New tools in molecular biology are significantly pushing the boundaries of new genetic therapies for human disease. While the U.S. has led the world in biomedical research advancements, our dominance may be slipping; at least in the new technologies launched by the genetic technique called CRISPR, which allows genome editing. China is now on the cutting edge in using this new technology in human clinical trials. Today, there are 10 clinical trials using CRISPR and nine of those are happening in China.

CRISPR is short for Clustered Regularly Interspaced Short Palindromic Repeats. Inbacteria, where CRISPR was first described and characterized, it acts as a primitive immune system that protects bacteria from invading viruses. Yes, bacteria are plagued by viruses too! Some clever molecular biologists recognized that CRISPR could be used for genome editing. Genome editing means making changes in a genome by design. This means we have a powerful new tool to correct genetic mistakes and to alter the properties of human cells for purposes like fighting cancer.

In the journal Science, a story reviewed the status of bringing CRISPR therapies to the clinic to benefit humans. China is pushing far ahead with applying this technology to people. In 2015, Chinese scientists reported the use of CRISPR to edit the genome of a human embryo. This sent shock waves around the scientific world. Now other Chinese scientists are using CRISPR to alter human immune cells to enhance their cancer fighting properties.

One new approach involves using this genetic technique to inactivate a gene for a protein called programmed cell death protein 1 (PD-1). PD-1 causes our immune cells to self-destruct, and it is activated by cancer cells. Inactivating PD-1 means that immune cells do not self-destruct and remain efficient at destroying the cancer cells. Different groups across China are using variations of this approach to attack cancers in the prostate, lungs, bladder and throat. Another Chinese group has used CRISPR technology to inactivate two genes and replace them with another to make a certain kind of immune cell recognize and destroy leukemia cells.

CRISPR can also be used to treat diseases other than cancer. It can be used to control HIV infections. To do this, scientists inactivate a certain protein on T cells, which HIV uses to infect new cells. This short-circuits HIV infections by preventing the infection of additional cells. Another project is designed to destroy human papillomaviruses directly in women’s cervixes, which is pretty cool. Another one yet uses CRISPR to repair the mutated gene that leads to a genetic blood disorder called β- thalassemia.

There is amazing potential in this new technology for treating cancers and genetic disorders. The U.S. public is highly supportive of these approaches to treat human disease, but the technology is controversial because of the fears it could be used to create “customized” babies with desirable traits. Time will tell if U.S. scientists can compete with Chinese scientists in bringing CRISPR technologies to the clinic to treat human disease.

Medical Discovery News is hosted by professors Norbert Herzog at Quinnipiac University, and David Niesel of the University of Texas Medical Branch.  Learn more at www.medicaldiscoverynews.com.

Read or Share this story: https://arnne.ws/2DXdXXo