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Category: genetics – Page 74

Immunotherapy drug eliminates aggressive cancers in clinical trial

Over the past 20 years, a class of cancer drugs called CD40 agonist antibodies have shown great promise—and induced great disappointment. While effective at activating the immune system to kill cancer cells in animal models, the drugs had limited impact on patients in clinical trials and caused dangerously systemic inflammatory responses, low platelet counts, and liver toxicity, among other adverse reactions—even at a low dose.

But in 2018, the lab of Rockefeller University’s Jeffrey V. Ravetch demonstrated it could engineer an enhanced CD40 agonist antibody so that it improved its efficacy and could be administered in a manner to limit serious side effects. The findings came from research on mice, genetically engineered to mimic the pathways relevant in humans. The next step was to have a clinical trial to see the drug’s impact on cancer patients.

Now the results from the phase 1 clinical trial of the drug, dubbed 2141-V11, have been published in Cancer Cell. Of 12 patients, six patients saw their tumors shrink, including two who saw them disappear completely.

The researchers demonstrate that an engineered antibody improves a class of drugs that has struggled to make good on its early promise.

Zooming in reveals a world of detail: Protein mapping technique reveals inner workings of cells

In the past decade, there has been significant interest in studying the expression of our genetic code down to the level of single cells, to identify the functions and activities of any cell through the course of health or disease.

The identity of a cell, and the way that identity can go awry, is critical to its role in many of the biggest health challenges we face, including cancer, neurodegeneration, or genetic and developmental disorders. Zooming in on single cells allows us to tell the difference between variants which would otherwise be lost in the average of a region. This is essential for finding new medical solutions to diseases.

Most single-cell gene expression experiments make use of a technology called single-cell RNA sequencing (scRNA-seq), which produces a map of exactly which genes are being copied out into short “transcripts” inside the nucleus. However, scRNA-seq only gives us a window into the intermediate step between the genetic code and the proteins which take care of (almost) all the tasks inside our bodies.

Optimizing how cells self-organize: Computational framework extracts genetic rules

One of the most fundamental processes in all of biology is the spontaneous organization of cells into clusters that divide and eventually turn into shapes—be they organs, wings or limbs.

Scientists have long explored this enormously complex process to make artificial organs or understand cancer growth—but precisely engineering to achieve a desired collective outcome is often a trial-and-error process.

Harvard applied physicists consider the control of cellular organization and morphogenesis to be an that can be solved with powerful new machine learning tools. In new research published in Nature Computational Science, researchers in the John A. Paulson School of Engineering and Applied Sciences (SEAS) have created a computational framework that can extract the rules that cells need to follow as they grow, in order for a collective function to emerge from the whole.

Promising new method could treat inherited diseases

An innovative method that uses modified versions of a bacterial virus effective at delivering treatments to human cells shows promise as a more inexpensive and efficient way to treat some deadly genetic diseases. Researchers from the School of Pharmacy at the University of Waterloo use a modified version of a bacterial virus called M13 to target specific human cells while

Gut microbiota linked to autism spectrum disorder progression in mice

Autism spectrum disorder (ASD) affects an estimated 1 in 31 children in the United States by 2025, and prevalence in East Asian countries, such as South Korea, Singapore, and Japan, may be even higher than those in the United States. Despite its increasing prevalence, the underlying causes of ASD remain poorly understood, and there are currently no curative, preventive, or treatment options available.

A research team from POSTECH and ImmunoBiome in Korea, led by Professor Sin-Hyeog Im, who also serves as the CEO of ImmunoBiome, has made a discovery that reveals a multi-faceted mechanism behind ASD. This study, published in the July issue of Nature Communications, in collaboration with Dr. John C. Park and Prof. Tae-Kyung Kim, demonstrates that the and host immune system together can influence the progression of ASD in a .

ASD has long been regarded as a genetically driven disorder. However, growing evidence suggests that environmental and microbial factors also play a role. The human gut harbors more than ten times as many microbial cells as human cells, and these microbes play vital roles in metabolism and the development of the immune system.

Recreating a Rare Mutation Could Grant Almost Universal Virus Immunity For Days

A rare genetic mutation appears to make people basically invulnerable to viruses – and it could potentially be harnessed as a therapy. Researchers have now shown this surprising viral protection can be replicated in mice and hamsters.

“We have yet to find a virus that can break through the therapy’s defenses [in cell culture tests],” explains Columbia University immunologist Dusan Bogunovic, who first discovered this unusual antiviral superpower 13 years ago.

The mutation, a deficiency in interferon-stimulated gene 15 (ISG15), causes a mild yet persistent inflammation across the body. Examining patients’ immune cells revealed they’d had the usual run of encounters with flu, measles, chickenpox, and mumps, yet they’d never reported feeling particularly ill as a result.

First-Of-Its-Kind Cell Transplant Brings a Cure For Diabetes Closer

A patient with type 1 diabetes has begun producing his own insulin after receiving a transplant of pancreatic cells.

For the first time in humans, these islet cells have been genetically edited so they wouldn’t be rejected by the patient, removing the need for immunosuppressant drugs.

Type 1 diabetes usually begins when the immune system mistakenly attacks the islet cells in the pancreas, which are responsible for producing insulin. The condition is usually managed with a careful diet and regular insulin injections, but an emerging treatment involves replacing the damaged islet cells with functional ones.

One Fruit, 1600 Compounds, Countless Health Benefits

Fresh grapes contain a potent mix of over 1,600 compounds that benefit heart, brain, skin, and gut health. New evidence suggests they deserve official superfood recognition, with benefits even at the genetic level.

A new article appearing in the current issue of the peer-reviewed Journal of Agriculture and Food Chemistry explores the concept of “superfoods” and makes a case that fresh grapes have earned what should be a prominent position in the superfood family. The author, leading resveratrol and cancer researcher John M. Pezzuto, Ph.D., D.Sc., Dean of the College of Pharmacy and Health Sciences at Western New England University, brings forth an array of evidence to support his perspective on this issue.

As noted in the article, the term “superfood” is a common word without an official definition or established criteria. Mainstream superfoods are typically part of the Mediterranean Diet and generally rich in natural plant compounds that are beneficial to a person’s health. Pezzuto addresses the broader topic of superfoods in detail, then makes the scientific case for grapes, noting that fresh grapes are underplayed in this arena and often not included with mention of other similar foods, such as berries.

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