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CGT are composed of a diverse group of medicinal products. Cell therapies (including ex vivo gene therapies) involve the transfer of cells with a relevant function into the patient. Cells can have different origins, i.e., human (autologous or allogeneic), different differentiation stages, i.e., stem cells or differentiated cells, and can be genetically modified to exert the intended therapeutic effect. In genetically modified cell therapy, a functional transgene is transfected into cells ex vivo using viral (for example, lentiviruses) or nonviral (e.g., electroporation) vectors. Next, the modified cells are administered to the patient where the transgene will promote a therapeutic effect. Examples of these therapies include chimeric antigen receptor (CAR) T cells and genetically modified human stem cells (HSCs).


This article highlights the importance of measuring potency for cell and gene therapies.

With the help of AI, researchers at Chalmers University of Technology, Sweden, have succeeded in designing synthetic DNA that controls the cells’ protein production. The technology can contribute to the development and production of vaccines, drugs for severe diseases, as well as alternative food proteins much faster and at significantly lower costs than today. How our genes are expressed is a process that is fundamental to the functionality of cells in all living organisms. Simply put, the genetic code in DNA is transcribed to the molecule messenger RNA (mRNA), which tells the cell’s factory which protein to produce and in which quantities.

Researchers have put a lot of effort into trying to control gene expression because it can, among other things, contribute to the development of protein-based drugs. A recent example is the mRNA vaccine against Covid-19, which instructed the body’s cells to produce the same protein found on the surface of the coronavirus. The body’s immune system could then learn to form antibodies against the virus. Likewise, it is possible to teach the body’s immune system to defeat cancer cells or other complex diseases if one understands the genetic code behind the production of specific proteins. Most of today’s new drugs are protein-based, but the techniques for producing them are both expensive and slow, because it is difficult to control how the DNA is expressed. Last year, a research group at Chalmers, led by Aleksej Zelezniak, Associate Professor of Systems Biology, took an important step in understanding and controlling how much of a protein is made from a certain DNA sequence.

“First it was about being able to fully ‘read’ the DNA molecule’s instructions. Now we have succeeded in designing our own DNA that contains the exact instructions to control the quantity of a specific protein,” says Aleksej Zelezniak about the research group’s latest important breakthrough. The principle behind the new method is similar to when an AI generates faces that look like real people. By learning what a large selection of faces looks like, the AI can then create completely new but natural-looking faces. It is then easy to modify a face by, for example, saying that it should look older, or have a different hairstyle. On the other hand, programming a believable face from scratch, without the use of AI, would have been much more difficult and time-consuming. Similarly, the researchers’ AI has been taught the structure and regulatory code of DNA. The AI then designs synthetic DNA, where it is easy to modify its regulatory information in the desired direction of gene expression.

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The technology, which was created by Barcelona-based researchers at the Centre for Genomic Regulation (CRG) and the Institute for Research in Biomedicine (IRB Barcelona), combines high-resolution microscopy with sophisticated computer modeling. It is the most comprehensive technique to date for studying the shape of genes.

The new technique allows researchers to create and digitally navigate three-dimensional models of genes, seeing not just their architecture but also information on how they move or how flexible they are. Understanding how genes function might help us better understand how they influence the human body in both health and disease since almost every human disease has some genetic basis.

Scientists at University of Florida (UF) Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.

The potential medication is described this week in the scientific journal Proceedings of the National Academy of Sciences. It is designed to be taken as a pill or an injection, said the lead inventor, professor Matthew Disney, Ph.D., chair of the UF Scripps chemistry department. Importantly, experiments showed that the compound is small enough to cross the blood-brain barrier, a hurdle other approaches have failed to clear, he said.

Amyotrophic lateral sclerosis, or ALS, progressively destroys neurons that control muscles, leading to worsening muscle loss and eventually death. The mutation, a leading cause of inherited ALS, is referred to as “C9 open reading frame 72,” or C9orf72. This mutation also leads to one form of frontotemporal dementia, a brain disease that causes the brain’s frontal and to shrink, resulting in changes in personality, behavior and speech, ultimately resulting in death.

A new technology developed at Tel Aviv University (TAU) makes it possible to destroy cancerous tumors in a targeted manner via a combination of ultrasound and the injection of nanobubbles into the bloodstream.

According to the research team, this latest technology enables the destruction of the tumor in a non-invasive manner, unlike invasive treatment methods or the injection of microbubbles into the tumor itself.

Accidental science is the best. One of the greatest lifesaving drugs in history – penicillin – was discovered purely by accident, and such experiments truly show how intricate and unpredictable science is.

So, when researchers from the University of Pennsylvania began their journey looking for a type 2 diabetes treatment and stumbled across a potentially incredible weight-loss treatment, needless to say, they were more than surprised.

The researchers describe their new results in a paper in the journal Science, in which they applied a treatment to a group of lab mice in the pursuit of counteracting type 2 diabetes. Instead of performing as expected, the experiment took a surprising turn – the mice started secreting a slimy substance through their skin.

Analysts and e-commerce leaders have been predicting a muted online holiday shopping season this year, with sales in the first three weeks of November essentially flat over a year ago due to a weaker economy, inflation, and more people returning to shopping in stores again in the wake of the Covid-19 pandemic. But on the face of it, the Thanksgiving long weekend appears to be more buoyant than expected — albeit growth has definitely slowed down this year after the pandemic-period boom.

Black Friday broke $9 billion in sales for the first time yesterday, with online sales of $9.12 billion, according to figures from Adobe Analytics. This is a record figure for the day, and up 2.3% on sales figures a year ago, and slightly higher than Adobe had estimated leading up to the day. Adobe doesn’t break out volumes in its report, so it’s hard to know if those figures are due to items simply costing more this year because of inflation, or if the higher numbers are a result of more buying.

Black Friday is a key focus for those gauging how the e-commerce market, and consumer confidence, are both faring in what is the most important and biggest period for shopping in the year.