Lifeboat Foundation

Target validation is a crucial step in pre-clinical drug discovery workflows that builds confidence on the identification of a genetic target as relevant to a disease. With recent advancements, CRISPR serves as a particularly powerful tool for this process, as it enables researchers to accurately modify genes and determine their function in a variety of experimental systems.

One scientist leveraging CRISPR gene editing in this way is Dr. Panos Zalmas, Head of the Open Targets Validation Lab based at the Wellcome Sanger Institute, whose work focuses on discovering and validating new putative disease targets for the development of safe and effective medicines.

In this SelectScience^® interview, we speak with Zalmas to learn how he is working to improve the rate of target adoption into drug discovery pipelines across therapy areas such as oncology, neurodegeneration, and immunology and inflammation. Here, Zalmas explains the importance of gene editing in his target validation workflows and highlights how CRISPR technologies in particular are key to the success of drug discovery.

Design Cells / iStock.

The tool can correct genetic errors, control gene activity, and potentially treat diseases like cancer. However, its use raises ethical concerns regarding altering human genes and embryos.

New research suggests that biological age, as indicated by DNA methylation, more significantly impacts cognitive abilities like memory and processing speed than chronological age. This finding could reshape our understanding of aging and cognitive health.

Researchers have been able to reduce dramatically the level of bad cholesterol in human subjects after injecting them with an experimental gene editing treatment, according to the science journal Nature, which is the first time this technique, called base editing, has been done on humans.

But at least one person died after receiving an infusion, prompting a round of safety concerns.

In the clinical trial, 10 subjects with congenitally high levels of bad cholesterol, aka low-density lipoprotein (LDL), were given an injection of VERVE-101, a gene-editing treatment that uses the base editing technique. This treatment then turned off the gene for the protein PCSK9, which is found in the liver and regulates LDL. High levels of LDL can lead to coronary heart disease.

Veterinarian experts at Basepaws, a genetics testing company for pets in California, looked into the possibilities of how dog breeds of today will evolve 10,000 years down the line. The experts give their inputs to neural networks to generate some interesting visualizations.

Take a moment to see if you can recognize the breeds in the images below.

It is well known that modern-day dogs evolved from wolves that got friendly with humans. The exact timeline of when this friendship began is up for debate in the scientific community. But now that it has been established, it is unlikely that the bond will be shaken by anything in the future.

About 10–15% of pregnancies fail after conception has been recognized, amounting to 23 million losses a year. Chromosomal anomalies underlie many embryonic and fetal losses, but their exact frequency and localization to the embryo or placenta are still unclear. A new study published in Nature Medicine reports on a chromosomal analysis of over 1,700 spontaneous early miscarriages.

The most common period of pregnancy loss is before the ninth week, though many may occur earlier and pass unrecognized. While about 11% of women have at least one miscarriage, the proportion goes down with two or three, at 2% and 0.7. respectively.

CU Cancer Center member, Michelle Springer, MS, CGC, presents “Genetic Testing for Family Members” at the Hereditary Cancer Conference.

Nervous systems are complex networks, comprised of billions of neurons connected by trillions of synapses. These connections are subject to specific wiring rules that are thought to result from competitive selection pressures to minimise wiring costs and promote complex, adaptive function. While most connections in the brain are short-range, a smaller subset of metabolically costly projections extend over long distances to connect disparate anatomical areas. These long-range connections support integrated brain function and are concentrated between the most highly connected network elements; the hubs of the brain. Hub connectivity thus plays a vital role in determining how a given nervous system negotiates the trade-off between cost and value, and natural.
selection may favour connections that provide high functional benefit for low cost.

Consistent with this view, Professor Alex Fornito will present evidence.
that hub connectivity is under strong genetic control. He will show that the strength of connectivity between hubs in the human brain is more heritable than connectivity between other nodes, and that the genetic variants influencing hub connectivity overlaps with those implicated in mental illness and intelligence. He will also discuss the progress and challenges of developing generative models that evaluate the role of different cost-value trade-offs in driving complex brain topology.

Continue reading “Brain network hubs: maps, molecules, and models” »

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