Five ways to apply CRISPR gene editing

CRISPR gene-editing technology has the potential to treat—and possibly cure—any number of diseases and there are currently five ways it can be delivered into humans.

This is a summary of some information from an article at MIT Technology Review Emily Mullin September 22, 2017.

1. Gels and creams for anti-HPV. – Among the first attempts to use CRISPR in humans is a trial planned in China for treating human papillomavirus, the most common sexually transmitted infection. Chinese researchers are developing a gel that contains DNA coding for the CRISPR machinery. The topical treatment is designed to inactivate the viral genes of HPV while leaving the DNA of healthy cells intact. The trial will enroll women with HPV infections; they’ll receive the gel twice a week for four weeks. The gel will be applied directly to the cervix.

2. Drinkable or edible CRISPR to kill specific bacteria

an-Peter Van Pijkeren at the University of Wisconsin-Madison, along with startup companies like Eligo Bioscience and Locus Bioscience, are developing CRISPR therapies that tell harmful bacteria to make fatal cuts to its own DNA. The CRISPR mechanism would be added to “good” bacteria, or a probiotic, that a person could swallow as a pill or a liquid. Unlike antibiotics, which kill both bad and beneficial bacteria, a drinkable or edible CRISPR probiotic would be specific to a patient’s bacterial infection, only killing the harmful germs.

3. Ear injections to treat genetic hearing loss

Zhen-Yi Chen, an ear specialist at Harvard, is interested in whether CRISPR could reverse gradual hearing loss. Delicate hair cells in the inner ear are what pick up sound and allow us to hear. These cells can become damaged by too much exposure to loud noises or a flaw in a person’s DNA. Mice tests have been successful and pigs are next.

4. Skin grafts agaist type 2 diabetes

CRISPR skin patches could be a needle-free way to manage type 2 diabetes. Researchers at the University of Chicago used the skin grafts on mice to reduce obesity and blood sugar levels. Scientists modified a gene that makes a hormone needed for insulin production. They then inserted the gene into skin cells, grew the cells in the lab, and grafted the gene-altered skin onto mice. After eating a high-fat diet, mice with the CRISPR skin grafts gained less weight than a control group that didn’t get the grafts. They also showed less insulin resistance, a precursor to type 2 diabetes. Xiaoyang Wu, an associate professor at the University of Chicago, says the grafts could be used for other diseases, too. For diabetic patients, he says, the biggest benefit is that they wouldn’t have to inject insulin daily.

5. Treat cells outside the body and then re-insert

Targeted gene-editing to select cells or tissues in the body, such as bone marrow cells. Ex vivo gene therapy allows researchers to remove certain cells, apply CRISPR to them, and culture them in a lab and then reinsert the modified cells.