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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.

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.

Discover the power of gene editing, including the widely used CRISPR-Cas9 technology, and explore major publicly traded CRISPR companies.

The groundbreaking gene-editing technology known as Crispr, which acts like a molecular pair of scissors that can be used to cut and modify a DNA sequence, has moved rather quickly from the pages of scientific journals to the medical setting. Earlier this month, about three years after Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in Chemistry for describing how bacteria’s immune system could be used as a tool to edit genes, regulators in the U.K. approved the first Crispr-based treatment for sickle cell disease and beta-thalassemia patients. The treatment, from Vertex Pharmaceuticals and Crispr Therapeutics, could be approved by the U.S. Food and Drug Administration early next month for sickle cell patients.

While many obstacles lie ahead for the nascent field, such as how to pay for treatments that typically cost more than $1 million, these regulatory approvals are just the start as newer gene-editing technologies such as base and prime editing make their way through human studies. In an interview, Prof. Doudna says the approval is “a turning point in medicine because it really shows how genome editing can be used as a one-and-done cure for disease.”

Gene editing is part of a broader therapeutic revolution that encompasses genetic and cellular medicine. The pills and injections we are all familiar with generally target proteins or pathways in the body to treat disease. With gene and cell therapy, we can now target the root cause of disease, sometimes curing patients.

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Cell toxicity and genomic instability are potential side effects from the use of CRISPR-Cas9. The gene editing tool can also cause large rearrangements of DNA through retrotransposition to theoretically trigger tumor development.

While rare, the fact that CRISPR is used to edit millions of cells for some therapies means precautionary steps are warranted given the potential increase in cancer risk. However, retrotransposition is much rarer during base editing, a more precise technique that chemically changes just one “letter” of the genetic code without causing a double-strand break in DNA.

Although MHRA decided that the benefits of Casgevy outweigh its risks, the U.K. regulator granted a one-year conditional marketing authorization of the world-first gene therapy based on the findings of two global clinical trials, noting that no significant safety concerns were identified during the trials.

The bioengineered potato plant provides a reliable indicator of potentially hazardous radiation levels without requiring complicated sensor machines or monitoring methods.

UTIA

Referred to as a phytosensor, it is a type of sensor or detector that detects certain compounds or environmental conditions by using plants (phyto-meaning plant).

Biological computing machines, such as micro and nano-implants that can collect important information inside the human body, are transforming medicine. Yet, networking them for communication has proven challenging. Now, a global team, including EPFL researchers, has developed a protocol that enables a molecular network with multiple transmitters.

First, there was the Internet of Things (IoT) and now, at the interface of computer science and biology, the Internet of Bio-Nano Things (IoBNT) promises to revolutionize medicine and health care. The IoBNT refers to biosensors that collect and process data, nano-scale Labs-on-a-Chip that run medical tests inside the body, the use of bacteria to design biological nano-machines that can detect pathogens, and nano-robots that swim through the bloodstream to perform targeted drug delivery and treatment.

“Overall, this is a very, very exciting research field,” explained Assistant Professor Haitham Al Hassanieh, head of the Laboratory of Sensing and Networking Systems in EPFL’s School of Computer and Communication Sciences (IC). “With advances in bio-engineering, synthetic biology, and nanotechnology, the idea is that nano-biosensors will revolutionize medicine because they can reach places and do things that current devices or larger implants can’t,” he continued.

Advancements in genetic engineering, gene therapies, and anti-aging research may eventually allow for age reversal and the restoration of youthful health and longevity.

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—The key idea is that advancements in genetic engineering and anti-aging research may eventually allow for age reversal and the restoration of youthful health and longevity.

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