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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 temporal lobes to shrink, resulting in changes in personality, behavior and speech, ultimately resulting in death.

Is pancreatic cancer hereditary? Are there any genetic mutations associated with it? Our pancreatic cancer expert Florencia McAllister, M.D., weighs in on these questions and seven more.

No questions concerning plasma dilution or E5, but a good interview with chapters.

Professor Matt Kaeberlein discusses the Dog Aging Project, longevity, Rapamycin, mTOR, and if we can ‘solve aging’

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Engineered immune cells, known as CAR T cells, have shown the world what personalized immunotherapies can do to fight blood cancers. Now, investigators have reported highly promising early results for CAR T therapy in a small set of patients with the autoimmune disease lupus. Penn Medicine CAR T pioneer Carl June, MD, and Daniel Baker, a doctoral student in Cell and Molecular Biology in the Perelman School of Medicine at the University of Pennsylvania, discuss this development in a commentary published today in Cell.

“We’ve always known that in principle, CAR T therapies could have broad applications, and it’s very encouraging to see early evidence that this promise is now being realized,” said June, who is the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine at Penn Medicine and director of the Center for Cellular Immunotherapies at Penn’s Abramson Cancer Center.

T cells are among the immune system’s most powerful weapons. They can bind to, and kill, other cells they recognize as valid targets, including virus-infected cells. CAR T cells are T cells that have been redirected, through genetic engineering, to efficiently kill specifically defined cell types.

A multi-gene expression signature in tumors is associated with aggressive disease and poor patient outcomes, and it has the potential to become a genetic cancer biomarker.

The human cell’s primary source of energy, the mitochondria plays an important role in the metabolism of cancer cells. In a study recently published in PLOS ONE, researchers from throughout the world, including Dario C. Altieri, M.D., president and chief executive officer, director of the Ellen and Ronald Caplan Cancer Center, and Robert and Penny Fox Distinguished Professor at The Wistar Institute, have identified a particular gene signature indicative of mitochondrial reprogramming in tumors that is associated with a poor patient outcome.