Lifeboat Foundation

Machine learning is essential to designing the polymers, Murthy emphasizes, because they must be tailored to the specific gene therapy.

“There’s a tight interplay between the payload and in vivo mechanism of action, and the delivery vehicle needed to bring [the therapy] to that location,” he says. “You can’t have one without the other, so they have to be integrated at an early stage.”

The company hopes to use machine learning to explore the polymer design space, giving them a starting point to design a polymer. Subsequently, as the gene therapy moves from the preclinical to clinical stage, they can use artificial intelligence to tweak the polymer to make the therapy work better.

Researchers at the University of Wisconsin–Madison and Academia Sinica of Taiwan have harnessed a combination of lab-grown cells to regenerate damaged heart muscle.

The study is published in Circulation. It addresses major challenges of using heart muscle cells, called cardiomyocytes, grown from stem cells, and takes a crucial step toward future clinical applications.

Previous research has shown that transplanting cardiomyocytes made from induced pluripotent stem cells (iPSC) can replace muscle in the hearts of mammals. Researchers have struggled to bring the treatment to the clinic, in part because the implanted cells haven’t developed enough life-sustaining blood vessels to survive very long.

A UK-led team of researchers restrained mice for 6 hours to induce a stress response and then analyzed the rodents’ brains on a molecular level.⁠

This led to the discovery of increased levels of five microRNAs (miRNAs) — small molecules that help determine which genes in a cell are expressed and which aren’t — in the amygdala, the brain region implicated in anxiety. When the researchers took a closer look at the miRNA that reached the highest levels, miR-483-5p, they saw that it suppressed the expression of the Pgap2 gene — and that this suppression appeared to provide stress relief and reduce anxiety-related behavior.⁠

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Continue reading “Scientists discover ‘anxiety gene’ in the brain — and a natural way to turn it off” »

Nanotechnology sounds like a futuristic development, but we already have it in the form of CPU manufacturing. More advanced nanotech could be used to create independent mobile entities like nanobots. One of the main challenges is selecting the right chemicals, elements, and structures that actually perform a desired task. Currently, we create more chemically oriented than computationally oriented nanobots, but we still have to deal with the quantum effects at tiny scale.

One of the most important applications of nanotechnology is to create nanomedicine, where the technology interacts with biology to help resolve problems. Of course, the nanobots have to be compatible with the body (e.g. no poisonous elements if they were broken down, etc).

Continue reading “How nanobots and nanomedicine will improve our health” »

This Review discusses how embryonic transcriptional programs, such as epithelial–mesenchymal plasticity and stemness, may be harnessed in adult tissues to drive processes and diseases such as regeneration and cancer.

Nano electron guns can help zap cancers and drive new classes of lasers.

A cholesterol-lowering drug may help reduce the risk of heart failure in people with lymphoma who receive chemotherapy drugs called anthracyclines, results from a clinical trial suggest.

Anthracyclines, such as doxorubicin, are used to treat many types of cancer. But these drugs may affect the heart’s ability to pump blood, potentially leading to heart failure.

In the trial, atorvastatin (Lipitor) was found to reduce the risk of some cardiac changes linked to heart failure among patients treated with anthracyclines.

The anti–PD-1 agent tislelizumab was noninferior, but not superior, to the tyrosine kinase inhibitor sorafenib for unresectable hepatocellular cancer.

First-line treatment for patients with unresectable hepatocellular cancer (HCC) is either combination immunotherapy with bevacizumab and atezolizumab or tremelimumab and durvalumab. Although immunotherapy-based combination therapy has replaced tyrosine kinase inhibitors (TKIs) as initial therapy, randomized trials have not indicated superiority of single agent anti–PD-1 or PD-L1 therapy over TKIs.

Investigators have now conducted an industry-sponsored, global, open-label, randomized, phase 3 trial (RATIONALE-301) to compare first-line treatment with the anti–PD-1 agent tislelizumab versus the TKI sorafenib in 674 patients with Child-Pugh class A unresectable HCC. Of the patients, 85% were men, 58% had distant metastases, 76% had received prior locoregional therapy, 60% had hepatitis B, 13% had hepatitis C, and 3% had hepatitis B and C.

The primary endpoint of noninferiority for overall survival (OS) was achieved with tislelizumab compared with sorafenib (median 15.9 vs. 14.1 months; hazard ratio, 0.85; 95% confidence interval, 0.71–1.02), reaching the noninferiority margin upper limit of a hazard ratio 1.08. Superiority for OS with tislelizumab was not met. Antitumor response was numerically higher with tislelizumab than with sorafenib (14.3% vs. 5.4%), and median duration of response was longer with tislelizumab than with sorafenib (36.1 vs. 11.0 months). Median progression-free survival was similar with tislelizumab and sorafenib (2.1 and 3.4 months, respectively). Grade 3–4 treatment-related adverse events were more common with sorafenib than with tislelizumab (53.4% vs. 22.2%).

Continue reading “Tislelizumab vs. Sorafenib for Unresectable Hepatocellular Cancer” »

An international research consortium led by Ludwig Maximilian University of Munich (LMU) has tested a rapid new analytical tool which needs just a blood sample from the fingertip.

About 240,000 children worldwide die of tuberculosis every year. The disease is among the top 10 causes of death in children under the age of 5. One of the main reasons for this mortality is that tuberculosis is often misdiagnosed or not diagnosed in time, particularly in regions with limited resources.

A new diagnostic tool, which an international research consortium led by LMU medical scientists Laura Olbrich and Norbert Heinrich from the Division of Infectious Diseases and Tropical Medicine at LMU University Hospital Munich has tested as part of a large-scale study in five countries, offers significant progress in this area. The authors report on their findings in The Lancet Infectious Diseases.