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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.

Monoclonal antibodies are immune system proteins that are created in the lab. Antibodies are produced naturally by your body and help the immune system recognize germs that cause disease, such as bacteria and viruses, and mark them for destruction. Like your body’s own antibodies, monoclonal antibodies recognize specific targets.

Many monoclonal antibodies are used to treat cancer. They are a type of targeted cancer therapy, which means they are designed to interact with specific targets. Learn more about targeted therapy.

Some monoclonal antibodies are also immunotherapy because they help turn the immune system against cancer. For example, some monoclonal antibodies mark cancer cells so that the immune system will better recognize and destroy them. An example is rituximab, which binds to a protein called CD20 on B cells and some types of cancer cells, causing the immune system to kill them. B cells are a type of white blood cell.

Cleveland Clinic researchers analyzed genes and brain tissue of patients with Alzheimer’s and found that differences in brain immunometabolism – the interactions between the immune system and the ways cells create energy – may contribute to women’s increased risk for the disease and its severity.

The findings, published in Alzheimer’s and Dementia, offer important insight into developing sex-specific treatment and prevention options for Alzheimer’s disease, the sixth-leading cause of death in the United States.

“Our immune systems depend on communication between different cell types in our bodies, which are fueled by energy created from unique metabolic processes,” said Justin Lathia, Ph.D., vice chair of the Department of Cardiovascular and Metabolic Sciences and co-author on the paper. “As sex influences both the immune system and metabolic process, our study aimed to identify how all of these individual factors influence one another to contribute to Alzheimer’s disease.”

The next big advance in cancer treatment could be a vaccine that can shrink tumors and stop cancer from coming back. Among the targets for the experimental shots: melanoma, pancreatic cancer, breast cancer and lung cancer. Scientists are predicting new cancer vaccine approvals within five years (June 26) (AP Video by Manuel Valdes & Carla Johnson)

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Researchers in Texas have developed a method to keep a brain alive and functioning for several hours without being connected to the body — a truly weird scientific experiment that recalls the head in jars bit in the iconic cartoon “Futurama.”

A team led by the University of Texas Southwestern Medical Center in Dallas took two pigs and severed connections between their heads and bodies, instead hooking the brains up to a device they call the extracorporeal pulsatile circulatory control (EPCC), which they detailed in a paper published in the journal Scientific Reports. The machine keeps blood pumping through the brain, mimicking the natural flow when it’s connected to the rest of the body.

Continue reading “New Device Can Keep Brain Alive for Hours Without Body” »

A research team from the Hefei Institutes of Physical Science of the Chinese Academy of Sciences (CAS) has developed an analysis service platform called CRISPRimmunity, which was an interactive web server for identifying important molecular events related to CRISPR and regulators of genome editing systems. The study is published in Nucleic Acids Research.

The new CRISPRimmunity platform was designed for integrated analysis and prediction of CRISPR-Cas and anti-CRISPR systems. It includes customized databases with annotations for known anti-CRISPR proteins, anti-CRISPR-associated proteins, class II CRISPR-Cas systems, CRISPR array types, HTH structural domains and mobile genetic elements. These resources allow the study of molecular events in the co-evolution of CRISPR-Cas and anti-CRISPR systems.

To improve prediction accuracy, the researchers used strategies such as homology analysis, association analysis and self-targeting in prophage regions to predict anti-CRISPR proteins. When tested on data from 99 experimentally validated Acrs and 676 non-Acrs, CRISPRimmunity achieved an accuracy of 0.997 for anti-CRISPR protein prediction.

Phase III trial results on a precision radiotherapy technique support a “new gold standard” for treating head and neck cancer patients. The research suggests the new approach can reduce the risk of swallowing problems after radiotherapy, without impacting the success of treatment.

The Dysphagia-Aspiration Related Structures (DARS) trial compared dysphagia-optimized intensity-modulated radiotherapy (DO-IMRT) with standard IMRT.

DO-IMRT optimizes IMRT to reduce the risk of swallowing difficulties, known as dysphagia. This common side effect of radiotherapy for head and neck cancer can, in some cases, leave patients needing a permanent feeding tube. DO-IMRT lowers the risk of dysphagia by reducing radiation to the pharyngeal muscles, which support swallowing.

A recent study has raised questions about the impact of chronic caffeine consumption on our brain’s ability to adapt and learn. In a new study published in Frontiers in Psychiatry, scientists found that long-term caffeine users may exhibit decreased brain plasticity, a critical factor in the processes of learning and memory, when subjected to a brain stimulation protocol.

Caffeine is a common stimulant found in coffee, tea, soda, and other beverages. It’s known to help with alertness and concentration, but its effects on the brain’s ability to change and adapt over time, a process called plasticity, have been less clear.

Previous studies have shown mixed results when it comes to caffeine’s influence on brain plasticity. Some studies hinted that caffeine might hinder the brain’s ability to learn and adapt, while others suggested potential benefits. The researchers conducted this study to investigate the effects of caffeine on human brain plasticity, specifically focusing on its impact on long-term potentiation (LTP) and its potential interaction with a neuromodulation technique called repetitive transcranial magnetic stimulation (rTMS).

Veterinarians are encouraging dog owners to be cautious about a new mystery respiratory illness affecting dogs in the U.S.

Some of the first reports of the “atypical canine infectious respiratory illness,” which has symptoms similar to kennel cough and respiratory infections, appeared in Oregon in August.

“In August 2023, the Oregon Department of Agriculture (ODA) received reports of an atypical canine infectious respiratory illness being seen in dogs in the Portland Metro and Willamette Valley areas over the summer. To date, ODA has received over 200 reports of atypical canine infectious respiratory disease from Oregon veterinarians,” the Oregon Veterinary Medical Association shared in a release about the illness.