Some call it the most groundbreaking scientific discovery of the 21st century and now experts are studying the gene editing technique at the Medical College of Georgia.
CRISPR is a family of DNA sequences in your genome. It is also the name of a technique in which disease-causing mutations can be taken out and replaced with good, healthy versions. CRISPR is paving the way for doctors to be able to correct or even prevent genetic diseases. MCG just hired two experts in the field.
“We’re already working with people all over the country,” says Dr. Lin Gan.
Neurodevelopmental biologist and geneticist Dr. Lin Gan and experimental pathologist and molecular biologist Dr. Joseph Miano came to MCG this fall to continue their research. The pair were previously conducting research at the University of Rochester in New York.
“CRISPR is an editing system that basically can, with precision, go and change any letter of the alphabet of our book of life,” says Dr. Miano about the technique he and Dr. Gan are using to learn more about the human genome.
Dr. Miano says CRISPR acts like spellcheck when a misspelled word is fixed when the wrong letter is deleted and replaced with the correct one.
“For example, sickle cell anemia, there is a one letter mutation that causes that awful disease. CRISPR will allow us to go in and with those molecular scissors, change that mutation to the correct one,” Dr. Miano explains.
Dr. Miano points out, most mutations happen in unknown territory.
Think of the genome as an instruction manual. We understand about 1.5% of the instruction manual, but 99% of it we have no clue of,” Dr. Miano says.
Therefore, in a process called reverse genetics, Dr. Gan says they can use CRISPR to help figure out what disease is associated with a specific mutation.
“We know they are there. We can use CRISPR to manipulate and say to mutate and make a model to see what is the function of the particular gene so that way we can say if we change and alter that gene in the eye and then [we can see] whether we have any kind of disease associated with vision,” Dr. Gan explains.
Dr. Gan and Miano will be doing of lot of this human disease modeling at MCG.
“We take a large piece of the instruction manual, the DNA, and we put it into the mouse genome and so in that way, we can study the human gene in the mouse and use CRISPR to edit that in any way we’d like,” says Dr. Miano.
Dr. Miano says it is reasonable to assume scientists will be able to eradicate some diseases using gene editing techniques like CRISPR in the future.
“Diseases like sickle cell, duchenne muscular dystrophy, these diseases are going to be… tackled within the next 5 years,” Dr. Miano says. “These are well defined mutations, but we didn’t have a real simple method of correcting them. CRISPR now is just a game changer. It allows us to, with precision, go in and fix those mutations and therefore cure the disease.”
This year, award winning film makers released a documentary about CRISPR. It is currently screening in Europe and played for the first time at the SXSW Conference and Festival in Austin back in March.