of Health (NIH), have identified the protein GSK3α as a “stemness checkpoint” that drives differentiation and that can be inhibited to maintain stem cell identity.

This discovery introduces a new conceptual framework: Rather than viewing stem cell maintenance as the result of many unrelated signaling conditions, distinct stem cell types share common checkpoints.

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