Ultrasound + gene therapy

CRISPR and ultrasound cured liver cancer in mice in 15 days

Svetlana Maslova, Hi-tech+

With the help of CRISPR/Cas gene editing, scientists managed to circumvent the main problem in the treatment of liver cancer — a protective reaction that increases the aggressiveness of cancer. It is equally important that such therapy has an effect only on cancer cells without harm to healthy ones.

Today, in the treatment of cancer, sonodynamic therapy is used — ultrasound in combination with drugs to release reactive oxygen species in the tumor. Meanwhile, tumor cells can activate antioxidant defense systems and resist treatment. Understanding the mechanism of this problem, scientists decided to use the CRISPR gene editing method to fight cancer. The results of their work are published in the journal ACS Central Science (Yin et al., Ultrasound-Controlled CRISPR/Cas9 System Augments Sonodynamic Therapy of Hepatocellular Carcinoma).

In liver cancer, the protective reaction to sonodynamic therapy is associated with the NFE2L2 gene, so scientists decided to reduce its expression. The CRISPR/Cas system and ROS precursor molecules were packed into lipid nanoparticles and treated with hepatocellular carcinoma cells. As a result of sonodynamic therapy, many more cells died than without suppression of NFE2L2.

The same approach was then tested on mice with hepatocellular carcinoma, the most common form of liver cancer.

After 15 days of therapy, all the tumors in the mice disappeared and the cancer did not return.

It is important to note that editing occurred only in tumor cells, without harming healthy ones, the authors specify. There is still a lot of work to be done, but scientists see great potential for a new approach to the treatment of cancer in humans.

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