CRISPR-Cas9, a unique technology that enables geneticists and medical researchers to edit parts of the genome by cutting out, replacing or adding parts to the DNA sequence, can be used to cure rare blood diseases, autoimmune diseases and cancer. This new technology has been thoroughly researched by a University of Connecticut alumnus, James Kozubek, in his new book, “Modern Prometheus: Editing the Human Genome with CRISPR-Cas9.”
“CRISPR-Cas9 has a specific GPS system associated with it,” James Kozubek, 2011 UConn masters in genetics grad, said. “You can program the device to a specific location or address of a genome and make genetic modification of it. It is an extremely precise and powerful thing to be able to do.”
"This completely changes how we can treat diseases and cancer," Kozubek said. "For the past 30 to 40 years, we have had the ability to splice genes or to randomly drop a gene into any chromosome in a cell, but we would have no control where is lands."
CRISPR-Cas9 was discovered in 2012 in bacteria. It was then repurposed to target human cells, Kozubek said.
“Basically the bacterial system that bacteria use to defend itself from some kinds of attacks, but by reprogramming it you can use it to alter genetically modify human cells,” Kozubek said.
"The first human trial with CASPR-Cas9 is moving ahead at the University of Pennsylvania since they have received support from the DNA advisory committee to move this technology into human cells," Kozubek said.
Kozubek was first introduced to CRISPR-Cas9 while volunteering with Derrick Rossi at the Harvard Department of Stem Cell and Regenerative Biology.
“It really lead me on this odyssey,” Kozubek said. “I ended up writing 500 pages of book written over four years. It was really this thing where I would go to work every day then I would come home and write for 14 hours over the weekend.”
During Kozubek’s quest for information about CRISPR-Cas9, he was able to meet bubble boy. Bubble boy is a young boy from Illinois who was born without an immune system. His family used gene therapy to alter his genetic code to help his immune system. Kozubek was one of the first people besides family to interact with and touch bubble boy since having his surgery.
“His mom was telling me about how she couldn’t have friends or go out. When she would go out to the grocery store, as soon as she would come home she had to take off all her clothes and wash them, scrub her hands vigorously and take a shower before seeing her son,” Kozubek said.
Bubble boy did not use the CRISPR-Cas9 technology to help his immune system; however, CRISPR-Cas9 could have been used in this case.
Bubble boy used a retrovirus to package gene therapy into T-cells to develop an immune system. This risk to this is that doctors had to randomly drop the cell into a genome, which could disrupt another gene, possibly leading to cancer.
However, the implications of CRISPR-Cas9 go beyond curing disease. Kozubek said this technology could be used for cosmetic reasons as well.
CRISPR-Cas9 can be used for in vitro fertilization techniques, Kozubek said. For example, if you wanted a child to have blonde hair, you could use this technology to change the genes of a fetus.
This is opening up several ethical discussions in terms of government regulation, Kozubek said.
“There is a sense of reality now that we are approaching a moment that in these next few years we can safely and effectively alter our code,” Kozubek said. “There are a whole range of opinions, but from what I understand the fed government has existing rules to handle these kinds of tools. Anything you do to a human cell is regulated as a drug under existing FDA rules.”
This technology is even being sold online, Kozubek said. People on the internet are selling gene modification kits to do these things themselves on bacteria and yeasts.
“You can use it to make yeast glow,” Kozubek said. “However the simplicity is concerning. What if you created a pathogenic strain?”
Kozubek said his book, on sale Sept. 30, could not have been written without the support of the strong community of scientists at UConn.
“I learned a tremendous amount,” Kozubek said. “The rigorous setting gave me confidence to go out into the world and write a book.”
Emma Krueger is a staff writer for The Daily Campus. She can be reached via email at firstname.lastname@example.org.