A groundbreaking cancer gene therapy can now be activated remotely at a specific location in the body. Researchers have developed a version of CRISPR that responds to ultrasound waves, showing its effectiveness in eliminating cancer in mice.
CRISPR, a precise genetic editing tool using the Cas9 enzyme, sometimes poses challenges by moving to unintended areas in the body and continuing to edit genes even when not required, which could trigger an immune response.
Scientists at the University of Southern California (USC) have devised a method to control the timing and location of CRISPR activity. In experiments with mice, they successfully used CRISPR to target and eliminate cancer cells.
In practical applications, CRISPR can be delivered into the body via virus carriers intravenously. Subsequently, focused ultrasound pulses can activate the gene editing tool at a specific site in response to the heat generated by the ultrasound waves.
Peter Yingxiao Wang, co-lead author of the study, explained, “Our controllable system allows you to turn it on and off as needed. Once activated, the CRISPR molecule will function precisely where directed. It will then naturally degrade after a period, remain inactive, and can be reactivated at any time.”
To combat cancer, the researchers targeted telomeres with CRISPR, prompting cancer cells to perish and initiating an immune response to eradicate tumors. Additionally, specialized CAR T cells were used to target CD19 protein, which is abundant in certain cancer types, with CRISPR amplifying its production.
In mouse models with skin tumors, the combination therapy of CRISPR and CAR T cells resulted in a 100% survival rate, completely eradicating the cancers. In comparison, mice receiving only CAR T cell therapy had a 40% survival rate.
While the results are promising, further research is needed to determine whether these benefits can be translated to human subjects. Future studies should focus on refining the technique and expanding its applications beyond CAR T cell therapy.
The study was published in Nature Communications.
Source: USC
