CRISPR, short for clustered regularly interspaced palindromic repeats, is a genetic tool used to alter specific genetic codes within organisms to achieve desired changes. It differs from genetic modification, which replaces genes or genetic pairs.
But the scope of areas where CRISPR could be effective is relatively narrow compared to genetically modified organisms or GMOs, according to UW-Madison professor Krishanu Saha.
Saha and Jo Handelsman, director of the Wisconsin Institute for Discovery, presented at a recent Wisconsin Technology Council virtual luncheon the possibilities surrounding CRISPR. This came after three Wisconsin groups received grants from the National Institute of Health to fund CRISPR research.
One group is working on methods to safely deliver CRISPR cells to the human brain to potentially treat Alzheimer’s disease, but Saha said it could be used to treat other brain diseases if proven successful.
Another group is using the grant to develop models of human eye tissues so other CRISPR models designed to treat eye diseases and blindness can be quickly tested.
Saha said the third grant went to the Medical College of Wisconsin to fund a coordination effort between 20 groups nationwide that received similar CRISPR grants from NIH.
Handelsman said the gene-editing technology could be especially effective in treating sickle cell disease because the edit necessary to reverse the effects is much simpler than modifying the entire gene like older technologies.
CRISPR could also play a vital role in providing drought- and pest-resistant crops as the effects of climate change ramp up in the future.
“All of a sudden perhaps CRISPR plants will be more appealing in places that they’ve been banned, it’ll be a matter of survival,” said Handelsman.
Many of the gene therapy products on the market today are fairly expensive due to their high value for patients, Saha said. But these older products aren’t as effective as CRISPR could be in some cases, he added.
Other therapies using gene addition, akin to GMOs, add a gene “randomly” into the genome, he said. “And what the CRISPR tool allows us to do is much more precise modifications in specific spots in the genome.”