Autoimmune diseases in three patients went into remission after experimental CRISPR therapy

Gong, 57 years old, cannot speak or grasp objects with his hands because his mouth and fingers are hardened. He suffers from systemic sclerosis, an autoimmune disease that affects the connective tissue of his body. Although he led a healthy lifestyle, one day his body decided to harden and thicken his skin.

A year ago, Gong underwent experimental treatments involving gene editing, bioengineering and donating third-party immune cells. A recent study from Shanghai Naval University, China, published in the journal cellconcludes that Gong’s disease is in the process of acquiring the status of “in remission”.

Chinese scientists introduced modified immune cells into Gong’s body using the CRISPR method. This method, one of the greatest revolutions in genetic treatment, allows sequences that cause errors in the body to be edited (or cut out) as if it were a book proofreader.

New approach to gene editing

Chimeric antigen receptor (TCAR) T cells have previously been used to treat some types of cancer. Until now, this treatment has only been applied to the patient’s blood cells due to the high rejection rate. In short, human cells are removed, then modified, fortified, and finally reintroduced. Therefore, the procedure is prohibitively expensive and scarce.

A recent study took a different approach. Instead of using Gong’s white blood cells, cells from a 21-year-old donor were modified. The researchers explain that they removed five genes from TCAR cells using CRISPR to prevent them from attacking the host. At the same time, the modifications made them invisible to the native immune system.

Sharing immune cells

Three patients with various autoimmune diseases were treated with TCAR cells from an anonymous donor. . This has reduced the costs associated with the extraction, transportation and modification of leukocytes. So far, none of the people tested have experienced side effects and their symptoms are under control.

“During 6 months of follow-up, we observed deep remission without cytokine release syndrome or other serious adverse events in all three patients. Our results demonstrate the high safety and promising immunomodulatory effects of available TCAR cells in the treatment of severe refractory diseases. autoimmune diseases,” the study concluded.

Just because a disease goes into “remission” does not mean it is cured. The term is used to describe the reduction or disappearance of signs of a disease. This is good news for any patient, but it does not relieve them of the need to continue monitoring their health or taking medications. For example, type 1 diabetes has recently been cured using stem cells, but it will only be considered “cured” if there is no change for five years.

The CRISPR gene editing method has enormous potential to improve people’s lives. In addition to being used to treat genetic diseases, researchers are looking to improve the resistance of crops, enhance endangered species and improve existing drugs.